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

Launched by SWEDISH ORPHAN BIOVITRUM · Jan 17, 2024

Keywords

ClinConnect Summary

This clinical trial is studying how well a medication called Nitisinone works for patients with Hereditary Tyrosinemia, Type I (HT-1) in China. The trial is not comparing treatments or requiring specific tests; instead, it is collecting information from patients who are already receiving Nitisinone as part of their regular medical care. The goal is to understand the outcomes of this treatment in everyday clinical settings.

To participate, patients must have a confirmed diagnosis of HT-1 and be receiving Nitisinone treatment or be prescribed it during the study. Patients, or their authorized representatives if they are minors, need to give their consent to join the study. Participants should not be involved in any other clinical trials or take any experimental medications three months prior to joining this study. Overall, this trial aims to gather valuable information without placing extra burdens on the participants.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. 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.
• • 2. 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
• Exclusion Criteria:
• • 1. Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study