NCT05619900
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +36 more
- Locations
-
- University of California San Francisco San Francisco, California, United States
Clinical trial condition
Mucopolysaccharidosis I
Explore clinical trials studying Mucopolysaccharidosis I. Study availability and eligibility vary by location and protocol.
25 public trials
2 recruiting studies
Mucopolysaccharidosis I trial results
Recruiting
MPS (RaDiCo Cohort) (RaDiCo-MPS)
NCT06036693
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +33 more
- Locations
-
- Centre Hospitalier Universitaire d'Angers Angers, France
- Hôpital des Enfants - Groupe Hospitalier Pellegrin Bordeaux, France
- Hôpital Morvan Brest, France
- Hôpital d'Estaing Clermont-Ferrand, France
Enrolling by Invitation
Long Term Follow-Up for RGX-111
NCT06103487
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- Children's Hospital of Orange County Orange, California, United States
- Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
NCT05134571
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- Investigational Site Number :1560003 Beijing, China
- Investigational Site Number :1560002 Beijing, China
- Investigational Site Number :1560004 Guangzhou, China
- Investigational Site Number :1560006 Hangzhou, China
Completed
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
NCT04453085
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Instituto de Genética e Erros Inatos do Metabolismo - IGEIM São Paulo, Brazil
- Fukuoka Children's Hospital Fukuoka, Japan
Completed
Baby Detect : Genomic Newborn Screening
NCT05687474
- Conditions
- 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
- Locations
-
- CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
NCT04227600
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Instituto de Genética e Erros Inatos do Metabolismo - IGEIM São Paulo, Brazil
- Fukuoka Children's Hospital Fukuoka, Japan
NCT00695279
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +31 more
- Locations
-
- St. Jude Children's Research Hospital Memphis, Tennessee, United States
NCT03053089
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- HCPA - Hospital das Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
Completed
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
NCT03071341
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- HCPA - Hospital das Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil