NCT04437771

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

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 9 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Fanconi Anemia Complementation Group A
• Fanconi Anemia

Interventions

Type	Name	Description
OTHER	Safety and efficacy assessments	Long term disease and gene therapy specific safety evaluations and efficacy assessments

Timeline

• Start date: 2020-06-01
• Primary completion: 2034-01-17
• Completion: 2034-01-17
• First posted: 2020-06-18
• Last updated: 2026-04-13

Locations

1 site across 1 country: Spain