NCT04069533

A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype A

Status unknown Phase 2 Last updated 20 May 2024

What this trial tests

Phase 2 trial testing RP-L102 in Fanconi Anemia Complementation Group A in 7 participants. Status unknown.

Timeline

28 November 2019

Primary endpoint
1 February 2025

1 February 2025

Quick facts

Lead sponsor	Rocket Pharmaceuticals Inc.
Phase	Phase 2
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	7
Start date	28 November 2019
Primary completion	1 February 2025
Estimated completion	1 February 2025
Sites	2 locations across Spain, United Kingdom

Drugs / interventions tested

• RP-L102 — full drug profile →

Conditions studied

• Fanconi Anemia Complementation Group A — all drugs for Fanconi Anemia Complementation Group A →

Sponsor

Rocket Pharmaceuticals Inc. — full company profile →

Who can join

Adults 1 to 17, any sex, with Fanconi Anemia Complementation Group A. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Phenotypic correction of bone marrow colony forming units after infusion of RP-L102
  Time frame: 3 years
  During months 12-36 post-infusion, the survival of bone marrow colony forming units to 10nM mitomycin C (MMC) increases to over or equal to 10% with respect to values determined at baseline (pretreatment evaluation).

Sponsor's own description

This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).

Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges.
   Martínez-Molina E, Chocarro-Wrona C, Martínez-Moreno D, Marchal JA, et al · · 2020 · cited 55× · PMID 33153183 · DOI 10.3390/pharmaceutics12111051
2. Immunological barriers to haematopoietic stem cell gene therapy.
   Charlesworth CT, Hsu I, Wilkinson AC, Nakauchi H. · · 2022 · cited 47× · PMID 35301483 · DOI 10.1038/s41577-022-00698-0
3. Fanconi anemia and the underlying causes of genomic instability.
   Rageul J, Kim H. · · 2020 · cited 33× · PMID 31983075 · DOI 10.1002/em.22358
4. Clinical hematopoietic stem cell-based gene therapy.
   John T, Czechowicz A. · · 2025 · cited 6× · PMID 40285354 · DOI 10.1016/j.ymthe.2025.04.029
5. 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
6. Pediatric Bone Marrow Failure: A Broad Landscape in Need of Personalized Management
   Vissers L, van der Burg M, Lankester A, Smiers F, et al · · 2023