FANCOLEN-II
Research type
Research Study
Full title
A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced witha Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A
IRAS ID
271745
Contact name
Jonathan D. Schwartz
Contact email
Sponsor organisation
Rocket Pharmaceuticals Inc
Eudract number
2018-002502-31
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
n/a, n/a
Duration of Study in the UK
3 years, 4 months, 13 days
Research summary
Participants in this study have Fanconi anaemia (FA), a rare genetic disorder that affects the ability of the cells to repair damage to genetic material (DNA). In the majority of patients, FA leads to underproduction of the blood cells from the bone marrow (material inside the bones that make the blood cells) and eventually to bone marrow failure in the first decade of life. Currently, the only potentially curative treatment for FA is bone marrow transplant (BMT) from a compatible healthy donor, which may not be available for all patients. This procedure is associated with risk of infection or graft-versus-host disease where the donor cells may attack the patient's tissues. Also cells of FA patients do not have the ability to repair damage and are more susceptible to harm from chemotherapy, radiation and other medicines used during BMT. In this study, blood stem cells are taken from the participant, a copy of the correct gene is inserted, (this is called gene therapy) with the intent of enabling them to work correctly, and the cells are transfused back into the participant. It is hoped that corrected stem cells will improve bone marrow health and blood cell counts and eventually, protect against or decrease the chances of suffering bone marrow failure. The study will also determine if the procedure is safe. Gene therapy has been used before to treat children with FA in previous studies, but with a different virus used to introduce the good gene in stem cells. The current study will use a virus called a lentivirus, believed to be safer than viruses used in previous studies. This therapy has been given to 11 paediatric patients with long-term follow-up available for 4 of them. To date, the safety profile has been very favourable.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1778
Date of REC Opinion
7 Feb 2020
REC opinion
Further Information Favourable Opinion