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Clinical trial condition

Mucopolysaccharidosis III

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

41 public trials 8 recruiting studies
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Mucopolysaccharidosis III trial results

Enrolling by Invitation

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799

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
  • UPMC Children's Hospital Pittburgh Pittsburgh, Pennsylvania, United States
  • Fundación Cardio Infantil - Instituto de Cardiología Bogotá, Colombia
  • University Medical Center Hamburg-Eppendorf Hamburg, Germany
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Completed

Open-label Study of Anakinra in MPS III

NCT04018755

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations
  • The Lundquist Institute at Harbor-UCLA Medical Center Torrance, California, United States
View Trial Details
Completed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

NCT03811028

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Children´s Hospital and research center Oakland, California, United States
  • University of North Carolina hospitals Chapel Hill, North Carolina, United States
  • Gazi University Hospital Ankara, Turkey (Türkiye)
View Trial Details
Completed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

NCT03423186

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Childrens's Hospital and Research Center Oakland, California, United States
  • University of North Carolina Hospitals Chapel Hill, North Carolina, United States
  • University Medical Center Hamburg-Eppendorf Hamburg, Germany
  • Gazi University Hospital Ankara, Turkey (Türkiye)
View Trial Details

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