Hereditary Hepatorenal Tyrosinemia Natural History in Egy... | NCT03446586

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

NCT03446586

Sponsor

Yassin Abdelghaffar Charity Center for Liver Disease and Research

Status

Unknown status

Last Update Posted

Jan 10, 2019Actual

Enrollment

50Estimated

Phase

Not provided

Conditions

Hereditary Tyrosinemia, Type I

Interventions

Not provided

Countries

Egypt

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jan 2019

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 5, 2019Estimated

Primary Completion Date

Nov 5, 2023Estimated

Completion Date

Dec 31, 2023Estimated

First Submitted Date

Feb 4, 2018

First Submission Date that Met QC Criteria

Feb 19, 2018

First Posted Date

Feb 27, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 9, 2019

Last Update Posted Date

Jan 10, 2019Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Dr. Tawhida Yassin Abdel Ghaffar, Professor, Yassin Abdelghaffar Charity Center for Liver Disease and ResearchPrincipal Investigator

Lead Sponsor

Yassin Abdelghaffar Charity Center for Liver Disease and ResearchOTHER

Collaborators

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

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

5 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

50Estimated

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Biochemical or molecular diagnosis of Tyrosinemia Type I.
Examined/followed by one of the participating sites.
Parental/guardian permission (informed consent) for participation.

Exclusion Criteria:

Diagnosis of tyrosinemia has been excluded.
Not examined/followed by one of the participating sites.
Unwilling to provide informed consent for participation.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Mamdouh A. Ahmed	Contact	02-01221707770		dr.yassinabdelghaffar@gmail.com

Overall Officials

Name	Affiliation	Role
Tawhida Y Abdelghaffar, MD	Yassin Abdelghaffar Charity Center for Liver Disease and Research	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research	Recruiting	Madīnat an Naşr	Cairo Governorate		Egypt

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D020176	Tyrosinemias

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

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Browse Branches

Not provided