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

NCT ID: NCT06227429

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2025-09-30

Study Completion Date

2027-06-30

Brief Summary

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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.

Detailed Description

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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, though the expectation is that most of the tests and examinations listed in the protocol will be performed in the context of routine clinical care and relevant data will be captured. At enrollment, data on patient treatment, medical and surgical history together with other patient characteristics will be captured.Patients enrolled in the study will be followed for at least 1 year and for a maximum of 3.5 years.

The study aims to enroll at least 15 HT-1 patients aged 0-18 years. If adult patients are enrolled the study population will be larger as all eligible patients will be invited to participate. However, the enrollment will close when the target of 15 patients aged 0-18 years has been reached.

Conditions

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Hereditary Tyrosinemia, Type I

Keywords

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rare diseases fumarylacetoacetate hydrolase (FAH) deficiency liver failure drug treatment nitisinone observational research phase IV China pediatric patient

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

Nitisinone

Intervention Type DRUG

According to prescription

Interventions

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Nitisinone

According to prescription

Intervention Type DRUG

Other Intervention Names

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Orfadin®

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ioannis Kottakis, MD, PhD

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum

Xiaoping Luo, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology

Locations

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Swedish Orphan Biovitrum Research Site

Beijing, , China

Site Status

Swedish Orphan Biovitrum Research Site

Chongqing, , China

Site Status

Swedish Orphan Biovitrum Research Site

Hefei, , China

Site Status

Swedish Orphan Biovitrum Research Site

Wuhan, , China

Site Status

Countries

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China

Other Identifiers

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Sobi.NTBC-008

Identifier Type: -

Identifier Source: org_study_id