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Clinical Trial Report

Gene Therapy Trial Report

Summary

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
Development Status Active
Indication Leukocyte Adhesion Defect - Type I
Disease Ontology Term DOID:0110910
Compound Name KRESLADI
Compound Alias mametegragene autotemcel, RP-L201
Sponsor Rocket Pharmaceuticals Inc.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 9 (ACTUAL)
Results Posted Not Available

Therapy Information


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 none
Dose 1 Dose range: 2.8-10E6 transduced CD34+ cells/kg
Dose 2 Median dose: 4.3E6 transduced CD34+ cells/kg (n=9)
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2019-01-18
Completion Date 2023-09-12
Last Update 2023-11-15

Participation Criteria


Eligible Age >=3 Months
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


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

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates FDA review of limited additional CMC information ongoing for KRESLADI; PDUFA date 3/28/26

Resources/Links