The World Federation of Hemophilia Gene Therapy Registry (WFH GTR)

May 11, 2021 updated by: World Federation of Hemophilia
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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Anticipated)

5000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Donna Coffin, MSc
  • Phone Number: 514.875.7944
  • Email: dcoffin@wfh.org

Study Contact Backup

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 100 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

All people with hemophilia who have received a gene therapy product

Description

Inclusion Criteria:

  • People with hemophilia who have received a gene therapy product

Exclusion Criteria:

  • None

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
From baseline, through study completion (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.
From baseline, through study completion (15 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

World Federation of Hemophilia

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

January 1, 2022

Primary Completion (Anticipated)

January 1, 2050

Study Completion (Anticipated)

January 1, 2050

Study Registration Dates

First Submitted

February 20, 2020

First Submitted That Met QC Criteria

May 11, 2021

First Posted (Actual)

May 12, 2021

Study Record Updates

Last Update Posted (Actual)

May 12, 2021

Last Update Submitted That Met QC Criteria

May 11, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2020-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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