AzTrials
AzTrials

Healthy VolunteersPhase 1NCT03380780

A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

A Phase 1 study testing Emicizumab in people with Healthy Volunteers and Hemophilia A, run by Hoffmann-La Roche at 1 site in China.

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

This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects. Read more about this study

Who is it for?
Ages 20 to 45 years, men onlyHealthy volunteers can take part
Conditions the study is for
Healthy Volunteers, Hemophilia A
What is being tested?
Emicizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteChina
Study dates
12 Mar 2018to 18 Sep 2018
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 20 to 45 years
  • Men only
  • Healthy volunteers accepted
  • Adult

Who may be able to join

  • Healthy Chinese male subjects, aged 20-45 years inclusive at the time of screening
  • Chinese subjects must have Chinese parents and grandparents, all of whom were born in China
  • A body mass index (BMI) between 19 and 24 kilograms per height in meters squared (kg/m^2), inclusive
  • Able to participate and willing to give written informed consent and to comply with the study requirements

Who cannot join

  • Any history or presence of a clinically significant disorder, or any other condition or disease which in the judgement of the investigator would place the subject at undue risk; interfere with absorption, distribution, metabolism, and excretion of emicizumab; or interfere with the ability of the subject to complete the study
  • Major illness within 1 month prior to dosing, and/or any condition which could relapse during or immediately after the study
  • Use of any prescribed or over-the-counter (OTC) medication or herbal medicine taken within 14 days prior to dosing or within 5 times the elimination half-life of the medication prior to dosing (whichever is longer), with some exceptions
  • Any significant donation/loss of blood or plasma (greater than 450 milliliters) within the 3 months prior to dosing
  • Regular smoker with consumption of more than 10 cigarettes per day or the equivalent amount of tobacco
  • Participation within a clinical study with an investigational drug or device within the last 3 months prior to dosing
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  • Any clinically relevant history of hypersensitivity or allergic reactions, either spontaneous or following drug administration or exposure to foods or environmental agents
  • Previous or concomitant thromboembolic disease such as deep vein thrombosis (DVT) or signs of thromboembolic disease, or family history of thromboembolic disorder such as serious DVT
  • At high risk for thrombotic microangiopathy (e.g., have a previous medical or family history of thrombotic microangiopathy), in the investigator's judgment
  • Previous or concomitant autoimmune or connective tissue disease
  • History of tuberculosis or active tuberculosis with positive test result at screening
  • Any other reason that, in the judgment of the investigator, would render the subject unsuitable for study participation

Trial locations and contacts

1 site in 1 country.

China1 site
  • Peking University Third Hospital

    Beijing, 100083

Other trials in China

About this study

Official title: A Single-Center, Open-Label, Single Dose Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects.

Being tested
Emicizumab
Medical subject headings
Hemophilia A

What participants receive

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

  • EmicizumabDrug

    Also called RO5534262

    Participants will receive a single 1 milligram per kilogram of body weight (mg/kg) subcutaneous dose of emicizumab on Day 1.

Study groups in NCT03380780
Study groupRoleWhat they receive
EmicizumabExperimentalNot described
Receives: Drug: Emicizumab

What is being measured

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

  1. Maximum Observed Plasma Concentration (Cmax) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  2. Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

Secondary outcomes (62)
  1. AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  2. Time to Cmax (Tmax) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  3. Apparent Terminal Half-Life (t1/2) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  4. Apparent Clearance (CL/F) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  5. Apparent Volume of Distribution (Vz/F) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  6. Mean Residence Time (MRT) of Emicizumab

    Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

    Measured: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  7. Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade

    The WHO Toxicity Grading Scale was used for assessing adverse event severity. Any adverse event not specifically listed in the WHO Toxicity Grading Scale was assessed according to the following levels of severity: Grade 1 is mild; Grade 2 is moderate; Grade 3 is severe; and Grade 4 is life-threatening. Investigator text for adverse events were encoded using the Medical Dictionary for Regulatory Activities (MedDRA) version 21.1. After informed consent had been obtained but prior to initiation of study drug, only serious adverse events (SAEs) caused by a protocol-mandated intervention were to have been reported. After initiation of study drug, all adverse events, regardless of relationship to study drug, were to have been reported until the participant completed his last study visit. After this period, any SAEs believed to be related to prior study drug treatment were to be reported.

    Measured: From screening to study completion (20 weeks)

  8. Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study

    Participants were considered to be 'ADA Negative (Treatment Unaffected)' if baseline and all post-baseline samples were negative, or if they were ADA positive at baseline but did not have any post-baseline samples with a titer that was at least 4-fold greater than the titer of the baseline sample. 'Total ADA Positive' is the sum of all participants who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Induced)', those who were ADA negative at baseline and tested positive for ADA following study drug administration; and 'ADA Positive (Treatment Boosted)', those who were pre-dose ADA positive and had post-baseline samples with a titer that was at least 4-fold greater compared to the baseline measurement.

    Measured: Predose at Baseline (Day 1) and postdose on Days 57 and 113

  9. Number of Participants With Laboratory Test Abnormalities

    The number of participants with a laboratory abnormality during treatment (numerator) is reported among the 'number analyzed' in the table below (denominator), which represents the number of participants without that abnormality at baseline (last observation prior to initiation of study drug). Note that samples from all participants were analyzed for each laboratory parameter. Values falling above or below the Roche predefined standard reference range were laboratory abnormalities labelled accordingly as 'high' or 'low'. Not every laboratory abnormality qualified as an adverse event; only if it was accompanied by clinical symptoms, resulted in a change in study treatment or in a medical intervention, or was clinically significant in the investigator's judgment. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase

    Measured: Baseline and Days 2, 4, 8, 11, 15, 29, 43, 57, 71, 85, and 113

  10. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  11. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  12. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  13. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  14. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  15. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  16. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  17. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  18. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  19. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  20. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  21. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  22. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  23. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  24. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  25. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  26. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  27. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  28. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  29. Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  30. Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time

    Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 11, 29, 57, and 113

  31. Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration

    Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 11, 29, 57 and 113

  32. Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time

    Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 11, 29, 57 and 113

  33. Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR)

    The INR is a standardized measure of the prothrombin time. Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 11, 29, 57 and 113

  34. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  35. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  36. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin

    Erythrocyte mean corpuscular hemoglobin (MCH) is a measure of the average amount 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  37. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume

    Erythrocyte mean corpuscular volume is a measure of the average volume of a 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  38. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  39. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1)

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  40. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  41. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  42. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  43. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  44. Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

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

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

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

    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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113

  47. Number of Participants by Test Results for Blood in Urine by Timepoint

    Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for blood in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 8, 29, 57, 85, and 113

  48. Number of Participants by Test Results for Glucose in Urine by Timepoint

    Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for glucose in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 8, 29, 57, 85, and 113

  49. Number of Participants by Test Results for Protein in Urine by Timepoint

    Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for protein in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.

    Measured: Baseline and Days 2, 8, 29, 57, 85, and 113

  50. Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure

    Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  51. Change From Baseline in Vital Signs by Timepoint: Pulse Rate

    Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  52. Change From Baseline in Vital Signs by Timepoint: Respiratory Rate

    Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  53. Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure

    Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  54. Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary)

    Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

  55. Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  56. Change From Baseline in ECG Results by Timepoint: PR Duration

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  57. Change From Baseline in ECG Results by Timepoint: QRS Duration

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  58. Change From Baseline in ECG Results by Timepoint: QT Duration

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  59. Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula)

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  60. Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula)

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  61. Change From Baseline in ECG Results by Timepoint: RR Duration

    The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. 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. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.

    Measured: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113

  62. Number of Participants With Concomitant Medications

    The original terms recorded by the investigator for concomitant medications were standardized by the sponsor by assigning preferred terms. The duration of treatment with the concomitant medications ranged from 1 day to 5 days. Except for 1 participant who was treated during the in-clinic period (Days -1 to 4), all other concomitant medications were recorded during the ambulatory period (Days 6 to 113).

    Measured: From screening to study completion (20 weeks)

Timeline

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

  1. First posted21 Dec 2017
  2. Study start12 Mar 2018
  3. Primary completion18 Sep 2018
  4. Study completion18 Sep 2018

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03380780
Sponsor's protocol ID
YP39308
Status last verified
Sep 2019
First submitted
13 Dec 2017
Last updated
30 Sep 2019
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Emicizumab in people with Healthy Volunteers and Hemophilia A, run by Hoffmann-La Roche at 1 site in China.

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 4 inclusion and 12 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 30 Sep 2019. 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 China. 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 30 Sep 2019). 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?

Maximum Observed Plasma Concentration (Cmax) of Emicizumab (time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 30 Sep 2019.

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