Contact SCGE




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
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