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Clinical trial condition

Metabolism, Inborn Errors

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

2,496 public trials 457 recruiting studies
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Metabolism, Inborn Errors trial results

Enrolling by Invitation

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +13 more
Locations
  • Cedars Sinai Medical Center Los Angeles, California, United States
  • Palo Alto Veteran Affairs Hospital Palo Alto, California, United States
  • Northwestern Medicine Chicago, Illinois, United States
  • Providence Heart and Vascular Institute Portland, Oregon, United States
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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
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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
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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
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Enrolling by Invitation

A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

NCT06431893

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Emory University Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 52 locations
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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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