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Clinical Trial Report

Gene Therapy Trial Report

Summary

Lentiviral Gene Therapy for p47 AR-CGD


NCTID NCT05207657 (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 Great Ormond Street Hospital for Children NHS Foundation Trust
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 5
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 2021-06-24
Completion Date 2029-04-01
Last Update 2023-05-16

Participation Criteria


Eligible Age >=23 Months
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United Kingdom

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Updated NCTID is NCT06253507 (NIH-sponsored trial)

Resources/Links


Resources/Links

No External Links Available.