Status:
ENROLLING_BY_INVITATION
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
Lead Sponsor:
Rocket Pharmaceuticals Inc.
Conditions:
Fanconi Anemia Complementation Group A
Fanconi Anemia
Eligibility:
All Genders
Brief 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 FAN...
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.
Eligibility Criteria
Inclusion
- Enrolled in the FANCOLEN-I study
- Treated with gene therapy in the FANCOLEN-I study
- Able to adhere to the study visit schedule and protocol requirements
- Provided written informed consent and, as applicable, assent to participate
Exclusion
- There are no exclusion criteria for this study
Key Trial Info
Start Date :
June 1 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 30 2035
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT04437771
Start Date
June 1 2020
End Date
January 30 2035
Last Update
June 22 2020
Active Locations (1)
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1
Hospital Infantil Universitario Niño Jesús (HIUNJ)
Madrid, Spain, 28009