AzTrials
AzTrials

MELASPhase 1NCT02544217

A Dose-escalating Clinical Trial With KH176

A Phase 1 study testing KH176 in people with MELAS and LHON, run by Khondrion BV at 1 site in Belgium.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial diseases, including MELAS (Mitochondrial Encephalomyopathy, Lactic acidosis, and… Read more about this study

Who is it for?
Ages 18 to 55 years, men onlyHealthy volunteers can take part
Conditions the study is for
MELAS, LHON, Leigh Syndrome
What is being tested?
KH176
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
32 participants
Where
1 siteBelgium
Study dates
May 2015to Oct 2015
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 55 years
  • Men only
  • Healthy volunteers accepted
  • Adult

Who may be able to join

  • Healthy as assessed by medical history, physical examination, Vital Signs, Clinical Laboratory, ECG

Who cannot join

  • Allergies,
  • Concomitant medication,
  • concomitant disease,
  • relevant surgery,
  • recent blood donation

Trial locations and contacts

1 site in 1 country.

Belgium1 site

  • Drug Research Unit Ghent

    Ghent

Other trials in Belgium

About this study

Official title: A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176

Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial diseases, including MELAS (Mitochondrial Encephalomyopathy, Lactic acidosis, and Stroke-like episodes), Leigh's Disease and Leber's Hereditary Optic Neuropathy (LHON). KH176 is a potent intracellular redox modulating agent targeting the reactive oxygen species which are important in the pathogenesis of disorders of mitochondrial oxidative phosphorylation. After demonstrating a favourable safety profile in the pre-clinical testing, the safety, tolerability and pharmacokinetic and pharmacodynamic characteristics of the compound will now be evaluated in healthy male subjects in this trial

Being tested
KH176, placebo
Medical subject headings
MELAS Syndrome, Leigh Disease, Mitochondrial Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KH176Drug

  • placeboDrug

Study groups in NCT02544217
Study groupRoleWhat they receive
Single EscalatingExperimental2 alternating groups receiving escalating single doses of active/placebo
Receives: Drug: KH176; Drug: placebo
Multiple EscalatingExperimental3 multiple escalating groups, receiving active/placebo
Receives: Drug: KH176; Drug: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF)

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation of the corrected QT intervals the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing. QTcF is calculated as the quotient between the QT interval in milliseconds and the cube root of the RR interval in seconds, according to Fridericia's formula.

    Measured: Baseline, 1, 2, 4, 6, 8, 12, 24 hours, 7 day follow up

  2. Pharmacodynamics of KH176

    Change from baseline in biochemistry related to Oxidative Phosphorylation (OXPHOS) (glutathione, lactate); MAD group

    Measured: Day 1, day 7

  3. Relationship to Study Drug and Severity of Treatment-emergent Adverse Events

    Measured: 4 months

  4. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)

  5. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Sedimentation Rate (SAD Group)

    Measured: Baseline (pre-dose Day1), 24h post dose, FU (7 days after last dosing)

  6. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Sedimentation Rate MAD Group

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days after last dosing)

  7. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit SAD Group

    Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24 h postdose, FU (7 days after last dosing)

  8. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Haemoglobin. SAD

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24 h post dose, FU (7 days after last dosing)

  9. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Hemoglobin Concentration - SAD

    Erythrocyte mean corpuscular hemoglobin concentration (MCHC) is a measure of the average concentration of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24 h post dose, FU (7 days after last dosing)

  10. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Volume - SAD

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24 h post dose, FU (7 days after last dosing)

  11. MAD: Change From Baseline in ECG Results by Time Point: QTcF

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation of the corrected QT intervals the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple-dose part.

    QTcF is calculated as the quotient between the QT interval in milliseconds and the cube root of the RR interval in seconds, according to Fridericia's formula.

    Measured: Baseline, Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7

  12. Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)

  13. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  14. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Neutrophils. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)

  15. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit MAD Group

    Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  16. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)

  17. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Thrombocytes. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h, FU (7 days post-dose)

  18. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value ateach timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post-dose)

  19. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post-dose)

  20. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Volume. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  21. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  22. Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count. MAD

    Blood samples were collected from participants at the indicated time points for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each time point are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  23. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Neutrophils. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  24. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Haemoglobin Concentration. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  25. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  26. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Hemoglobin. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  27. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  28. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  29. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  30. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Thrombocytes. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  31. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)

  32. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Aspartate Aminotransferase. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  33. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Total Protein. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  34. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Gamma-GT. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  35. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  36. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Creatinine. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  37. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Chloride. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  38. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Total Bilirubin. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  39. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Alanine Aminotransferase. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  40. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Creatinine Kinase. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  41. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Urea. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  42. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Sodium. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  43. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Potassium. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  44. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Phosphate. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  45. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Calcium (Corrected for Albumin). SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  46. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Uric Acid. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  47. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Triiodothyronine (T3). SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  48. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Lipase. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  49. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: (Fasting) Glucose. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  50. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Human Serum Albumin. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  51. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Cholesterol. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  52. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: High Density Lipoproteins. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  53. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Triglycerides. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  54. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Low Density Lipoproteins. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  55. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Bicarbonate. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  56. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Thyroid-stimulating Hormone. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  57. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Thyroxine (T4). SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  58. Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Lactate. SAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)

  59. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Total Protein. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline (pre-dose Day1), Day 3, Day 8, FU (one week after last dosing)

  60. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Alkaline Phosphatase. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  61. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Aspartate Aminotransferase. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  62. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Creatine Kinase. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  63. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Gamma GT. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  64. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Total Bilirubin. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  65. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Urea. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  66. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Creatinine. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  67. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Sodium. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  68. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Potassium. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  69. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Chloride. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  70. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Calcium (Corrected for Albumin). MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  71. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Uric Acid. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  72. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Lipase. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  73. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Alanine Aminotransferase. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  74. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Phosphate. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  75. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Human Serum Albumin. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  76. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Fasting Glucose. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  77. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Cholesterol. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  78. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Triglycerides. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  79. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Low Density Lipoproteins. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  80. Change From Baseline in Chemistry Laboratory Test Results by Time Point: High Density Lipoproteins. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  81. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Thyroid-stimulating Hormone. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  82. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Trilodothyronine (T3). MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  83. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Thyroxine (T4). MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  84. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Bicarbonate. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  85. Change From Baseline in Chemistry Laboratory Test Results by Time Point: Lactate. MAD Group

    Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.

    Measured: Baseline, Day 3, Day 8, FU (one week after last dosing)

  86. Phospholipidosis

    Change from Day 1 to Day 7 in di-docosahexaenoyl (22:6)-bis(monoacylglycerol) phosphate (di-22:6-BMP) and normalized di-22:6-BMP (urine) - MAD

    Measured: Day 1, Day 7

  87. MAD: Change From Baseline in ECG Results by Time Point: Corrected QT Interval According to Barret's Formaula (QTcB)

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple- dose part.

    Measured: Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up

  88. MAD: Change From Baseline in ECG Results by Time Point: P Wave-Q Wave Interval (PQ Interval)

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation of the PQ interval the average of the 3 recordings will be taken as baseline.

    Measured: Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up

  89. MAD: Change From Baseline in ECG Results by Time Point: the Interval That Denotes Depolarization of the Ventricles, Between the Beginning of the Q Wave and the End of the S Wave (QRS Interval)

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple- dose part.

    Measured: Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up

  90. MAD: Change From Baseline in ECG Results by Time Point: QT Interval

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple- dose part.

    Measured: Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up

  91. SAD: Change From Baseline in ECG Results by Time Point: PQ Interval

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.

    Measured: Pre-dose, Day 1, Day 7

  92. SAD: Change From Baseline in ECG Results by Time Point: QRS Interval

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.

    Measured: Pre-dose, Day 1, Day 7

  93. SAD: Change From Baseline in ECG Results by Time Point: QT Interval

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.

    Measured: Pre-dose, Day 1, Day 7

  94. SAD: Change From Baseline in ECG Results by Time Point: QTcB Interval

    The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.

    Measured: Pre-dose, Day1, Day 7

  95. Terminal Elimination Half-life (T1/2) of KH176 Over 24 Hours: SAD

    Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  96. Terminal Elimination Half-life (T1/2) of KH183: SAD

    Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  97. Maximum Concentration (Cmax) of KH176: SAD Group

    Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  98. Maximum Concentration (Cmax) of KH183 Over 24 Hours: SAD

    Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  99. Maximum Concentration (Cmax) of KH183 (Active Metabolite of KH176): MAD Group

    Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.: Cmax was obtained directly from the concentration-time data.

    Measured: Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  100. Maximum Concentration (Cmax) of KH176: MAD

    Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  101. Time to Maximum Concentration (Tmax) of KH176 Over 24 Hours: SAD

    Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  102. Time to Reach Peak Plasma Concentration (Tmax) of KH183: SAD

    Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  103. Time to Maximum Concentration (Tmax) of KH176 at Day 1, Day 7: MAD Group

    Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  104. Time to Maximum Concentration (Tmax) of KH183 (Active Metabolite of KH176): MAD Group

    Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.. tmax was obtained directly from the concentration-time data.

    Measured: Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  105. Accumulation Factor (Racc) of KH176 Over 7 Days: MAD Group

    Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.. Racc was calculated as follows: AUCtau day 7/ AUCtau day 1.

    Measured: Pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  106. Accumulation Factor (Racc) of KH183 (Active Metabolite of KH176): MAD Group

    Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.: Accumulation factor was calculated as follows: AUCtau day 7/ AUCtau day 1.

    Measured: Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  107. Area Under the Plasma Concentration Versus Time Curve (AUClast) of KH183: SAD Group

    Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  108. Area Under the Plasma Concentration Versus Time Curve (AUClast) of KH176: SAD Group

    Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  109. Area Under the Plasma Concentration-time Curve (AUCtau) of KH176: MAD Group:

    Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: Pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  110. Area Under the Plasma Concentration-time Curve During a Dose Interval (AUCtau) of KH183 (Active Metabolite of KH176): MAD Group

    Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours

  111. Area Under the Plasma Concentration-time Curve From Time Zero Until Infinity (AUCl0-inf) of KH183: SAD

    Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  112. Area Under the Plasma Concentration-time Curve From Time Zero Until Infinity (AUCl0-inf) of KH176: SAD

    Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.

    Measured: Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose

  113. KH176: Percentage of Administered Dose Excreted in Urine: SAD

    Urine concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: 24 hours post-dose

  114. KH183: Percentage of Administered Dose Excreted in Urine: SAD

    Urine concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: 24 hours post-dose

  115. KH176 + KH183: Percentage of Administered Dose Excreted in Urine: SAD

    Urine concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.

    Measured: 24 hours post-dose

  116. KH176: Percentage of Administered Dose Excreted in Urine: MAD

    Urine concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: Day 7 post dose

  117. KH183: Percentage of Administered Dose Excreted in Urine: MAD

    Urine concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: Post dose Day 7

  118. KH173 + KH183: Percentage of Administered Dose Excreted in Urine: MAD

    Urine concentrations of KH176 and KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.

    Measured: Day 7 Post dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Sep 2015 (estimated)
  2. Study startMay 2015
  3. Primary completionSep 2015
  4. Study completionOct 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
32 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02544217
Sponsor's protocol ID
KH176-101
Status last verified
Feb 2016
First submitted
19 Apr 2015
Last updated
18 Oct 2021
Data monitoring committee
No

Publications

  • Koene S, Spaans E, Van Bortel L, Van Lancker G, Delafontaine B, Badilini F, Beyrath J, Smeitink J. KH176 under development for rare mitochondrial disease: a first in man randomized controlled clinical trial in healthy male volunteers. Orphanet J Rare Dis. 2017 Oct 16;12(1):163. doi: 10.1186/s13023-017-0715-0.

    PubMed 29037240

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing KH176 in people with MELAS and LHON, run by Khondrion BV at 1 site in Belgium.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 Nov 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF) (time frame: Baseline, 1, 2, 4, 6, 8, 12, 24 hours, 7 day follow up). The study lists 118 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02544217, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2021.

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