WithdrawnNot Applicable

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

Stopped: No patients were prescribed to the study treatment due to the generic treatment being available.

China0Started 2025-09

Plain-language summary

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 clinical setting. No tests or examinations are mandated in the study.

Who can participate

SexALL

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Inclusion criteria

✓. Patients with a confirmed diagnosis of HT-1 treated with, or at enrollment prescribed, Nitisinone treatment (product manufactured by Sobi) in a routine clinical care setting. The decision to initiate treatment shall be made by the treating physician independently from the decision to include the patient in the study.
✓. Signed and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age, should be obtained before any study-related activities are undertaken. Assent should be obtained from pediatric patients according to local regulations

What they're measuring

Occurrence of hepatic, renal or hematological adverse events (AEs) or death

Timeframe: Data will be collected for all routine visits completed during the study period which is at least 12 months but no more than 3.5 years.

Trial details

NCT IDNCT06227429

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2027-06-30

View on ClinicalTrials.gov More Hereditary Tyrosinemia, Type I trials