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Clinical trial condition

Mucopolysaccharidosis I

Explore clinical trials studying Mucopolysaccharidosis I. Study availability and eligibility vary by location and protocol.

53 public trials 6 recruiting studies
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Mucopolysaccharidosis I trial results

Active, not recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • University of Minnesota, Pediatrics Minneapolis, Minnesota, United States
  • Ospedale San Raffaele Milan, Italy
  • Princess Maxima Center Utrecht, Netherlands
  • UMC Utrecht Utrecht, Netherlands
View Trial Details
Completed

Mucopolysaccharidosis I (MPS I) Registry

NCT00144794

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • Arkansas Children's Hospital- Site Number : 840109 Little Rock, Arkansas, United States
  • Southern California Permanente Medical Group- Site Number : 840108 Los Angeles, California, United States
  • USC Health Sciences Center Dept of Genetics- Site Number : 840082 Los Angeles, California, United States
View all 141 locations
View Trial Details
Completed

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

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
View Trial Details
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
View Trial Details
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
View Trial Details
Completed

A Study of JR-171 in Patients With Mucopolysaccharidosis I

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
View Trial Details
Completed

Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

NCT06406153

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine Mashhad, Iran
  • Growth and Development Research Center, Childrens Medical Center Tehran, Iran
  • Loghman Hospital Tehran, Iran
  • Mofid Childrens Hospital Tehran, Iran
View Trial Details

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