Gene Therapy for Fanconi Anemia, Complementation Group A
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|ClinicalTrials.gov Identifier: NCT04248439|
Recruitment Status : Recruiting
First Posted : January 30, 2020
Last Update Posted : November 24, 2020
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for 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 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 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A|
|Actual Study Start Date :||July 15, 2020|
|Estimated Primary Completion Date :||June 2022|
|Estimated Study Completion Date :||June 2023|
RP-L102 is CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene
CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a 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
- 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 12-36 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.
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): NCT04248439
|Contact: Clinical Information||646-627-0033||FAclinicaltrial@rocketpharma.com|
|United States, California|
|Stanford, California, United States, 94304|
|Contact: Elisabeth Merkel firstname.lastname@example.org|
|United States, Minnesota|
|University of Minnesota||Active, not recruiting|
|Minneapolis, Minnesota, United States, 55454|
|Principal Investigator:||Agnieszka Czechowicz, MD||Stanford University|
|Principal Investigator:||Margaret MacMillan, MD, MSc||University of Minnesota|