SNPMiner Trials by Shray Alag

SNPMiner SNPMiner Trials (Home Page)

Report for Clinical Trial NCT03818763

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

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

This is a Phase I study. This research study is being conducted to find new ways to treat severe hemophilia A. This study is a gene therapy study. Gene therapy is an experimental way to introduce, into a person's cells, specific genetic material. A gene can be delivered/introduced into a cell using a carrier known as a "vector." In this study, a virus (lentivirus), the "vector", is used to introduce or deliver a gene that creates and stores a protein Factor VIII (FVIII) in your platelets. These platelets are made from stem cells (mother cells for your bone marrow) that are removed from your blood by a procedure called apheresis. This research study will take some of the patient's own stem cells, from the apheresis procedure, and genetically modify them using the vector in order to make them produce FVIII in platelets that arise from the stem cells. They will then give the genetically modified stem cells back to the patient so that they can possibly create platelets that produce and store Factor VIII on their own.

NCT03818763 Hemophilia A
MeSH:Hemophilia A
HPO:Reduced factor VIII activity

1 Interventions

Name: Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII

Description: 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 inhibitors to FVIII The infusion volume of transduced cells will not exceed 20 ml/kg body weight.
Type: Biological
Group Labels: 1

Autologous CD34+PBSC transduced with a lentiviral vector

Primary Outcomes

Description: Assessed by availability of ≥4x106 transduced clinical grade CD34+PBSC per kg meeting release criteria for infusion; undetectable microbiological contamination and cell viability ≥70%.

Measure: Number of enrolled participants with adequate gene transduced hematopoietic stem cells for FVIII gene therapy infusion

Time: Through study completion, an average of 4 years

Secondary Outcomes

Description: Number of events meeting CTCAE criteria grade 3 or 4 toxicity

Measure: Incidence of toxicity from gene therapy

Time: Within 3 months of gene therapy infusion

Purpose: Treatment

Allocation: N/A

Single Group Assignment

There is one SNP


1 G20210A

Those with a known co-existing clinically significant thrombophilic disorder, or as determined by the presence of any of the below identified on screening laboratory assessments: - FV Leiden - Protein S deficiency - Protein C deficiency - Prothrombin mutation (G20210A) - D-dimer >3 x the upper limit of normal (ULN) at Screening All known patients with the above and any patient with a personal or significant family history of thrombotic events (DVT, PE, arterial clots) as deemed by the principal investigator will be screened for the above disorders. --- G20210A ---

HPO Nodes