Clinical Trials in Spain / NCT04437771
Active, not recruiting
Observational
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
NCT04437771 · tracked via the Priya Life Science Spain tracker
Condition(s) studied
Fanconi Anemia Complementation Group AFanconi Anemia
Investigational drug(s) / intervention(s)
Safety and efficacy assessments
Safety and efficacy assessments: Long term disease and gene therapy specific safety evaluations and efficacy assessments
Study 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.
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
Primary outcome measure(s)
- 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
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Hospital Infantil Universitario Niño Jesús (HIUNJ) | Madrid | Spain |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04437771 on ClinicalTrials.gov ↗ ← All trials in Spain