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

Rocket Pharmaceuticals

Status: Invitation-only

Conditions: Fanconi Anemia; Fanconi Anemia Complementation Group A

Treatments: Other: Safety and efficacy assessments

Study type: Observational

Funder types: Industry

Identifiers

NCT04437771

RP-L102-0116-LTFU

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

9 participants in 1 patient group

Subjects with Fanconi Anaemia Subtype A (FA-A)

Description:

Subjects treated with ex vivo lentiviral gene therapy product in FANCOLEN-I trial and agree to participate in this long-term follow-up (LTFU) study

Treatment:

Other: Safety and efficacy assessments