Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
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|ClinicalTrials.gov Identifier: NCT04069533|
Recruitment Status : Recruiting
First Posted : August 28, 2019
Last Update Posted : October 20, 2020
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.
|Condition or disease||Intervention/treatment||Phase|
|Fanconi Anemia Complementation Group A||Biological: RP-L102||Phase 2|
This is a pediatric open-label Phase II clinical trial to assess the efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in pediatric subjects with FA-A.
Enriched CD34+ hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype A|
|Actual Study Start Date :||November 28, 2019|
|Estimated Primary Completion Date :||January 2023|
|Estimated Study Completion Date :||January 2023|
RP-L102 is CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with lentiviral vector carrying the FANCA gene
CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with lentiviral vector carrying the FANCA gene
- Phenotypic correction of bone marrow colony forming units after infusion of RP-L102 [ Time Frame: 3 years ]During months 12-36 post-infusion, the survival of bone marrow colony forming units to 10nM mitomycin C (MMC) increases to over or equal to 10% with respect to values determined at baseline (pretreatment evaluation).
- Phenotypic correction of T-lymphocytes in peripheral blood after infusion of RP-L102 [ Time Frame: 3 years ]Assessment of the percentage of peripheral blood T-cells with diepoxybutane (DEB)-induced chromosomal aberrations that decreases from over or equal to 50% at baseline (defined as the interval between the pre-treatment evaluation and 2 months post-infusion) to less than 50% during the interval between 12 and 36 months post-infusion.
- Engraftment of gene-corrected hematopoietic cells after infusion of RP-L102 [ Time Frame: 3 years ]The level of gene marking of the FANCA-lentiviral vector (LV) provirus in total peripheral blood cells is at least 0.1 vector copy number (VCN) in peripheral blood cells during months 6-36 post-infusion.
- Prevention or rescue of bone marrow failure after infusion of RP-L102 [ Time Frame: 3 years ]Assessment of the need for treatment of bone marrow failure 6-36 months post-infusion. During the 3rd year post-infusion, peripheral blood parameters: hemoglobin levels, neutrophils, and platelets will be assessed and considered stable if they remain at over or equal to 80% of values determined at pre-treatment evaluation visit or immediately prior to mobilization before the administration of granulocyte-colony stimulating factor.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04069533
|Contact: Fanconi Anemia Clinical Trial||+1 646-627-0033||FAclinicaltrial@rocketpharma.com|
|Contact: Fanconi Anemia Clinical Trial||FAclinicaltrial@rocketpharma.com|
|Hospital Infantil Universitario Niño Jesús (HIUNJ)||Recruiting|
|Madrid, Spain, 28009|
|Principal Investigator:||Julián Sevilla Navarro, MD, PhD||Hospital Infantil Universitario Niño Jesús (HIUNJ)|