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Clinical trial condition

Mucopolysaccharidosis III

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

42 public trials 7 recruiting studies
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Mucopolysaccharidosis III trial results

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
View Trial Details
Enrolling by invitation

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
Locations
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • Women's and Children's Hospital North Adelaide, South Australia, Australia
  • Vall d'Hebron Barcelona Campus Barcelona, Spain
  • Hospital Clínico Universitario de Santiago Santiago de Compostela, Spain
View Trial Details
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
View Trial Details
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

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

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