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Gene Therapy Trial Browser
Clinical Trial Report
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
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
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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
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
SEC Form 10-K: Rocket Pharmaceuticals, Inc. FY2022
Preclinical Publications
Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis
Lentiviral gene transfer of TCIRG1 into peripheral blood CD34(+) cells restores osteoclast function in infantile malignant osteopetrosis
Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice
Regulation and Function of Lentiviral Vector-Mediated TCIRG1 Expression in Osteoclasts from Patients with Infantile Malignant Osteopetrosis: Implications for Gene Therapy
(Review) Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal