NCT04883710

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.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5,000

participants targeted

Target at P75+ for all trials

Timeline
289mo left

Started Jan 2022

Longer than P75 for all trials

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress15%
Jan 2022Jan 2050

First Submitted

Initial submission to the registry

February 20, 2020

Completed
1.2 years until next milestone

First Posted

Study publicly available on registry

May 12, 2021

Completed
8 months until next milestone

Study Start

First participant enrolled

January 1, 2022

Completed
28 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2050

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2050

Last Updated

May 12, 2021

Status Verified

May 1, 2021

Enrollment Period

28 years

First QC Date

February 20, 2020

Last Update Submit

May 11, 2021

Conditions

Hemophilia

Keywords

Gene therapy

Outcome Measures

Primary Outcomes (2)

  • Change in long term safety: adverse events of interest

    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

    Months 3, 6, 9, 12, 18, 24; and annually thereafter for 15 years.

    From baseline, through study completion (15 years)

Interventions

Gene therapyOTHER

This is a prospective patient registry.

Eligibility Criteria

Age0 Years - 100 Years
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

All people with hemophilia who have received a gene therapy product

You may qualify if:

  • People with hemophilia who have received a gene therapy product

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Hemophilia A

Interventions

Genetic Therapy

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Biological TherapyTherapeuticsGenetic EngineeringGenetic TechniquesInvestigative Techniques

Central Study Contacts

Donna Coffin, MSc

CONTACT

514.875.7944dcoffin@wfh.org

Mayss Naccache, MSc

CONTACT

514.875.7944mnaccache@wfh.org

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
100 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 20, 2020

First Posted

May 12, 2021

Study Start

January 1, 2022

Primary Completion (Estimated)

January 1, 2050

Study Completion (Estimated)

January 1, 2050

Last Updated

May 12, 2021

Record last verified: 2021-05