Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A
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|ClinicalTrials.gov Identifier: NCT03818763|
Recruitment Status : Recruiting
First Posted : January 28, 2019
Last Update Posted : October 26, 2021
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A||Biological: Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open-label, nonrandomized, single-center phase I cohort study|
|Masking:||None (Open Label)|
|Official Title:||Phase I Study Evaluating Safety and Feasibility of Hematopoietic Stem Cell Gene Transfer That Targets Factor VIII Delivery From Platelets for Patients With Hemophilia A|
|Actual Study Start Date :||April 29, 2020|
|Estimated Primary Completion Date :||May 1, 2023|
|Estimated Study Completion Date :||May 1, 2033|
Experimental: Autologous CD34+PBSC transduced with a lentiviral vector
Patients will receive a patient specific (autologous) cytokine mobilized CD34+Peripheral Blood Stem Cells (PBSC) transduced ex vivo with a lentiviral vector containing cDNA encoding the human B-domain deleted FVIII protein.
Biological: Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Reduced intensity conditioning with melphalan and fludarabine, followed by a single infusion of autologous CD34+PBSC, transduced with a lentiviral vector (-889ITGA2B-BDDFVIII-WPTS(MUT6)(VSVg)) also known as (Pleightlet(MUT6)) encoding the B domain deleted from of human coagulation factor VIII (BDDFVIII) in up to five hemophilia A patients with a history of FVIII inhibitors (≥0.6BU).
The infusion volume of transduced cells will not exceed 20 ml/kg body weight.
- Number of enrolled participants with adequate gene transduced hematopoietic stem cells for FVIII gene therapy infusion [ Time Frame: Through study completion, an average of 4 years ]Assessed by availability of ≥4x106 transduced clinical grade CD34+PBSC per kg meeting release criteria for infusion; undetectable microbiological contamination and cell viability ≥70%.
- Incidence of toxicity from gene therapy [ Time Frame: Within 3 months of gene therapy infusion ]Number of events meeting CTCAE criteria grade 3 or 4 toxicity
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): NCT03818763
|Contact: Mary Eapen, MDemail@example.com|
|Contact: Arielle Baimfirstname.lastname@example.org|
|Principal Investigator:||Mary Eapen, MD||Froedtert Hosptial and Medical College of Wisconsin|