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

Fanconi Anemia Clinical Trial

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04437771
• Other study ID #: RP-L102-0116-LTFU
• Status: Enrolling by invitation
• Start date: June 1, 2020
• Est. completion date: January 30, 2035

Study information

• Verified date: June 2020
• Source: Rocket Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 9
• Est. completion date: January 30, 2035
• Est. primary completion date: October 30, 2034
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

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

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Anemia Complementation Group A
• Fanconi Syndrome

Intervention

Other:
• Safety and efficacy assessments
Long term disease and gene therapy specific safety evaluations and efficacy assessments

Locations

Country	Name	City	State
Spain	Hospital Infantil Universitario Niño Jesús (HIUNJ)	Madrid

Sponsors (1)

Lead Sponsor	Collaborator
Rocket Pharmaceuticals Inc.

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	Replication competent lentivirus (RCL)	Evaluate RCL in peripheral blood	15 years post-drug product infusion
Primary	Insertion site analysis in blood	Determine long term clonality	15 years post-drug product infusion
Primary	Phenotypic correction	Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents	15 years post-drug product infusion
Primary	Assessment for Malignancies	Monitor for incidence of hematologic malignancies and solid organ tumors	15 years post-drug product infusion
Primary	Hematologic stabilization	Monitor for long term stability and normalization of blood counts	15 years post-drug product infusion