Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)

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

UNKNOWN

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-04-05

Study Completion Date

2023-12-31

Brief Summary

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The purpose of the registry/repository is to understand the natural history of tyrosinemia in our region and to provide a mechanism to store data and specimens to support the conduct of future research about hereditary tyrosinemia among the Arabs.

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Detailed Description

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The purpose of this study is to create an electronic registry of phenotypic, laboratory information, treatment and outcomes options for tyrosinemia type I. The registry is longitudinal in nature including retrospective clinical data from birth to the most recent encounter with all data entered in chronological fashion. The goals of this registry are the better understanding of the natural history and treatment outcomes of these patients and to determine/evaluate biochemical and clinical parameters for monitoring and prognosis of tyrosinemia type I.

Conditions

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Hereditary Tyrosinemia, Type I

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Eligibility Criteria

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Inclusion Criteria

1. Biochemical or molecular diagnosis of Tyrosinemia Type I.
2. Examined/followed by one of the participating sites.
3. Parental/guardian permission (informed consent) for participation.

Exclusion Criteria

1. Diagnosis of tyrosinemia has been excluded.
2. Not examined/followed by one of the participating sites.
3. Unwilling to provide informed consent for participation.

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No