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

April 7, 2026 updated by: Rocket Pharmaceuticals Inc.

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

9

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Spain
      • Madrid, Spain, 28009
        • Hospital Infantil Universitario Niño Jesús (HIUNJ)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Subjects with Fanconi anemia subtype A who have been treated with ex vivo gene therapy product in FANCOLEN-I study

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Subjects with Fanconi Anaemia Subtype A (FA-A)
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
Other: Safety and efficacy assessments
Long term disease and gene therapy specific safety evaluations and efficacy assessments

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Monitor long term safety of patients through blood laboratory evaluations and general health status
Time Frame: 15 years post-drug product infusion
Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV)
15 years post-drug product infusion
Long term genetic correction assessed in bone marrow and blood
Time Frame: 15 years post-drug product infusion
Determine long term persistence of therapeutic LV in hematopoietic cells in bone marrow and blood
15 years post-drug product infusion
Replication competent lentivirus (RCL)
Time Frame: 15 years post-drug product infusion
Evaluate RCL in peripheral blood
15 years post-drug product infusion
Insertion site analysis in blood
Time Frame: 15 years post-drug product infusion
Determine long term clonality
15 years post-drug product infusion
Phenotypic correction
Time Frame: 15 years post-drug product infusion
Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents
15 years post-drug product infusion
Assessment for Malignancies
Time Frame: 15 years post-drug product infusion
Monitor for incidence of hematologic malignancies and solid organ tumors
15 years post-drug product infusion
Hematologic stabilization
Time Frame: 15 years post-drug product infusion
Monitor for long term stability and normalization of blood counts
15 years post-drug product infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rocket Pharmaceuticals Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 1, 2020

Primary Completion (Estimated)

January 17, 2034

Study Completion (Estimated)

January 17, 2034

Study Registration Dates

First Submitted

June 15, 2020

First Submitted That Met QC Criteria

June 16, 2020

First Posted (Actual)

June 18, 2020

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 7, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RP-L102-0116-LTFU

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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