Tyrosinemias

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Tyrosinemias trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT06941532

GMP Powdered Substitutes in PKU and TYR

Four new GMP-based protein substitutes have been developed to support the dietary management of PKU and TYR. These products are powdered protein substitutes, lo…

United Kingdom40 participantsStarted 2025-05-20

Treatment: GMP Intervention Product

All recent trials (21 shown)

CompletedNot Applicable

NCT05051657

Evaluation of the Express Plus Range

A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The follow…

United Kingdom28 participantsStarted 2021-06-14

Treatment: PKU express plus

WithdrawnNot Applicable

NCT06227429

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

This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine cli…

China0Started 2025-09

Treatment: Nitisinone

Tyrosinemias

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

GMP Powdered Substitutes in PKU and TYR

All recent trials (21 shown)

Evaluation of the Express Plus Range

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

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

GMP Powdered Substitutes in PKU and TYR

All recent trials (21 shown)

Evaluation of the Express Plus Range

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

Evaluation of TYR Sphere in France

Baby Detect : Genomic Newborn Screening

Early Check: Expanded Screening in Newborns

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

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

Evaluation of TYR Sphere

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

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

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

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

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

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

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

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

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

Study of NTBC for Tyrosinemia I

Taste and Palatability of Orfadin Suspension

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