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

Last updated: January 9, 2019
Sponsor: Yassin Abdelghaffar Charity Center for Liver Disease and Research
Overall Status: Active - Recruiting

Phase

N/A

Condition

N/A

Treatment

N/A

Clinical Study ID

NCT03446586
Tyrosinemia Registry E&AW 1
  • Ages < 18
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

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

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.

Study Design

Total Participants: 50
Study Start date:
April 05, 2019
Estimated Completion Date:
December 31, 2023

Study Description

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.

Connect with a study center

  • Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research

    Nasr City, Cairo
    Egypt

    Active - Recruiting

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