Clinical trials: Genetics Syndrome
Retinitis Pigmentosa
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients.
- Trial name
- MOON (NCT06292650)
- Testing
- ZM-02-L, ZM-02-H, ZM-02-S
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Zhongmou Therapeutics
Stargardt Disease 1, STGD1
A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following…
- Conditions
- Stargardt Disease 1, STGD1, Healthy Volunteers
- Trial name
- NCT07836452
- Testing
- CITY-RBP4
- Where
- Australia
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- City Therapeutics
Atopic Dermatitis
Safety, PK, PD, and Clinical Activity of Orally Administered KT-621 in Adult Patients With Atopic Dermatitis (AD)
This is a study to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of orally administered KT-621 in adult male and female patients with moderate to severe atopic dermatitis (AD).
- Trial name
- BroADen (NCT06945458)
- Testing
- KT-621
- Where
- 12 sites in United States
- Volunteers
- 22 people
- Sponsor
- Kymera Therapeutics, Inc.
Autoimmune Diseases, Rheumatoid Arthritis (RA)
Study of S-4321 in Participants With an Autoimmune or Immune-mediated Disease
This is a multi-center, open-label Ph 1b basket study to assess safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, biomarker response, and preliminary efficacy of multiple doses of S-4321 in…
- Conditions
- Autoimmune Diseases, Rheumatoid Arthritis (RA), Psoriatic Arthritis (PsA), Psoriasis (PsO)
- Trial name
- NCT07455578
- Testing
- S-4321
- Where
- 2 sites in Australia
- Volunteers
- 24 people
- Sponsor
- Seismic Therapeutic AU Pty Ltd
Severe Hemophilia A
A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
The primary objectives of the study are to evaluate the Pharmacokinetics，Safety and tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW117) in patients with severe hemophilia A.
- Trial name
- NCT04864743
- Testing
- ADVATE, FRSW117
- Where
- 4 sites in China
- Volunteers
- 13 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Osteogenesis Imperfecta
A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers
To understand if AGA2115 is safe and well tolerated in healthy adult volunteers.
- Trial name
- NCT06086613
- Testing
- AGA2115
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Angitia Incorporated Limited
Hemophilia A, Prophylaxis of Bleeding
An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.
This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied.
- Trial name
- JOIHA (NCT05643560)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- Italy
- Volunteers
- 33 people
- Sponsor
- Bayer
Thyroid Eye Disease
Efficacy and Safety of Lonigutamab in Subjects With Thyroid Eye Disease (TED)
Phase 1/2, multicenter, multiple dose clinical study designed to evaluate lonigutamab in subjects with TED.
- Trial name
- TED (NCT05683496)
- Testing
- lonigutamab
- Where
- 11 sites in United States, Australia
- Volunteers
- 31 people
- Sponsor
- ACELYRIN Inc.
Chronic Gout Refractory to Conventional Therapy
Re-exposure Study of Pegloticase Intravenous (i.v.) in Symptomatic Gout Patients
The purpose of the study is to evaluate the safety and clinical effect of re-exposure to a 24 week course of treatment of pegloticase i.v. in subjects whose last exposure to pegloticase i.v.
- Trial name
- NCT00675103
- Testing
- pegloticase 8 mg i.v.
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Savient Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Dermatitis Atopic
A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis
This is a parallel group, Phase 3, multinational, multicenter, randomized, double blind, placebo-controlled, 3-arm monotherapy study for treatment of participants diagnosed with moderate to severe atopic dermatitis (AD), whose…
- Trial name
- COAST 1 (NCT06130566)
- Testing
- Amlitelimab
- Where
- 148 sites in United States, Argentina and 13 more
- Volunteers
- 601 people
- Sponsor
- Sanofi
Acute Gouty Arthritis
Canakinumab in the Treatment of Gouty Arthritis Flare(s) and Prevention of New Flares in Patients With Chronic Kidney Disease
The purpose of this study is to confirm the efficacy and safety results obtained in a chronic kidney disease (CKD) subgroup of patients participating in Phase III studies in a larger, independent patient population with difficult…
- Trial name
- NCT01593527
- Testing
- ACZ885, Triamcinelone acetonide
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…
- Trial name
- ENVISION (NCT00909727)
- Testing
- Ivacaftor
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 52 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC…
- Trial name
- NCT04214002
- Where
- United States
- Sponsor
- Krystal Biotech, Inc.
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
A Phase 2b Study of Zagociguat in Patients With MELAS
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs.
- Trial name
- PRIZM (NCT06402123)
- Testing
- zagociguat 15mg, zagociguat 30mg
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 43 people
- Sponsor
- Tisento Therapeutics
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Skin Laxity
Retrospective Evaluation of Combination Treatment With the Ulthera System
A retrospective study to evaluate the Ulthera® System for its potential interaction with toxins and fillers. Up to 500 subjects will be enrolled.
- Trial name
- NCT02444169
- Testing
- Ultherapy, Incobotulinumtoxin A, Radiesse
- Where
- 5 sites in United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
Gout, Arthritis, Gouty
Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-302)
This study will assess the serum uric acid lowering effect and safety of AR882 in gout patients at two doses compared to placebo over 12 months
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Trial name
- NCT06439602
- Testing
- AR882 50 mg, AR882 75 mg
- Where
- 68 sites in United States, Australia and 3 more
- Volunteers
- 818 people
- Sponsor
- Arthrosi Therapeutics
Phenylketonuria (PKU)
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570).
- Trial name
- NCT03694353
- Testing
- Pegvaliase
- Where
- 17 sites in United States
- Volunteers
- 37 people
- Sponsor
- BioMarin Pharmaceutical
Lax Skin
Treatment for Lifting Upper Arm Lax Skin
Open-label, non-randomized, prospective, multi-center, self-controlled clinical study with masked evaluation.
- Trial name
- NCT05358327
- Testing
- Sofwave
- Where
- 4 sites in United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD