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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia a
NCTID
NCT03818763
(View at clinicaltrials.gov)
Description
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.
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Development Status
Active
Indication
Hemophilia A
Disease Ontology Term
DOID:12134
Compound Name
Pleightlet
Compound Description
Auto CD34+PBSCs -889ITGA2B-BDDFVIII-WPTS(MUT6)(VSVg)
Sponsor
Medical College of Wisconsin
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
5
Results Posted
Not Available
Therapy Information
Target Gene/Variant
F8
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
Megakaryocytes
Delivery System
Viral transduction
Vector Type
VSV-G
Editor Type
none
Dose 1
Transduced CD34+ cells (not to exceed 20ml/kg body weight)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2019-01-15
Completion Date
2033-05-01
Last Update
2025-02-07
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
First patient enrolled in March 2022, only 2 patients dosed so far 8/16/24
Resources/Links
News and Press Releases
https://www.platelettargetedtherapeutics.com/
Preclinical Publications
Thromboelastometry assessment of hemostatic properties in various murine models with coagulopathy and the effect of factor VIII therapeutics
Megakaryocyte- and megakaryocyte precursor-related gene therapies
Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells
Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A
(Poster) Platelet-Targeted FVIII LV-HSC for Severe Hemophilia A Pre-Clinical Research Supporting a Clinical Protocol for a First-In-Human Trial - NHF 2021
Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII
Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies
Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity
Protocol
(Abstract #1907) First-in-Human Use of Platelet-Derived FVIII for Severe Hemophilia a with a History Inhibitors - ASGCT 2024