Clinical trial

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

Name

Sobi.NTBC-008

Description

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.

Trial arms

Trial start

2023-12-14

Estimated PCD

2027-06-30

Trial end

2027-06-30

Status

Recruiting

Treatment

Nitisinone

According to prescription

Arms:

Full Analysis Set (FAS)

Other names:

Orfadin®

Size

Primary endpoint

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

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.

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

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'CASE_ONLY', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 15, 'type': 'ESTIMATED'}}

Updated at

2024-02-07

1 organization

1 product

2 indications

Organization

Swedish Orphan Biovitrum

Product

Nitisinone

Indication

Hereditary Tyrosinemia

Indication

Type I