Clinical trial NCT06227429: A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
Official title: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hereditary Tyrosinemia, Type I
Keywords: rare diseases
fumarylacetoacetate hydrolase (FAH) deficiency
liver failure
drug treatment
nitisinone
observational research
phase IV
China
pediatric patient
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2024-01-26
Enrollment: 15
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Investigators at selected sites in China will attempt to consecutively enroll all eligible patients who present for a routine clinical visit.

Brief 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.

Detailed 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, 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.

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

Primary outcomes:
measure: 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.
Secondary outcomes:
measure: Occurrence of 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.
measure: Occurrence of liver transplantation
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.
measure: Occurrence of hepatic malignancy
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.
measure: Occurrence of other (non-hepatic) malignancies
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.
measure: Occurrence of ophthalmic events
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.
measure: Occurrence of neurological events
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.
measure: Occurrence of cognitive, developmental function AEs
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.
measure: Occurrence of any reportable AEs
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.
measure: Treatment and diet compliance
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.
measure: Extent of exposure
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.
measure: Extent of Exposure
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.
measure: Laboratory investigations - Blood Coagulation (1)
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.
measure: Laboratory investigations - Blood Coagulation (2)
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.
measure: Laboratory investigations - Blood Coagulation (3)
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.
measure: Laboratory investigations - Blood Chemistry (1)
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.
measure: Laboratory investigations - Blood Chemistry (2)
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.
measure: Laboratory investigations - Blood Chemistry (3)
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.
measure: Laboratory investigations - Blood Chemistry (4)
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.
measure: Overall clinical condition as assessed by the investigator
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.
measure: Ophthalmic status as assessed by the investigator
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.
measure: Neurocognitive/developmental status as assessed by the investigator
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.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Swedish Orphan Biovitrum Research Site
city: Chongqing
country: China
facility: Swedish Orphan Biovitrum Research Site
city: Hefei
country: China
facility: Swedish Orphan Biovitrum Research Site
city: Wuhan
country: China
facility: Swedish Orphan Biovitrum Research Site
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Ioannis Kottakis, MD, PhD
role: STUDY_DIRECTOR
affiliation: Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology
name: Xiaoping Luo, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: