Clinical trials: Genetic Disorder, open to volunteers
Huntington Disease
Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in Huntington's Disease
The purpose of this research study is to determine the optimal pulse repetition frequency of low-intensity focused ultrasound that is safe and effective in improving motor symptoms in patients with Huntington's disease.
- Trial name
- NCT07513844
- Testing
- Low-Intensity Focused Ultrasound Stimulation（LIFU）
- Where
- China
- Volunteers
- 24 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Sickle Cell Disease, Cerebral Stroke
Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography
The purpose of the study protocol is to identify imaging biomarkers for brain tissue under high metabolic stress at risk for permanent injury.
- Trial name
- HDDOT CVR (NCT07776444)
- Testing
- Carbon Dioxide
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
School Readiness Intervention for Preschool Children With Sickle Cell Disease
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease.
- Trial name
- NCT06367192
- Testing
- The adapted Kids in Transition to School Intervention, Standard school resources
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)
'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949.
- Trial name
- NCT02824471
- Testing
- SCD Group
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Diabetes Mellitus, Type 2
Project Dulce for Arab Americans With Type 2 Diabetes
Arab Americans (AA) face many challenges in diabetes self-management due to the limited educational resources and support available for them.
- Trial name
- NCT05580536
- Testing
- Project Dulce + Dulce Digital
- Where
- 2 sites in United States
- Volunteers
- 48 people
- Sponsor
- Scripps Whittier Diabetes Institute
Duchenne Muscular Dystrophy (DMD)
DMD and Gamified Physiotherapy
Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular disease that limits children's physical function, mobility, and participation in daily life.
- Trial name
- Gamified DMD (NCT07368400)
- Testing
- Gamified Mobile Physiotherapy Exercise Application, Standard Home Exercise Instructions
- Volunteers
- 46 people
- Sponsor
- Başak Çağla Arslan
Child Development
LIFE Child (LIFE Leipzig Research Centre for Civilization Diseases)
The Leipzig Research Centre for Civilization Diseases (LIFE) Child Study is a prospective longitudinal population-based study with a life course approach to health and disease.
- Trial name
- NCT02550236
- Where
- Germany
- Volunteers
- 7,000 people, healthy volunteers welcome
- Sponsor
- University of Leipzig
Sickle Cell Disease, Venous Thromboembolism
Comparison of Thrombin Generation, Platelet Activation and Aggregation Profiles in Patients With Sickle Cell Disease Depending on Their SS or SC Genotype (DrepAgreg)
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, painful crises called vaso-occlusive crises (VOC) and chronic inflammation.
- Trial name
- DrepAgreg (NCT07852442)
- Where
- 2 sites in France, Guadeloupe
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Eczema Atopic Dermatitis, Eczema
A Double-Blind, Randomized, Vehicle-Controlled Phase 2 Study to Assess the Efficacy and Safety of GX-03 in Adult Subjects With Moderate to Severe Atopic Dermatitis (Eczema)
This is a Phase 2, two-stage, adaptive, 1:1 randomized, double-blind, vehicle-controlled clinical study evaluating the efficacy, safety, and tolerability of GX-03 topical ointment in adult subjects with atopic dermatitis…
- Conditions
- Eczema Atopic Dermatitis, Eczema, Atopic Dermatitis
- Trial name
- GX-03 in AD (NCT07355075)
- Testing
- GX-03, Vehicle
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Turn Therapeutics
Wilson Disease, D-Penicillamine
Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity
Wilson's disease is a genetic disorder, resulting from an anomaly present on the ATP7B gene located on chromosome 13, causing a progressive accumulation of copper in various organs such as the liver, nervous system and cornea…
- Conditions
- Wilson Disease, D-Penicillamine, Effect of D-penicilline on Cutaneous Elastity of Wilson's Patient
- Trial name
- WILDERME (NCT06945081)
- Testing
- Preparation of forearm molds with SILFLO® silicone
- Where
- 2 sites in France
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Emphysema, Chronic Obstructive Pulmonary Disease
Emphysema Research Registry and Biosample Repository
The Comprehensive Lung Center (CLC) at the University of Pittsburgh Medical Center (UPMC Health System) provides patients with any type of breathing or lung disorder a full range of diagnostic and therapeutic services.
- Conditions
- Emphysema, Chronic Obstructive Pulmonary Disease, Chronic Bronchitis
- Trial name
- NCT00307281
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Cystic Fibrosis (CF)
Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis
The goal of this clinical trial is to support the development of personalized therapy tailored to each patient's individual characteristics.
- Trial name
- NCT07809867
- Testing
- Elexacaftor, Tezacaftor, Ivacaftor, Ivacaftor (VX-770)
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Istituto Giannina Gaslini
Stargardt, Stargardt's Disease
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75…
- Conditions
- Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
- Trial name
- POLARIS (NCT06435000)
- Where
- 20 sites in United States, Germany and 1 more
- Volunteers
- 75 people
- Sponsor
- Splice Bio
Ovarian Cancer
Identification of Genetic, Phenotypic, Clinical, Biological and Histological Factors Differentiating Patients With Ovarian Cancer Who Are Refractory to Platinum-based Therapy From Patients Defined as Long-term Responders to Platinum-based Therapy
After having constituted the ORTRAI cohort using the CONSORE database at the Jean PERRIN Center, the objective is to characterize the patients both clinically and biologically, histologically and genomically.
- Trial name
- ORTRAI (NCT07361471)
- Testing
- molecular analysis
- Where
- France
- Volunteers
- 55 people
- Sponsor
- Centre Jean Perrin
Parkinson Disease
French Parkinson's Disease Cohort - NS-PARK
The aim of NS-PARK cohort are to describe the natural history of Parkinson's disease (PD), and to propose patients stratification models based on PD pathophysiological mechanisms.
- Trial name
- NS-PARK (NCT04888364)
- Where
- France
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Dermatitis, Atopic, Eczema
Comparative Study of Crisaborole vs. Tacrolimus in Atopic Dermatitis
This study will compare the efficacy and safety of crisaborole ointment with tacrolimus ointment, in the treatment of patients with mild-to-moderate atopic dermatitis.
- Trial name
- NCT07367932
- Testing
- Application of crisaborole twice daily, Tacrolimus ointment in atopic dermatitis
- Volunteers
- 30 people
- Sponsor
- Cairo University
Huntington Disease
Longitudinal Endpoint Assessment of Disease Burden in HD
LEAD-HD is intended to collect and analyze self-reported health information from individuals with Huntington Disease (HD) or prodromal HD participating in a 24-month longitudinal natural history study using remote technologies.
- Trial name
- LEAD-HD (NCT06475898)
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- Huntington Study Group
Prostate Cancer, Genetic Predisposition
Prostate Tissue BioBank
Prostate cancer is also the most common cancer in men with inherited pathogenic variants in BRCA1 and BRCA2. Beyond BRCA1/2, other genes are known to increase the risk of prostate cancer, including ATM, TP53 and HOXB13.
- Trial name
- PTBB (NCT06659614)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Abramson Cancer Center at Penn Medicine
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
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