Clinical trials: Genetic Diseases
Breast Cancer Metastatic, Renal Cell Carcinoma (RCC)
Sapu003 in Advanced mTOR-sensitive Solid Tumors
This is a phase 1b, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics of Sapu003 in combination with Exemestane in in patients with advanced mTOR-sensitive solid tumors (HR+/HER2-negative…
- Conditions
- Breast Cancer Metastatic, Renal Cell Carcinoma (RCC), Neuroendocrine Tumors, Tuberous Sclerosis Complex (TSC)
- Trial name
- NCT07369505
- Testing
- Sapu003, Exemestane 25 MG
- Where
- Australia
- Volunteers
- 27 people
- Sponsor
- SAPU NANO (US) LLC
Cystic Fibrosis
Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF
The primary objective of this study is to evaluate the safety and efficacy of a CAT regimen with aztreonam for inhalation solution (AZLI) and tobramycin inhalation solution (TIS) in adult and pediatric subjects with cystic…
- Trial name
- AZLI CAT (NCT01641822)
- Testing
- AZLI, Tobramycin inhalation solution
- Where
- 71 sites in United States
- Volunteers
- 107 people
- Sponsor
- Gilead Sciences
Duchenne Muscular Dystrophy
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study.
- Trial name
- EXPEDITION (NCT05967351)
- Testing
- delandistrogene moxeparvovec
- Where
- 38 sites in United States, Belgium and 7 more
- Volunteers
- 400 people
- Sponsor
- Sarepta Therapeutics, Inc.
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
Primary Hypercholesterolemia or Mixed Hyperlipidemia
QLC7401 Monotherapy in Non-familial Hypercholesterolemia or Mixed Dyslipidemia
This Phase III clinical trial aims to assess the efficacy and safety of QLC7401 monotherapy in adults with non-familial primary hypercholesterolemia or mixed dyslipidemia who are not receiving lipid-lowering therapy.
- Trial name
- NCT07444086
- Testing
- QLC7401
- Volunteers
- 510 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Atopic Dermatitis
A Phase I Clinical Trial to Evaluate CU-10101 Unguent
The study was conducted in two parts:Part 1 and Part 2.Part 1 is a single-site,randomized,double-blind,vehicle-controlled single and multiple administration,dose-escalation study in healthy Chinese participants.There are four…
- Trial name
- NCT06744569
- Testing
- CU-10101
- Where
- China
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Cutia Therapeutics（Wuxi）Co.,Ltd
Sickle Cell Disease
Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287086
- Testing
- BRL-101 autologous hematopoietic stem and progenitor cells injection
- Volunteers
- 1 people
- Sponsor
- Bioray Laboratories
Hemophilia A
Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis
To assess and describe real-life treatment choices for rFⅧ contained regular prophylaxis/bleeding prevention treatment in pediatric hemophilia patients in China (2007-2013)
- Trial name
- ReCARE (NCT02263066)
- Testing
- BAY14-2222_Kogenate-FS FVIII
- Where
- China
- Volunteers
- 181 people
- Sponsor
- Bayer
Parkinson's Disease
Parkinson's Disease G2019S LRRK2 Genetic Testing Program
Increase awareness of the G2019S LRRK2 mutation in Parkinson's and no cost genetic testing program.
- Trial name
- NCT04919356
- Testing
- G2019S LRRK2
- Where
- United States
- Volunteers
- 836 people
- Sponsor
- Escape Bio, Inc.
Atopic Dermatitis
A Study to Determine the Safety and Tolerability of Dupilumab (REGN668/SAR231893) in Patients Aged ≥6 to <18 Years With Atopic Dermatitis (Eczema)
The primary objective of the study is to characterize the safety and pharmacokinetics (PK) of dupilumab in pediatric patients with moderate-to-severe atopic dermatitis (AD) (for adolescents ≥12 to <18 years of age) or severe AD…
- Trial name
- NCT02407756
- Testing
- Dupilumab
- Where
- 26 sites in Canada, Czechia and 4 more
- Volunteers
- 78 people
- Sponsor
- Regeneron Pharmaceuticals
Primary Immunodeficiency Disease
Efficacy, Safety, and Pharmacokinetics of Shu Yang IVIG
To evaluate the safety, efficacy, and pharmacokinetic properties of Shu Yang intravenous immune globulin in patients with primary immune deficiency aged less than 60 years.
- Trial name
- Imunoforte (NCT06089122)
- Testing
- IVIG
- Volunteers
- 50 people
- Sponsor
- Azidus Brasil
Primary Hyperoxaluria
Phase 2/3 Oxabact Study
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- NCT01037231
- Testing
- Oxalobacter formigenes
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 36 people
- Sponsor
- OxThera
Down Syndrome
A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.
The purpose of this double-blind, placebo-controlled, comparative study and open-label extension study is to confirm the efficacy and safety of E2020 in subjects with Down syndrome having regression symptoms and disabled…
- Trial name
- NCT02094053
- Testing
- E2020-Donepezil hydrochloride
- Where
- 10 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Eisai Co., Ltd.
Beta-Thalassemia
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe…
- Trial name
- NCT04938635
- Testing
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID
- Where
- 7 sites in United States, Bulgaria and 1 more
- Sponsor
- Vifor (International) Inc.
Cystic Fibrosis
A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis
The purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis.
- Trial name
- NCT01543191
- Testing
- PUR118
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Pulmatrix Inc.
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of Ruxolitinib Cream in Children and Adolescents (6 to <18 Years Old) With Moderate Atopic Dermatitis
The purpose of the study is to assess the efficacy and safety of ruxolitinib cream in children and adolescents (6 to <18 Years Old) with moderate atopic dermatitis.
- Trial name
- TRuE-AD5 (NCT06832618)
- Testing
- Ruxolitinib, Vehicle Cream
- Where
- 93 sites in United States, Belgium and 8 more
- Volunteers
- 159 people
- Sponsor
- Incyte Corporation
Advanced Solid Tumors Cancer, MSI-H Cancer
A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors.
The goal of this clinical trial is to learn if NDI-219216 is safe for patients, and if NDI-219216 might be a possible treatment for advanced solid tumors in the later phases of the study.
- Trial name
- NCT06898450
- Testing
- NDI-219216
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 134 people
- Sponsor
- Nimbus Wadjet, Inc.
Haemophilia
Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia
This study will help to better understand the course of disease in people with haemophilia (A or B [with or without inhibitors]) and the haemophilia care scenario in the selected countries and the experience so far in dealing…
- Trial name
- NCT07163260
- Testing
- No treatment given
- Where
- 4 sites in India, Malaysia and 2 more
- Volunteers
- 244 people
- Sponsor
- Novo Nordisk A/S
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Stargardt Disease-1
Study of STG-001 in Subjects With Stargardt Disease
This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT04489511
- Testing
- STG-001
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Stargazer Pharmaceuticals, Inc.
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