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

Sponsor

Rocket Pharmaceuticals Inc. (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT04437771

Collaborator

(none)

Enrollment

Location

176

Anticipated Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Fanconi Anemia Complementation Group A Fanconi Anemia	Other: Safety and efficacy assessments

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 Design

Study Type:

Observational

Anticipated Enrollment :

9 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Jun 1, 2020

Anticipated Primary Completion Date :

Oct 30, 2034

Anticipated Study Completion Date :

Jan 30, 2035

Arms and Interventions

Arm	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

Arm

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

Outcome Measures

Primary Outcome Measures

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
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
Assessment for Malignancies [15 years post-drug product infusion]
Monitor for incidence of hematologic malignancies and solid organ tumors
Hematologic stabilization [15 years post-drug product infusion]
Monitor for long term stability and normalization of blood counts