A Prospective, Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in Hereditary Tyrosinemia Type 1 (HT-1) Patients in Routine Clinical Care in China
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Observational
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.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
China
Swedish Orphan Biovitrum Research Site
NOT_YET_RECRUITING
Beijing
Swedish Orphan Biovitrum Research Site
NOT_YET_RECRUITING
Chongqing
Swedish Orphan Biovitrum Research Site
RECRUITING
Hefei
Swedish Orphan Biovitrum Research Site
RECRUITING
Wuhan
Contact Information
Primary
Catrine Berlin, MSc
medical.info@sobi.com
+4686972000
Backup
Ioannis Kottakis, MD, PhD
medical.info@sobi.com
+41 61 201 13 20
Time Frame
Start Date:2025-09
Estimated Completion Date:2027-06-30
Participants
Target number of participants:15
Treatments
Full Analysis Set (FAS)
The Full Analysis Set (FAS) will include all patients with a confirmed HT-1 diagnosis on Nitisinone treatment in routine clinical care, who provide signed informed consent. Patients must be either on treatment with Nitisinone at study entry or they must have been prescribed Nitisinone at enrollment. No specific exclusion criteria from the analysis set will be applied. The FAS will be used for all analyses.