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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT04437771
Other study ID # RP-L102-0116-LTFU
Secondary ID
Status Enrolling by invitation
Phase
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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