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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.

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Carbohydrate Metabolism, Inborn Errors trial results

Enrolling by Invitation

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

NCT07393373

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
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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
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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
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