Last reviewed · How we verify
NCT04883710: WFH GTR
The World Federation of Hemophilia Gene Therapy Registry
trial testing Gene therapy in Hemophilia in 5,000 participants. Not yet recruiting.
1 January 2050
Quick facts
| Lead sponsor | World Federation of Hemophilia |
|---|---|
| Status | Not yet recruiting |
| Study type | OBSERVATIONAL |
| Enrollment | 5,000 |
| Start date | 1 January 2022 |
| Primary completion | 1 January 2050 |
| Estimated completion | 1 January 2050 |
Drugs / interventions tested
- Gene therapy — full drug profile →
Conditions studied
- Hemophilia — all drugs for Hemophilia →
Sponsor
World Federation of Hemophilia
Who can join
Adults 0 to 100, any sex, with Hemophilia. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient.
Miesbach W, Mulders G, Breederveld D, Pinachyan K, et al · · 2024 · cited 5× · PMID 38741157 · DOI 10.1186/s13023-024-03181-2 -
Getting closer to hemophilia gene therapy for all?
Reiss UM. · · 2025 · PMID 40658442 · DOI 10.1182/bloodadvances.2025016642
Verify or expand the search:
- PubMed search for NCT04883710
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Gene therapy
Trials testing the same drug.
- NCT05991336 — Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy · recruiting
- NCT04797260 — Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID · NA · recruiting
Other recruiting trials for Hemophilia
Currently open trials in the same condition.
- NCT07579585 — Effects of Photobiomodulation in Hemophilia Patients. · NA · recruiting
- NCT07406139 — PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A） · Phase 4 · recruiting
- NCT07285460 — A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Year · Phase 3 · recruiting
- NCT07187661 — Intra-Articular Bevacizumab for Preventing Recurrent Hemarthrosis in Hemophilia With Chronic Synovitis · NA · recruiting
- NCT07099313 — Association of Prophylactic Treatment With Treatment Burden and Psychosocial Variables in Patients With Hemophilia · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04883710 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by World Federation of Hemophilia
- Last refreshed: 12 May 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04883710.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing