Genetic Diseases, X-Linked Clinical Trials

Completed

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +24 more
Locations: St. Joseph's Hospital Phoenix, Arizona, United States

Pediatric Clinical Research Center, Children's Hospital Oakland Oakland, California, United States

The Children's Hospital Denver, Colorado, United States

Harbin Clinic Rome, Georgia, United States

View all 52 locations

View Trial Details

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

NCT00069641

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations: Children's Hospital Oakland Oakland, California, United States

St. Louis Children's Hospital, Washington University St Louis, Missouri, United States

University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States

Texas Children's Hospital, Baylor College of Medicine Houston, Texas, United States

View Trial Details

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

NCT01449240

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations: Emory University Decatur, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States

Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States

University of North Carolina, Division of Genetics and Metabolism Chapel Hill, North Carolina, United States

View Trial Details

Completed

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Emory Division of Medical Genetics Decatur, Georgia, United States

Duke University Medical Center Durham, North Carolina, United States

Baylor University Medical Center Dallas, Texas, United States

University of Utah Hospital Salt Lake City, Utah, United States

View Trial Details

Completed

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

NCT00864851

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: AKDHC Tucson Access Center Tucson, Arizona, United States

University of Iowa Hospitals and Clinics Iowa City, Iowa, United States

New York Unversity School of Medicine New York, United States

O & O Alpan, LLC Springfield, Virginia, United States

View Trial Details

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

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

View Trial Details

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

Genetic Diseases, X-Linked

Genetic Diseases, X-Linked trial results

Related conditions