Clinical trials: Genetic Conditions
Alpha-1 Antitrypsin Deficiency
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
To evaluate different doses of "Kamada-AAT for Inhalation" on the levels of alpha 1-proteinase inhibitor and other analytes in epithelial lining fluid (ELF) and serum and to assess the safety of the treatment in subjects with AAT…
- Trial name
- NCT02001688
- Testing
- Kamada-AAT for Inhalation, 80mg, Kamada-AAT for Inhalation, 160mg
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Kamada, Ltd.
Fed
Bioequivalence Study of Olanzapine Orally Disintegrating Tablets, 5 mg Under Fed Condition
The purpose of this study is to compare the single oral dose bioequivalence of olanzapine 5 mg OD tablets with Zyprexa Zydis 5 mg tablets under fed conditions and to monitor adverse events and ensure safety of subjects.
- Trial name
- NCT01503450
- Testing
- Olanzapine OD Tablets 5 mg
- Where
- India
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Predisposition, Genetic
The Informed Genetics Annotated Patient Registry
This prospective and retrospective registry will evaluate the clinical effectiveness of Germline Genetic, Genomic, and other Biomarker testing results over time in different clinical populations, in order to shape guidelines for…
- Trial name
- iGAP (NCT04419896)
- Where
- 5 sites in United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Medneon
Gout, Hyperuricemia
Multiple Doses Study of SHR4640 in Male Subjects With High Serum Uric Acid Level
This is a single-Center, randomized, double-Blind, placebo-controlled, multiple ascending-dose Phase I trail.
- Trial name
- NCT03211403
- Testing
- SHR4640
- Where
- Australia
- Volunteers
- 24 people
- Sponsor
- Atridia Pty Ltd.
Liver Diseases, Primary Biliary Cirrhosis
Repeatability and Reproducibility of Multiparametric MRI
This study aims to prospectively assess the repeatability and reproducibility of iron-corrected T1 (cT1), T2*, and hepatic proton density fat fraction (PDFF) quantification with multiparametric MRI using the LiverMultiScan™ (LMS…
- Conditions
- Liver Diseases, Primary Biliary Cirrhosis, Non-Alcoholic Fatty Liver Disease, Non-Alcoholic Steatohepatitis
- Trial name
- NCT03743272
- Testing
- LiverMultiScan
- Where
- 4 sites in Netherlands, United Kingdom
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Perspectum
To Determine Bioequivalence Under Fed Conditions
A Comparative Bioavailability Study of Lisinopril Tablets, 40 mg - Effect of Food Study
The purpose of this study is to compare the bioavailability of Par Lisinopril 40 mg Tablets and IPR Pharmaceuticals, Inc. Zestril (R).
- Trial name
- NCT01380431
- Testing
- Lisinopril
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Skin Laxity, Wrinkle
Clinical Study to Evaluate the Efficacy and Safety of Matrix Pro Treatment
This is a non-randomized, multi-center, open-label, prospective clinical trial evaluating clinical treatments with the Matrix Pro Applicator for the improvement of facial and/or submental and neck laxity.
- Conditions
- Skin Laxity, Wrinkle, Skin Condition
- Trial name
- NCT06726122
- Testing
- Profound Matrix
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Candela Corporation
Fragile X Syndrome
CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction
The purpose of this First-in-Human Phase I study is to investigate the safety, tolerability and pharmacokinetics of CTH120 in adult healthy volunteers.
- Trial name
- FIH-CTH120 (NCT06480968)
- Testing
- CTH120
- Where
- Spain
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- Connecta Therapeutics, S.L.
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Chronic Granulomatous Disease
Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Chronic Granulomatous Disease (CGD).
- Trial name
- NCT05915897
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sanguine Biosciences
Gout, Hyperuricemia
Evaluate the PK, PD, and Safety of Arhalofenate in Combination With Febuxostat for Hyperuricemia in Patients With Gout
The purpose of this study is to evaluate pharmacokinetics, pharmacodynamics, safety and potential for drug-drug interaction of arhalofenate when combined with febuxostat in adult population with gout.
- Trial name
- NCT02252835
- Testing
- Arhalofenate, Febuxostat, Colchicine
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Gilead Sciences
Cadasil
CERebrolysin In CADASIL
The objective of this trial is the global risk-benefit assessment of Cerebrolysin as compared to Placebo in patients with genetically proven CADASIL.
- Trial name
- CERICA (NCT05755997)
- Testing
- Cerebrolysin, 0.9 % NaCl
- Where
- Czechia
- Volunteers
- 30 people
- Sponsor
- Ever Neuro Pharma GmbH
Pruritus, Atopic Dermatitis
Safety, Pharmacokinetics and Preliminary Efficacy of Asimadoline in Pruritus Associated With Atopic Dermatitis
Kappa-opioid receptors mediate the sensation of itch in animals and humans. Asimadoline is an orally active, selective kappa-opioid receptor agonist and has demonstrated efficacy in several preclinical pruritus models.
- Trial name
- NCT02475447
- Testing
- Asimadoline
- Where
- 22 sites in United States
- Volunteers
- 249 people
- Sponsor
- Tioga Pharmaceuticals
Niemann-Pick Disease, Type C
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
- Trial name
- NCT02534844
- Testing
- Parts A/B: Adrabetadex
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 56 people
- Sponsor
- Mandos LLC
Friedreich Ataxia, Cardiomyopathy, Secondary
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
- Trial name
- SUNRISE-FA 2 (NCT07721025)
- Testing
- LX2006, Usual Care
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Lexeo Therapeutics
Sickle Cell Disease, Thalassemia
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell…
- Trial name
- GLADIOLUS (NCT04987489)
- Testing
- Etavopivat tablets
- Where
- 32 sites in United States, Canada and 4 more
- Volunteers
- 53 people
- Sponsor
- Forma Therapeutics, Inc.
Digestive Disease, Gut Health
Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes
The study team is conducting a consumer-driven, decentralized clinical research study to assess the impact of the consumer-grade probiotic supplement, iVS-1, on adults with moderate to severe gastrointestinal discomfort.
- Trial name
- NCT07408518
- Testing
- Probiotic
- Where
- United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Synbiotic Health
Fabry Disease
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function.
- Trial name
- BALANCE (NCT02795676)
- Testing
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Where
- 29 sites in United States, Czechia and 10 more
- Volunteers
- 78 people
- Sponsor
- Protalix
Pruritus, Atopic Dermatitis
Study to Evaluate the Efficacy and Safety of Oral Difelikefalin (CR845) for Moderate to Severe Pruritus in Subjects With Atopic Dermatitis
This is a multicenter, randomized, double-blind, 4-arm, placebo-controlled study to evaluate the efficacy and safety of twice-daily (BID) oral difelikefalin (CR845) in adult subjects with atopic dermatitis (AD) and moderate to…
- Trial name
- KARE (NCT04018027)
- Testing
- difelikefalin 0.25 mg, difelikefalin 0.5 mg, difelikefalin 1.0 mg
- Where
- 40 sites in United States, Canada
- Volunteers
- 401 people
- Sponsor
- Cara Therapeutics, Inc.
Growth Hormone Deficiency, Turner Syndrome
Evaluation of Convenience and Compliance of the Easypod™ Electronic Self-injector
This is an international, multicenter study involving children treated with Saizen®, a growth hormone, who will be trained to use easypod, a new electronic injector and will complete a questionnaire after 12 week of use.
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- NCT00738205
- Volunteers
- 834 people
- Sponsor
- Merck KGaA, Darmstadt, Germany