HemophiliaWFH GTRNCT04883710
The World Federation of Hemophilia Gene Therapy Registry
An observational study of Hemophilia, run by World Federation of Hemophilia.
Trial summary
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed. Read more about this study
- Who is it for?
- Ages 0 to 100 years
- Conditions the study is for
- Hemophilia
- What is being tested?
- Gene therapy
- How many people
- 5,000 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2022to 1 Jan 2050
- How long will I take part?
- 100 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 0 to 100 years
- All sexes
- Child, Adult, Older adult
Study population: All people with hemophilia who have received a gene therapy product
Who may be able to join
- People with hemophilia who have received a gene therapy product
Who cannot join
- None
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
Read the detailed description
The first gene therapy product for hemophilia is expected to receive marketing authorization in 2020, with several others following closely behind. With these new therapies hitting the market in the near future, it is incumbent on us as a community to ensure that we have measures in place to allow monitoring of expected, unexpected and unknown safety issues, as well as duration of efficacy, over the long term. Low incidence adverse events require monitoring of a large number of patients, over a long period of time, necessitating a global initiative.
To fill this need, the World Federation of Hemophilia (WFH), under the guidance of a multi-stakeholder steering committee, is developing and implementation the WFH Gene Therapy Registry (GTR). The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
The WFH GTR steering committee is made up of hematologists, clinical trialists, methodologists, epidemiologists, manufacturers and patient advocates. All stakeholders have a role in ensuring the success of this important initiative.
The WFH GTR will collect a core set of demographic, safety and efficacy data, on all patients with hemophilia, who receive a gene therapy product via a clinical trial or post-marketing, from anywhere in the world. The core data set is currently in development; and the global implementation plan is being finalized. A database provider will be selected in Q1 2020, based on a request for proposals which was issued to several database providers. The database for the WFH GTR will begin development in Q2 2020, with the aim of having a final database ready for launch in by end of 2020, when the first gene therapy product is available to patients. At this time, we will also begin recruitment of patients who have received gene therapy through a clinical trial.
Patients will be recruited through hemophilia treatment centers (HTC) who provide gene therapy to patients with hemophilia. Investigators will recruit patients into the WFH GTR and enter baseline and follow up data over the long term. There is no determined end date for this registry. For countries who have an existing national registry, we offer the possibility of linking databases directly.
- Conditions
- Hemophilia
- Being tested
- Gene therapy
- Keywords
- Gene therapy
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene therapyOther
This is a prospective patient registry.
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in long term safety: adverse events of interest
Months 3, 6, 9, 12, 18, 24; and annually thereafter for 15 years.
Measured: From baseline, through study completion (15 years)
Change in efficacy: factor level and bleeding events
Months 3, 6, 9, 12, 18, 24; and annually thereafter for 15 years.
Measured: From baseline, through study completion (15 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2021
- Study start1 Jan 2022 (estimated)
- Primary completion1 Jan 2050 (estimated)
- Study completion1 Jan 2050 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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
- Prospective
- Enrolment
- 5,000 participants (estimated)
- Target duration
- 100 Years
Sponsor and investigators
- Lead sponsor
- World Federation of Hemophilia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04883710
- Sponsor's protocol ID
- 2020-001
- Acronym
- WFH GTR
- Status last verified
- May 2021
- First submitted
- 20 Feb 2020
- Last updated
- 12 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The World Federation of Hemophilia Gene Therapy Registry
- ClinicalTrials.gov ID
- NCT04883710
- Acronym / sponsor ID
- WFH GTR / 2020-001
- Conditions
- Hemophilia
- Keywords
- Gene therapy
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- World Federation of HemophiliaUniversity, hospital or other
- Study start
- 1 Jan 2022 (estimated)
- Primary completion
- 1 Jan 2050 (estimated)
- Study completion
- 1 Jan 2050 (estimated)
- First posted
- 12 May 2021 (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 aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
Detailed description
The first gene therapy product for hemophilia is expected to receive marketing authorization in 2020, with several others following closely behind. With these new therapies hitting the market in the near future, it is incumbent on us as a community to ensure that we have measures in place to allow monitoring of expected, unexpected and unknown safety issues, as well as duration of efficacy, over the long term. Low incidence adverse events require monitoring of a large number of patients, over a long period of time, necessitating a global initiative.
To fill this need, the World Federation of Hemophilia (WFH), under the guidance of a multi-stakeholder steering committee, is developing and implementation the WFH Gene Therapy Registry (GTR). The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
The WFH GTR steering committee is made up of hematologists, clinical trialists, methodologists, epidemiologists, manufacturers and patient advocates. All stakeholders have a role in ensuring the success of this important initiative.
The WFH GTR will collect a core set of demographic, safety and efficacy data, on all patients with hemophilia, who receive a gene therapy product via a clinical trial or post-marketing, from anywhere in the world. The core data set is currently in development; and the global implementation plan is being finalized. A database provider will be selected in Q1 2020, based on a request for proposals which was issued to several database providers. The database for the WFH GTR will begin development in Q2 2020, with the aim of having a final database ready for launch in by end of 2020, when the first gene therapy product is available to patients. At this time, we will also begin recruitment of patients who have received gene therapy through a clinical trial.
Patients will be recruited through hemophilia treatment centers (HTC) who provide gene therapy to patients with hemophilia. Investigators will recruit patients into the WFH GTR and enter baseline and follow up data over the long term. There is no determined end date for this registry. For countries who have an existing national registry, we offer the possibility of linking databases directly.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 100 Years
Who may qualify
- Ages
- 0 to 100 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- All people with hemophilia who have received a gene therapy product
Key inclusion criteria (1 listed)
- People with hemophilia who have received a gene therapy product
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gene therapyThis is a prospective patient registry. | Other | – |
Primary outcome
Change in long term safety: adverse events of interest
Time frame: From baseline, through study completion (15 years)
Months 3, 6, 9, 12, 18, 24; and annually thereafter for 15 years.
Change in efficacy: factor level and bleeding events
Time frame: From baseline, through study completion (15 years)
Months 3, 6, 9, 12, 18, 24; and annually thereafter for 15 years.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2021.
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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.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by World Federation of Hemophilia.
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 1 inclusion and 1 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: opening soon, as of the sponsor's last update on 12 May 2021. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Donna Coffin, MSc (514.875.7944, dcoffin@wfh.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in long term safety: adverse events of interest (time frame: From baseline, through study completion (15 years)). The study lists 2 primary outcomes in all. 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 12 May 2021.