Tyrosinemias clinical trials
The registry lists 20 studies for Tyrosinemias across more than 10 countries. 3 are recruiting or about to start. 15 of them are mainly about Tyrosinemias (how this is decided). Per ClinicalTrials.gov, last updated 2026-07-01.
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Who runs them
Swedish Orphan Biovitrum (4) · Cycle Pharmaceuticals Ltd. (3) · Vitaflo International, Ltd (3) · Nutricia UK Ltd (2) · RTI International (1) · National Center for Research Resources (NCRR) (1) · Centre Hospitalier Universitaire de Liege (1) · metaX Institut fuer Diatetik GmbH (1)
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026GMP Powdered Substitutes in PKU and TYR · Nutricia UK Ltd
Who these studies take
Of the 2 Tyrosinemias studies recruiting now, 1 (50%) set no upper age limit and 1 (50%) stop at 75 or younger. 2 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (50%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (100%) | 2 (100%) |
| 18 | 2 (100%) | 2 (100%) |
| 30 | 1 (50%) | 1 (50%) |
| 40 | 1 (50%) | 1 (50%) |
| 50 | 1 (50%) | 1 (50%) |
| 65 | 1 (50%) | 1 (50%) |
| 75 | 1 (50%) | 1 (50%) |
| 85 | 1 (50%) | 1 (50%) |
Across all 20 studies, recruiting or not: 50% stop at 75 or younger and 50% at 65; 80% take children; 0% are for women only and 0% for men only; 30% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 20 of 20. search them.
- GMP Powdered Substitutes in PKU and TYRRecruitingNCT06941532Not applicable1 country
- Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare AminoacidopathiesNot yet recruitingNCT06298292
- Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in KazakhstanRecruitingNCT059101511 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Evaluation of the Express Plus RangeCompletedNCT05051657Not applicable1 country
- A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 PatientsWithdrawnNCT062274291 country
- Evaluation of TYR Sphere in FranceCompletedNCT04761588Not applicable1 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical CareCompletedNCT0232008417 countries
- Evaluation of TYR SphereCompletedNCT04196959Not applicable1 country
- GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYRCompletedNCT05062226Not applicable1 country
- Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin)WithdrawnNCT032846583 countries
- Bio Equivalency 20 Mgm Orfadin and 20 Mgm of NitisonineStatus unknownNCT04113772Not applicable1 country
- Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)Status unknownNCT034465861 country
- Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)CompletedNCT02750345Phase 11 country
- Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)CompletedNCT02750332Phase 11 country
- Bioequivalence Study of Two Nitisinone Formulations Compared to OrfadinCompletedNCT02750709Phase 11 country
- Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1CompletedNCT02323529Phase 35 countries
- Taste and Palatability of Orfadin SuspensionCompletedNCT01734889Phase 13 countries
- Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type ICompletedNCT00004333Phase 2