Fanconi Anemia, Fanconi Anemia Complementation Group A
Conditions
Keywords
gene therapy, anaemia, autologous hematopoeitic stem cell transplant, safety, bone marrow failure, genetic diseases, anemia
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.
Interventions
Long term disease and gene therapy specific safety evaluations and efficacy assessments
Sponsors
Rocket Pharmaceuticals Inc.
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
Spain
Outcome results
None listed