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Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Fanconi Anemia Clinical Trial

Official title:
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Patients With Fanconi Anaemia Subtype A: FANCOLEN-I

Clinical Trial Summary

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.

Clinical Trial Description

This long-term follow-up protocol will evaluate the long term safety and efficacy of the infusion of autologous CD34+ cells transduced with lentiviral vector (LV) carrying the FANCA gene. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04437771
Study type Observational
Source Rocket Pharmaceuticals Inc.
Contact
Status Enrolling by invitation
Phase
Start date June 1, 2020
Completion date January 30, 2035
View Details
See also
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