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Gene Therapy Trial Report

Summary

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

NCTID NCT04525352 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Infantile Malignant Osteopetrosis
Disease Ontology Term DOID:0110942
Compound Name RP-L401
Compound Description EFS.hTCIRG1-LV
Sponsor Rocket Pharmaceuticals Inc.
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 1 (ACTUAL)
Results Posted Not Available

Therapy Information

Target Gene/Variant TCIRG1
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 LV
Editor Type none
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1
Submit Date 2020-08-11
Completion Date 2021-05-21
Last Update 2022-07-13

Participation Criteria

Eligible Age >=1 Month
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations United States

Regulatory Information

Has US IND True
FDA Designations Fast Track
Recent Updates Study terminated due to feasibility in December 2021, program returned to academic innovators

Resources/Links

News and Press Releases
Preclinical Publications