A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

NCTID	NCT03812263 (View at clinicaltrials.gov)
Description	The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201. The primary objectives of the Phase II portion of the study are evaluation of survival, as determined by the proportion of subjects alive at age 2 (24 months) and at least 1-year post-infusion without allogeneic hematopoietic stem cell transplant (HSCT) and characterization of the safety and toxicity associated with the infusion. (Show More)
Indication	Leukocyte Adhesion Defect - Type I
Compound Name	KRESLADI (mametegragene autotemcel) RP-L201
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Status	Completed
Enrollment Count	9

Target Gene/Variant	ITGB2
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	CD34+ cells
Delivery System	Viral transduction
Vector Type	Chim.hCD18-LV
Editor Type
Dose 1	Dose range: 2.8-10 x 10^6 transduced CD34+ cells/kg
Dose 2	Min dose: 2.0 x 10^6 CD34+ cells/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,United Kingdom,Spain

Has US IND	True
Recent Updates	BLA Submitted: Pending FDA (????)