Clinical trial discovery
Find Clinical Trials
Search studies by condition, treatment, location, eligibility, or trial identifier.
Showing 1–15 of 15 trials
Active filters
Clinical trials found
Published and searchable clinical trials
Searching for clinical trials...
NCT07054515
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
- Locations
-
- UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
- Mayo Clinic Childrens Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
NCT00029965
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Atrophy, +29 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States
NCT00668187
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- University of Minnesota - Pediatric Genetics and Metabolism Minneapolis, Minnesota, United States
NCT06539169
- Conditions
- Alpha-Thalassemia, Alzheimer Disease, Amyloidosis, +104 more
- Locations
-
- xCures Los Altos, California, United States
Enrolling by Invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05368038
- Conditions
- Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
- Locations
-
- Maimonides Medical Center Brooklyn, New York, United States
- NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
- North Shore University Hospital Manhasset, New York, United States
- NYU Langone Health - Tisch Hospital New York, United States
Not Yet Recruiting
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
NCT07479953
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
Not specified
NCT04041102
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Montreal Children's Hospital Research Institute - McGill University Montreal, Quebec, Canada
NCT05109793
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland, California, United States
- Mayo Clinic Rochester Rochester, Minnesota, United States
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
NCT04310163
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Casimir Trials Plymouth, Massachusetts, United States
NCT04470713
- Conditions
- AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
- Locations
-
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Mayo Clinic - Rochester Rochester, Minnesota, United States
- Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
- UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
NCT00383448
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT00176904
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT01626092
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +50 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Active, Not Recruiting
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
NCT07082543
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
- Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
Active, Not Recruiting
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
NCT04713475
- Conditions
- Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
- Locations
-
- Benioff Children's Hospital Oakland, California, United States
- University of Minnesota Minneapolis, Minnesota, United States
- Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States