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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.

2,499 public trials 456 recruiting studies
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Metabolism, Inborn Errors trial results

Completed

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

NCT01124643

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • O&O Alpan LLC Fairfax, Virginia, United States
  • The Royal Melbourne Hospital Parkville, Australia
  • 1st School of Medicine Charles University Prague, Czechia
View Trial Details
Completed

A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

NCT01298141

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Alberta Children's Hospital Calgary, Alberta, Canada
  • University of Alberta Hospital Edmonton, Alberta, Canada
  • Vancouver General Hospital Vancouver, British Columbia, Canada
  • University of Manitoba Winnipeg, Manitoba, Canada
View Trial Details
Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

NCT00607386

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +24 more
Locations
  • Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica Porto Alegre, Rio Grande do Sul, Brazil
  • Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii Warsaw, Poland
  • National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics Taipei, Taiwan
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Completed

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Duke Children's Hospital & Health Center Durham, North Carolina, United States
  • Your Health S.A. Buenos Aires, Argentina
  • Malabar Institute of Medical Sciences Ltd. Calicut, Kerala, India
  • All India Institute of Medical Sciences New Delhi, India
View Trial Details
Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +18 more
Locations
  • Los Angeles Biomedical Research Torrance, California, United States
  • University of Minnesota Department of Pediatrics Minneapolis, Minnesota, United States
  • University of North Carolina Chapel Hill, North Carolina, United States
  • Hospital Universitario Austral A Unidad de Investigacion Buenos Aires, Argentina
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Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

NCT00882921

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations
  • Children's Hospital & Research Center Oakland Oakland, California, United States
  • Children's Hospitals and Clinics of Minnesota, Division of Genetics Minneapolis, Minnesota, United States
  • Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica Porto Alegre, Rio Grande do Sul, Brazil
  • Birmingham Children's Hospital Birmingham, United Kingdom
View Trial Details
Completed

Sophisticated Assessment of Disease Burden in Patients With Fabry Disease

NCT01210196

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • ZNA Middelheim Antwerp, Belgium
  • Institute of Pathology et de Génétique (IPG) Gosselies, Belgium
  • Charles University in Prague and General University Hospital in Prague Prague, Czechia
  • National University Hospital, Rigshospitalet Copenhagen, Denmark
View Trial Details
Completed

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

NCT02416661

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • University Hospital Center Mother Teresa Tirana, Albania
  • Aristotle University of Thessaloniki, Ippokration General Hospital Thessaloniki, Greece
  • Centre for Human Genetics Bangalore, India
  • Shaare Zedek Medical Center Jerusalem, Israel
View Trial Details

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