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

Plain-language summary

This study provides long-term safety and efficacy follow-up of patients with Fanconi anaemia subtype A who were previously treated with ex vivo gene therapy in the FANCOLEN-I study. Patients who received gene therapy in the FANCOLEN-I study are eligible. It is a non-phase follow-up study and is active but not currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Rocket Pharmaceuticals Inc.

Intervention

Safety and efficacy assessments

Start date

6/1/2020

Estimated completion

1/17/2034

Countries

Spain

Related genes

Locations

• Madrid, Spain

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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