Clinical trials: Genetic Diseases, Inborn
Gout
Lesinurad/Allopurinol 200/300 Fixed-Dose Combination (FDC) Tablets Bioequivalence.
To assess the bioequivalence between lesinurad/allopurinol 200/300 FDC tablets and coadministered lesinurad and allopurinol tablets in the fasted state based on the pharmacokinetic (PK) evaluation of lesinurad and allopurinol in…
- Trial name
- NCT03272425
- Testing
- lesinurad/allopurinol 200/300 FDC tablets, lesinurad 200 mg, allopurinol 300 mg
- Where
- Brazil
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Ardea Biosciences, Inc.
Idiopathic Cardiomyopathy, Left Ventricular (LV) Systolic Dysfunction
Performance of the Cardiac Microcurrent (C-MIC) System With a Less Invasively Placed Left Ventricular Lead
Patients with idiopathic dilated cardiomyopathy in heart failure (NYHA class III - IV) with a baseline left ventricular ejection fraction between ≥25% and ≤35%, and patients with non-ischemic cardiomyopathy in heart failure (NYHA…
- Conditions
- Idiopathic Cardiomyopathy, Left Ventricular (LV) Systolic Dysfunction, Non-ischemic Cardiomyopathy
- Trial name
- NCT06920030
- Testing
- Subcutaneous cardiac microcurrent treatment
- Where
- 4 sites in Bosnia and Herzegovina, Serbia
- Volunteers
- 22 people
- Sponsor
- Berlin Heals GmbH
Rett Syndrome, RTT
A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
This study will be conducted to evaluate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in participants with Rett syndrome.
- Trial name
- NCT04252586
- Testing
- GWP42003-P
- Where
- 31 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- Jazz Pharmaceuticals
Tardive Dyskinesia, Huntington Disease
Safety, Tolerability and Pharmacokinetic of Multiple-ascending Doses of LPM3770164 in Healthy Subjects
This is a single-center, randomized, double-blind, placebo-controlled, multiple-ascending doses trial to evaluate the safety, tolerability and pharmacokinetic of LPM3770164 sustained-release tablets orally administered in healthy…
- Trial name
- NCT06216054
- Testing
- LPM3770164 sustained release tablet, LPM3770164 sustained release tablet simulant
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Luye Pharma Group Ltd.
Cryopyrin Associated Periodic Syndrome
A Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ZYIL1 in Subjects With Cryopyrin Associated Periodic Syndromes (CAPS)
ZYIL1 is expected to show benefit in patients with CAPS. The present study aims to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics of ZYIL1 when administered to subjects with CAPS.
- Trial name
- NCT05186051
- Testing
- ZYIL1 capsule
- Where
- Australia
- Volunteers
- 3 people
- Sponsor
- Zydus Lifesciences Limited
X-linked Hypophosphatemia
Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).
- Trial name
- NCT02537431
- Testing
- burosumab
- Where
- 14 sites in United States, Canada and 4 more
- Volunteers
- 14 people
- Sponsor
- Kyowa Kirin, Inc.
Anemia, Sickle Cell
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor…
- Trial name
- NCT04091737
- Testing
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Atopic Dermatitis
Phase 2a Study to Assess the Efficacy,Safety and Tolerability of GIA632 in Adult Participants With Moderate to Severe Atopic Dermatitis
The main purpose of this Ph2a study is to evaluate the preliminary efficacy, safety and tolerability of GIA632 when administered to adult participants with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT07220577
- Testing
- GIA632
- Where
- 28 sites in United States, Bulgaria and 7 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Digestive System Diseases, Pancreatic Disease
Open Label Safety Study of DCI-1020 in Pediatric Cystic Fibrosis (CF) Patients
Hypothesis:DCI 1020 capsules are safe and effective in treating exocrine pancreatic insufficiency in CF patients <= 2 years of age.
- Conditions
- Digestive System Diseases, Pancreatic Disease, Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Trial name
- NCT00663975
- Testing
- DCI 1020
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Digestive Care, Inc.
Mucopolysaccharidosis I, Hurler's Syndrome
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00144781
- Testing
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Where
- 5 sites in Brazil, Canada
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Prader-Willi Syndrome
A Study of Pitolisant in Patients With Prader-Willi Syndrome
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
- Trial name
- NCT06366464
- Testing
- Pitolisant tablet
- Where
- 57 sites in United States, Australia and 11 more
- Volunteers
- 134 people
- Sponsor
- Harmony Biosciences Management, Inc.
Cardiovascular Diseases, Congestive Heart Failure
Treating Congestive HF With hiPSC-CMs Through Endocardial Injection
Heart failure is the primary cause of morbidity and mortality worldwide. Currently drug treatments for heart failure manage the symptoms, but not restore the loss cardiomyocytes due to the very limited regenerative capability in…
- Conditions
- Cardiovascular Diseases, Congestive Heart Failure, Dilated Cardiomyopathy
- Trial name
- NCT04982081
- Testing
- hiPSC-CM therapy
- Where
- China
- Volunteers
- 20 people
- Sponsor
- HELP Therapeutics Co., Ltd.
Beta-Thalassemia
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT).
- Trial name
- NCT05577312
- Testing
- BRL-101
- Where
- 7 sites in China
- Volunteers
- 39 people
- Sponsor
- Bioray Laboratories
Hemophilia A or B
A Study to Describe Patient REal-world FitusiRan ExpeRience
This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who…
- Trial name
- PREFER (NCT07895381)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Sanofi
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Atopic Dermatitis
An Open-Labeled Exploratory Study to Evaluate Safety and Efficacy of FB825 in Adults With Atopic Dermatitis
The purpose of this study is to evaluate the Safety and Efficacy of FB825 in Adults with Atopic Dermatitis
- Trial name
- NCT03758716
- Testing
- FB825, FB825-15D11, Anti-CemX
- Where
- Taiwan
- Volunteers
- 12 people
- Sponsor
- Fountain Biopharma Inc.
Fabry Disease
Migalastat Food Effect Study
A 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects.
- Trial name
- NCT01489995
- Testing
- A (migalastat), B (migalastat), C (migalastat)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Lactose Intolerance
β-galactosidase Producing Probiotic Strains to Improve Lactose Digestion
The purpose of this study is to assess that two β-galactosidase Producing Probiotic Strains help improve lactose digestion in subjects with lactose maldigestion.
- Trial name
- NCT02518295
- Testing
- Positive control, Probiotic S. thermophilus, Probiotic B. longum
- Where
- Switzerland
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
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