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NCT04437771
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
trial testing Safety and efficacy assessments in Fanconi Anemia Complementation Group A in 9 participants. Participants enrolled and being followed up; not accepting new ones.
17 January 2034
Quick facts
| Lead sponsor | Rocket Pharmaceuticals Inc. |
|---|---|
| Status | Active, enrolled |
| Study type | OBSERVATIONAL |
| Enrollment | 9 |
| Start date | 1 June 2020 |
| Primary completion | 17 January 2034 |
| Estimated completion | 17 January 2034 |
| Sites | 1 location across Spain |
Drugs / interventions tested
- Safety and efficacy assessments — full drug profile →
Conditions studied
- Fanconi Anemia Complementation Group A — all drugs for Fanconi Anemia Complementation Group A →
- Fanconi Anemia — all drugs for Fanconi Anemia →
Sponsor
Rocket Pharmaceuticals Inc. — full company profile →
Who can join
Eligibility, any sex, with Fanconi Anemia Complementation Group A or Fanconi Anemia. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. No investigational drug product will be administered during this study.
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
-
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Río P, Zubicaray J, Navarro S, Gálvez E, et al · · 2025 · cited 19× · PMID 39642902 · DOI 10.1016/s0140-6736(24)01880-4 -
Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.
Lederer CW, Koniali L, Buerki-Thurnherr T, Papasavva PL, et al · · 2022 · cited 5× · PMID 35456627 · DOI 10.3390/pharmaceutics14040793 -
Towards a Cure for Diamond-Blackfan Anemia: Views on Gene Therapy.
Vale M, Prochazka J, Sedlacek R. · · 2024 · cited 2× · PMID 38891052 · DOI 10.3390/cells13110920
Verify or expand the search:
- PubMed search for NCT04437771
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Safety and efficacy assessments
Trials testing the same drug.
- NCT04836377 — A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02 · terminated
- NCT04008849 — A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplanta · terminated
- NCT02786511 — Longterm Follow-up of Subjects Treated With bb2121 · completed
Other recruiting trials for Fanconi Anemia Complementation Group A
Currently open trials in the same condition.
- NCT04248439 — Gene Therapy for Fanconi Anemia, Complementation Group A · Phase 2 · active not recruiting
Other Rocket Pharmaceuticals Inc. trials
Trials by the same sponsor.
- NCT05548855 — Natural History of Danon Disease · completed
- NCT06282432 — Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) · active not recruiting
- NCT04525352 — A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis · Phase 1 · terminated
- NCT04248439 — Gene Therapy for Fanconi Anemia, Complementation Group A · Phase 2 · active not recruiting
- NCT04105166 — Gene Therapy for Pyruvate Kinase Deficiency (PKD) · Phase 1 · completed
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 NCT04437771 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 9 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 Rocket Pharmaceuticals Inc.
- Last refreshed: 13 April 2026
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/NCT04437771.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing