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

January 9, 2019 updated by: Dr. Tawhida Yassin Abdel Ghaffar, Yassin Abdelghaffar Charity Center for Liver Disease and Research

Hereditary Hepatorenal Tyrosinemia Natural History (Multicenter Clinical Study): Registry for Patients With Tyrosinemia Type I in Egypt and the Arab World

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.

Study Overview

Status

Recruiting

Conditions

Detailed 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.

Study Type

Observational

Enrollment (Anticipated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Egypt
    • Cairo
      • Nasr City, Cairo, Egypt
        • Recruiting
        • Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research
        • Principal Investigator:
          • Tawhida Y. Abdel Ghaffar, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with tyrosinemia type I seen by one of the participating sites.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Create a registry for tyrosinemia type I.
Time Frame: 5 Years
This outcome is a binary 'yes/no' outcome as to whether or not this study can successfully create a repository with the intent to store data and specimens to support the conduct of future research on tyrosinemia type I.
5 Years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Yassin Abdelghaffar Charity Center for Liver Disease and Research

Collaborators

Society of Friends of Liver Patients in the Arab World (SLPAW)

Investigators

  • Principal Investigator: Tawhida Y Abdelghaffar, MD, Yassin Abdelghaffar Charity Center for Liver Disease and Research

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ANTICIPATED)

April 5, 2019

Primary Completion (ANTICIPATED)

November 5, 2023

Study Completion (ANTICIPATED)

December 31, 2023

Study Registration Dates

First Submitted

February 4, 2018

First Submitted That Met QC Criteria

February 19, 2018

First Posted (ACTUAL)

February 27, 2018

Study Record Updates

Last Update Posted (ACTUAL)

January 10, 2019

Last Update Submitted That Met QC Criteria

January 9, 2019

Last Verified

January 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Tyrosinemia Registry E&AW 1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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