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Clinical trial condition

Mucopolysaccharidoses

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

193 public trials 26 recruiting studies
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Mucopolysaccharidoses trial results

Active, not recruiting

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Ajou University Medical Center Suwon, Gyeongi-do, South Korea
  • Samsung Medical Center Seoul, South Korea
View Trial Details
Active, not recruiting

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

NCT04348136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Fukui Clinical site Fukui, Japan
  • Fukuoka Clinical site 2 Fukuoka, Japan
  • Fukuoka Clinical site Fukuoka, Japan
  • Gifu Clinical site Gifu, Japan
View all 20 locations
View Trial Details
Active, not recruiting

Long-term Follow-Up for RGX-121

NCT04597385

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • University of California, San Francisco (UCSF)- Benioff Children's Hospital San Francisco, California, United States
  • Saint Peter's Healthcare System New Brunswich, New Jersey, United States
  • The Children's Hospital of Philadelphia (CHOP) Philadelphia, Pennsylvania, United States
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
View Trial Details
Active, not recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Iowa Stead Family Children's Hospital Iowa City, Iowa, United States
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
  • Baylor College of Medicine and Texas Children's Hospital Houston, Texas, United States
View Trial Details
Active, not recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
View Trial Details
Active, not recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View all 25 locations
View Trial Details

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