Clinical trial NCT04883710: The World Federation of Hemophilia Gene Therapy Registry
Official title: The World Federation of Hemophilia Gene Therapy Registry
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Hemophilia
Keywords: Gene therapy
Lead sponsor: World Federation of Hemophilia
Sponsor class: OTHER
First posted: 2021-05-12
Enrollment: 5000
Sex: ALL
Minimum age (years): 0
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All people with hemophilia who have received a gene therapy product

Brief 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.

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.

Eligibility criteria: Inclusion Criteria:

* People with hemophilia who have received a gene therapy product

Exclusion Criteria:

* None

Primary outcomes:
measure: Change in long term safety: adverse events of interest
timeFrame: From baseline, through study completion (15 years)
measure: Change in efficacy: factor level and bleeding events
timeFrame: From baseline, through study completion (15 years)
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
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