Hereditary Tyrosinemia, Type INCT03446586
Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)
An observational study of Hereditary Tyrosinemia, Type I, run by Yassin Abdelghaffar Charity Center for Liver Disease and Research at 1 site in Egypt.
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Hereditary Tyrosinemia, Type I
- How many people
- 50 participants
- Where
- 1 siteEgypt
- Study dates
- 5 Apr 2019to 31 Dec 2023
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients with tyrosinemia type I seen by one of the participating sites.
Who may be able to join
- Biochemical or molecular diagnosis of Tyrosinemia Type I.
- Examined/followed by one of the participating sites.
- Parental/guardian permission (informed consent) for participation.
Who cannot join
- Diagnosis of tyrosinemia has been excluded.
- Not examined/followed by one of the participating sites.
- Unwilling to provide informed consent for participation.
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
Professor Yassin Abdel Ghaffar Charity Center for Liver Disease and Research
Madīnat an Naşr, Cairo Governorate
Mamdouh A. Ahmed, 02-01221707770, dr.yassinabdelghaffar@gmail.com
Tawhida Y. Abdel Ghaffar, MD
About this study
Official title: Hereditary Hepatorenal Tyrosinemia Natural History (Multicenter Clinical Study): Registry for Patients With Tyrosinemia Type I in Egypt and the Arab World
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.
- Conditions
- Hereditary Tyrosinemia, Type I
- Medical subject headings
- Tyrosinemias
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 5 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2018
- Study start5 Apr 2019 (estimated)
- Primary completion5 Nov 2023 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Tyrosinemia, Type IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 50 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Yassin Abdelghaffar Charity Center for Liver Disease and Research
- Sponsor type
- University, hospital or other
- Collaborators
- Society of Friends of Liver Patients in the Arab World (SLPAW)
- Responsible party
- Principal investigator, Dr. Tawhida Yassin Abdel Ghaffar, Yassin Abdelghaffar Charity Center for Liver Disease and Research
- Investigators
- Tawhida Y Abdelghaffar, MD, Principal investigator, Yassin Abdelghaffar Charity Center for Liver Disease and Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03446586
- Sponsor's protocol ID
- Tyrosinemia Registry E&AW 1
- Status last verified
- Jan 2019
- First submitted
- 4 Feb 2018
- Last updated
- 10 Jan 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hereditary Hepatorenal Tyrosinemia Natural History (Multicenter Clinical Study): Registry for Patients With Tyrosinemia Type I in Egypt and the Arab World
- ClinicalTrials.gov ID
- NCT03446586
- Acronym / sponsor ID
- Tyrosinemia Registry E&AW 1
- Conditions
- Hereditary Tyrosinemia, Type I
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Yassin Abdelghaffar Charity Center for Liver Disease and ResearchUniversity, hospital or other
- Collaborators
- Society of Friends of Liver Patients in the Arab World (SLPAW)
- Study start
- 5 Apr 2019 (estimated)
- Primary completion
- 5 Nov 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 27 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 5 Years
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with tyrosinemia type I seen by one of the participating sites.
Key inclusion criteria (3 listed)
- Biochemical or molecular diagnosis of Tyrosinemia Type I.
- Examined/followed by one of the participating sites.
- Parental/guardian permission (informed consent) for participation.
Key exclusion criteria (3 listed)
- Diagnosis of tyrosinemia has been excluded.
- Not examined/followed by one of the participating sites.
- Unwilling to provide informed consent for participation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tawhida Y Abdelghaffar, MD principal investigator
Yassin Abdelghaffar Charity Center for Liver Disease and Research
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Tyrosinemia, Type I, run by Yassin Abdelghaffar Charity Center for Liver Disease and Research at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mamdouh A. Ahmed (02-01221707770, dr.yassinabdelghaffar@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Create a registry for tyrosinemia type I. (time frame: 5 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2019.