NCT03446586

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

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

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Yassin Abdelghaffar Charity Center for Liver Disease and Research · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Hereditary Tyrosinemia, Type I

Timeline

• Start date: 2019-04-05
• Primary completion: 2023-11-05
• Completion: 2023-12-31
• First posted: 2018-02-27
• Last updated: 2019-01-10

Locations

1 site across 1 country: Egypt