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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

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

An Interventional Study of Infigratinib in Children With Hypochondroplasia

NCT06873035

Conditions
Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Johns Hopkins School of Medicine Baltimore, Maryland, United States
View all 24 locations
View Trial Details
Enrolling by invitation

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

NCT06075537

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Hackensack University Medical Center Hackensack, New Jersey, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
View all 29 locations
View Trial Details
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
View Trial Details
Enrolling by invitation

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Hospital Universitario Austral Buenos Aires, Argentina
  • Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
View Trial Details
Enrolling by invitation

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT05145010

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +15 more
Locations
  • USCF Benioff Children's Hospital, Oakland Oakland, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nemours Alfred I. Dupont Hospital for Children Wilmington, Delaware, United States
  • Johns Hopkins University Baltimore, Maryland, United States
View all 31 locations
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

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

NCT05208281

Conditions
Carbohydrate Metabolism, Inborn Errors, Cognition Disorders, Cognitive Dysfunction, +30 more
Locations
  • Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Academy of Sciences (Research Institute of Pediatrics and Child Health Protection of the Central Clinical Hospital of the Russian Academy of Sciences) Moscow, Russia
  • Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation Saint Petersburg, Russia
  • V.I. Vernadsky Crimean Federal University Simferopol, Russia
  • State Budgetary Healthcare Institution Republican Medical Genetic Center Ufa, Russia
View Trial Details
Active, not recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

NCT05371613

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Hackensack University Medical Center Hackensack, New Jersey, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
View all 30 locations
View Trial Details

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