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

• Conditions: Fanconi Anemia Complementation Group A; Fanconi Anemia
• Interventions: Other: Safety and efficacy assessments

• Registration Number: NCT04437771
• Lead Sponsor: Rocket Pharmaceuticals Inc.

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 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.

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 Timeline

• Status Verified: 2020-06
• Study Start: 2020-06-01
• Study First Submitted: 2020-06-15
• Study First Submitted QC: 2020-06-16
• Study First Posted: 2020-06-18
• Last Update Submitted: 2020-06-18
• Last Update Posted: 2020-06-22
• Primary Completion: 2034-10-30
• Study Completion: 2035-01-30

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 9

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

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Exclusion Criteria

• There are no exclusion criteria for this study

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Subjects with Fanconi Anaemia Subtype A (FA-A)	Safety and efficacy assessments	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

Primary Outcome Measures

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

Trial Locations

Locations (1)

Hospital Infantil Universitario Niño Jesús (HIUNJ); 🇪🇸 Madrid, Spain