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Clinical Trial Report

Gene Therapy Trial Report

Summary

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)


NCTID NCT06253507 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Autosomal Recessive Chronic Granulomatous Disease (CGD)
Disease Ontology Term DOID:0070192
Compound Name PCHIM-p47
Sponsor National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type NIH
Recruitment Status
Enrolling by invitation
Enrollment Count 10
Results Posted Not Available

Therapy Information


Target Gene/Variant NCF1
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, minimum dose: 3E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-02-09
Completion Date 2027-03-31
Last Update 2025-04-10

Participation Criteria


Eligible Age 3 Years - 65 Years
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
Recent Updates

Resources/Links