Search clinical trials for: Tyrosinemia

Total 20 results

metaX Institut fuer Diatetik GmbH
Great Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...

Not yet recruiting

Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies (ZeroMinisMR)

Alkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
Swedish Orphan Biovitrum

Recruiting

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Hereditary Tyrosinemia, Type I

China
West Kazakhstan Medical University

Recruiting

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Ornithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditions

Kazakhstan
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Vitaflo International, Ltd

Recruiting

Evaluation of the Express Plus Range (express plus)

Phenylketonurias | PKU | Maple Syrup Urine Disease | Homocystinuria | Glutaric Acidemia I | Hereditary Tyrosinemia

United Kingdom
Yassin Abdelghaffar Charity Center for Liver Disease...
Society of Friends of Liver Patients in the Arab World (SLPAW)

Recruiting

Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)

Hereditary Tyrosinemia, Type I

Egypt
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
Vitaflo International, Ltd

Completed

Evaluation of TYR Sphere

Alkaptonuria | Tyrosinemias | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III

United Kingdom
Vitaflo International, Ltd

Active, not recruiting

Evaluation of TYR Sphere in France (TYR sphere Fra)

Tyrosinemia | AKU

France
Nutricia UK Ltd

Completed

GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR

PKU | Tyrosinemias

United Kingdom
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin) (BioTyrosin)

Metabolic Disorders | Tyrosinosis | Hepatorenal Tyrosinemia | Fumarylacetoacetase Deficiency | Fah Deficiency

Germany, Sri Lanka, India
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Sutphin Drugs

Unknown

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine (Orfadin)

Hereditary Tyrosinemia, Type I

India
Cycle Pharmaceuticals Ltd.
Parexel

Completed

Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)

Hereditary Tyrosinemia, Type I

South Africa
Cycle Pharmaceuticals Ltd.
Parexel

Completed

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

Hereditary Tyrosinemia, Type I

South Africa
Cycle Pharmaceuticals Ltd.
Parexel

Completed

Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

Hereditary Tyrosinemia, Type I

South Africa
Swedish Orphan Biovitrum

Completed

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Sweden
University of Washington

Completed

Study of NTBC for Tyrosinemia I

Tyrosinemia I

United States, Canada
Swedish Orphan Biovitrum

Completed

Taste and Palatability of Orfadin Suspension

Hereditary Tyrosinemia, Type I

Germany, United Kingdom, France
National Center for Research Resources (NCRR)
University of Michigan

Completed

Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Tyrosinemia, Type I

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