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

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ClinicalTrials.gov Identifier: NCT03446586
Recruitment Status : Recruiting
First Posted : February 27, 2018
Last Update Posted : January 10, 2019
Sponsor:
Collaborator:
Society of Friends of Liver Patients in the Arab World (SLPAW)
Information provided by (Responsible Party):
Dr. Tawhida Yassin Abdel Ghaffar, Yassin Abdelghaffar Charity Center for Liver Disease and Research

Brief 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.

Condition or disease
Hereditary Tyrosinemia, Type I

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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Study Type : Observational [Patient Registry]
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration: 5 Years
Official Title: Hereditary Hepatorenal Tyrosinemia Natural History (Multicenter Clinical Study): Registry for Patients With Tyrosinemia Type I in Egypt and the Arab World
Estimated Study Start Date : April 5, 2019
Estimated Primary Completion Date : November 5, 2023
Estimated Study Completion Date : December 31, 2023





Primary Outcome Measures :
  1. 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.


Biospecimen Retention:   Samples With DNA
Fixed liver block samples & frozen whole blood.


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with tyrosinemia type I seen by one of the participating sites.
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03446586


Contacts
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Contact: Mamdouh A. Ahmed 02-01221707770 dr.yassinabdelghaffar@gmail.com

Locations
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Egypt
Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research Recruiting
Nasr City, Cairo, Egypt
Contact: Mamdouh A. Ahmed    02-01221707770    dr.yassinabdelghaffar@gmail.com   
Principal Investigator: Tawhida Y. Abdel Ghaffar, MD         
Sponsors and Collaborators
Yassin Abdelghaffar Charity Center for Liver Disease and Research
Society of Friends of Liver Patients in the Arab World (SLPAW)
Investigators
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Principal Investigator: Tawhida Y Abdelghaffar, MD Yassin Abdelghaffar Charity Center for Liver Disease and Research

Additional Information:

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Responsible Party: Dr. Tawhida Yassin Abdel Ghaffar, Professor, Yassin Abdelghaffar Charity Center for Liver Disease and Research
ClinicalTrials.gov Identifier: NCT03446586     History of Changes
Other Study ID Numbers: Tyrosinemia Registry E&AW 1
First Posted: February 27, 2018    Key Record Dates
Last Update Posted: January 10, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Tyrosinemias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases