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

Hereditary Tyrosinemia, Type I Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03446586
• Other study ID #: Tyrosinemia Registry E&AW 1
• Status: Recruiting
• Start date: April 5, 2019
• Est. completion date: December 31, 2023

Study information

• Verified date: January 2019
• Source: Yassin Abdelghaffar Charity Center for Liver Disease and Research
• Contact: Mamdouh A. Ahmed
• Phone: 02-01221707770
• Email: dr.yassinabdelghaffar[@]gmail.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: December 31, 2023
• Est. primary completion date: November 5, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hereditary Tyrosinemia, Type I
• Tyrosinemias

Locations

Country	Name	City	State
Egypt	Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research	Nasr City	Cairo

Sponsors (2)

Lead Sponsor	Collaborator
Yassin Abdelghaffar Charity Center for Liver Disease and Research	Society of Friends of Liver Patients in the Arab World (SLPAW)

Country where clinical trial is conducted

Egypt, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Create a registry for tyrosinemia type I.	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