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

N/AEnrolling By InvitationNCT04437771

Rocket Pharmaceuticals Inc.9 enrolled

Overview

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.

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 Type

OBSERVATIONAL

Enrollment

Conditions

Fanconi Anemia Complementation Group A Fanconi Anemia

Interventions

Safety and efficacy assessmentsOTHER

Long term disease and gene therapy specific safety evaluations and efficacy assessments

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: 1. Enrolled in the FANCOLEN-I study 2. Treated with gene therapy in the FANCOLEN-I study 3. Able to adhere to the study visit schedule and protocol requirements 4. Provided written informed consent and, as applicable, assent to participate Exclusion Criteria: * There are no exclusion criteria for this study

Locations (1)

Hospital Infantil Universitario Niño Jesús (HIUNJ)

Madrid, Spain

Outcomes

Primary Outcomes

Monitor long term safety of patients through blood laboratory evaluations and general health status

Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV)