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Status unknownNCT03446586Updated Jan 10, 2019

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

An observational study in Hereditary Tyrosinemia, Type I, sponsored by Yassin Abdelghaffar Charity Center for Liver Disease and Research. Status unknown at 1 site in Egypt. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2019-01-10.

Sponsored by Yassin Abdelghaffar Charity Center for Liver Disease and Research · Observational

The sponsor has not verified this record recently (last verified Jan 2019), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Cohort
Time perspective
Other
Enrollment
50
Ages
Up to 18 Years
Sex
All
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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.

Read the 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.

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Conditions studied

  • Hereditary Tyrosinemia, Type I

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Who can participate

Ages eligible
Up to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

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

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

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.
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Study design

Observational model
Cohort
Time perspective
Other
Enrollment
50 participants (estimated)
Target follow-up
5 Years
Patient registry
Yes
Biospecimen retention
Samples with dna
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What researchers measure

Primary outcomes

  1. 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.

    Time frame: 5 Years

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Study locations

1 of 1 sites recruiting
  • Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research
    Nasr City, Cairo, Egypt
    Recruiting
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

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Registry details

Key details

Study ID
NCT03446586
Lead sponsor
Yassin Abdelghaffar Charity Center for Liver Disease and Research
Collaborators
Society of Friends of Liver Patients in the Arab World (SLPAW)
Responsible party
Dr. Tawhida Yassin Abdel Ghaffar (Professor, Yassin Abdelghaffar Charity Center for Liver Disease and Research) — Principal investigator
First posted
Feb 27, 2018
Start date
Apr 5, 2019 (estimated)
Primary completion
Nov 5, 2023 (estimated)
Completion
Dec 31, 2023 (estimated)
Last update
Jan 10, 2019

Study contacts

Mamdouh A. Ahmed
Contact
dr.yassinabdelghaffar@gmail.com
02-01221707770
Tawhida Y Abdelghaffar, MD
principal investigator · Yassin Abdelghaffar Charity Center for Liver Disease and Research

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Jan 2019. You cannot join it, but the record below documents what was studied.

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