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Clinical trial condition

Carbohydrate Metabolism, Inborn Errors

Explore clinical trials studying Carbohydrate Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

551 public trials 96 recruiting studies
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Carbohydrate Metabolism, Inborn Errors trial results

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

Sirolimus for Leigh Syndrome

NCT06843811

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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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
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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

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