Clinical trials: Congenital Anomalies
VHL - Von Hippel-Lindau Syndrome, VHL Gene Mutation
A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)
This study is designed to investigate belzutifan as a treatment for VHL disease associated RCC.
- Conditions
- VHL - Von Hippel-Lindau Syndrome, VHL Gene Mutation, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03401788
- Testing
- Belzutifan
- Where
- 11 sites in United States, Denmark and 2 more
- Volunteers
- 50 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Hypoplastic Left Heart Syndrome, Single Ventricle
Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)
The purpose of this study is to evaluate the efficacy and safety of intracoronary injection of JRM-001 after reconstructive surgery in pediatric patients with functional single ventricle
- Trial name
- APOLLON (NCT02781922)
- Testing
- Autologous cardiac stem cells (JRM-001)
- Where
- 4 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Metcela Inc.
Atopic Dermatitis
Study to Assess the Long-term Safety of Dupilumab Administered in Participants ≥6 Months to <18 Years of Age With Atopic Dermatitis (AD)
The primary objective of the study is to assess the long-term safety of dupilumab in pediatric participants with AD. The secondary objectives of the study are: To assess the long-term efficacy of dupilumab in pediatric…
- Trial name
- NCT02612454
- Testing
- Dupilumab
- Where
- 84 sites in United States, Canada and 5 more
- Volunteers
- 880 people
- Sponsor
- Regeneron Pharmaceuticals
Urea Cycle Disorders, Organic Acidemias
At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism
The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home.
- Conditions
- Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
- Trial name
- NCT06953505
- Testing
- Ammonia Study Device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Sequitur Health Corp.
Primary Immunodeficiency Diseases (PID)
A Study to Evaluate the Long-term Safety of TAK-771 in Japanese Primary Immunodeficiency Disease (PID) Participants
The main aim of the study is to check side effect from the study treatment with TAK-771 in long term. Participants can have taken part in the previous study TAK-771-3004 (NCT05150340).
- Trial name
- NCT05513586
- Testing
- TAK-771
- Where
- 9 sites in Japan
- Volunteers
- 15 people
- Sponsor
- Takeda
Progressive Metastatic Prostate Cancer
An Asian Study to Evaluate Efficacy and Safety of Oral Enzalutamide in Progressive Metastatic Prostate Cancer Participants
Purpose of the study was to assess the effect of enzalutamide on time to Prostate Specific Antigen (PSA) progression as compared to placebo in chemotherapy naïve participants with progressive metastatic prostate cancer who have…
- Trial name
- NCT02294461
- Testing
- Enzalutamide
- Where
- 47 sites in China, Hong Kong and 2 more
- Volunteers
- 395 people
- Sponsor
- Astellas Pharma Inc
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
Fragile X Syndrome
Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
The study objective is to explore the efficacy, safety and tolerability of STX209 for treatment of irritability in subjects with FSX.
- Trial name
- NCT00788073
- Testing
- STX209
- Where
- 12 sites in United States
- Volunteers
- 63 people
- Sponsor
- Seaside Therapeutics, Inc.
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Genetic Disorder, Noonan Syndrome
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as…
- Trial name
- NCT03435627
- Testing
- Somatropin
- Where
- 22 sites in Japan
- Volunteers
- 71 people
- Sponsor
- Novo Nordisk A/S
Healthy
Bioequivalence Study of Colchicine Tablets
This randomized, single dose, three-way crossover study will evaluate the bioequivalence of two formulations of colchicine, the test product (colchicine 0.6mg Mutual) and a marketed combination product (colchicine 0.5 mg with…
- Trial name
- NCT01021020
- Testing
- Colchicine, Colchicine/Probenecid
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Mutual Pharmaceutical Company, Inc.
Cystic Fibrosis
Study of Interferon Gamma-1b by Injection for the Treatment of Patients With Cystic Fibrosis
The purpose of this research study is to evaluate the safety, tolerability, and efficacy of Interferon gamma-1b (IFN-gamma 1b) when administered by subcutaneous injection over a period of 4 weeks to patients with mild-to-moderate…
- Trial name
- NCT00043342
- Testing
- interferon gamma-1b
- Volunteers
- 51 people
- Sponsor
- InterMune
Atopic Dermatitis
POC Study to Evaluate BSI-045B in Moderate-to-severe Atopic Dermatitis
The study is a multicenter clinical trial and is designed as a proof-of-concept study to evaluate the efficacy, safety, tolerability, PK, immunogenicity, and PD of BSI-045B following SC injections.
- Trial name
- ADAMANT (NCT05932654)
- Testing
- BSI-045B
- Where
- 17 sites in United States
- Volunteers
- 22 people
- Sponsor
- Biosion, Inc.
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants…
- Trial name
- AFFINE (NCT04370054)
- Testing
- PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Where
- 45 sites in United States, Australia and 14 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
Cardiomyopathies, Heart Diseases
PKP2-ACM Natural History Study
The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2 Variants (PKP2-ACM) managed with standard of care.
- Conditions
- Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Genetic Diseases
- Trial name
- NCT06644742
- Where
- 3 sites in United States, Netherlands
- Volunteers
- 36 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Neonatal Abstinence Syndrome
Study to Collect Data for Neonatal Abstinence Syndrome (NAS) and Evaluate the Automated Data Collection Process
Substance abuse during pregnancy is on the rise through both prescribed and illicit use of controlled substances, which has increased neonatal abstinence syndrome (NAS).
- Trial name
- NCT06303986
- Testing
- NeoMonki
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Rekovar Inc.
Atopic Dermatitis, Atopic Dermatitis, Unspecified
A Study to Learn About the Study Medicine Etrasimod in Adults With Moderate to Severe Atopic Dermatitis (AD) Who Have Already Tried Treatments Taken by Mouth or by Injection
The purpose of this study is to learn about the safety and effects of the study medicine called etrasimod for the possible treatment of atopic dermatitis (AD), also called eczema, in adults who have already tried AD treatments…
- Conditions
- Atopic Dermatitis, Atopic Dermatitis, Unspecified, Eczema, Eczema, Atopic
- Trial name
- NCT05732454
- Testing
- etrasimod
- Where
- 74 sites in United States, Canada and 2 more
- Volunteers
- 58 people
- Sponsor
- Pfizer
Spinal Deformity
Post Market Surveillance to Evaluate the Effectiveness the Polaris Deformity Spinal System
The purpose of this Post Market Surveillance Validation is to document the performance and clinical outcomes of the Polaris Deformity Spinal System \& Trivium 3D Spinal Deformity Correction System.
- Trial name
- NCT01220505
- Testing
- Surgery
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 66 people
- Sponsor
- Zimmer Biomet
Atopic Dermatitis
Topical YR001 Ointment in Adult With Mild to Moderate Atopic Dermatitis
This study is to evaluate the safety, tolerability, and pharmacokinetics of YR001 topical ointment in adult patients with mild to moderate atopic dermatitis
- Trial name
- NCT06309355
- Testing
- YR001 ointment
- Where
- 4 sites in United States
- Volunteers
- 35 people
- Sponsor
- Hangzhou Yirui Pharmaceutical Technology Co., Ltd
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