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

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: Enrolling by invitation
• Conditions: Fanconi Anemia; Fanconi Anemia Complementation Group A
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Anticipated): 9

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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

How is the study designed?

Design Details

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

Number of groups / cohorts

1

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	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	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)	Evaluate RCL in peripheral blood	15 years post-drug product infusion
Insertion site analysis in blood	Determine long term clonality	15 years post-drug product infusion
Phenotypic correction	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	Monitor for incidence of hematologic malignancies and solid organ tumors	15 years post-drug product infusion
Hematologic stabilization	Monitor for long term stability and normalization of blood counts	15 years post-drug product infusion

Collaborators and Investigators

• Sponsor: Rocket Pharmaceuticals Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2020
• Primary Completion (Anticipated): October 30, 2034
• Study Completion (Anticipated): January 30, 2035

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): June 22, 2020
• Last Update Submitted That Met QC Criteria: June 18, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Keywords: safety; anemia; gene therapy; anaemia; autologous hematopoeitic stem cell transplant; bone marrow failure; genetic diseases
• Additional Relevant MeSH Terms: Metabolic Diseases; Kidney Diseases; Urologic Diseases; Bone Marrow Diseases; Hematologic Diseases; Genetic Diseases, Inborn; DNA Repair-Deficiency Disorders; Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Renal Tubular Transport, Inborn Errors; Anemia; Fanconi Syndrome; Fanconi Anemia
• 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