Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06559176
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-07-11
Brief Title:
A Study of the Safety and Efficacy of Prime Editing PM359 in Participants with P47phox Autosomal Recessive Chronic Granulomatous Disease CGD
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Phase 12 Study Evaluating Gene Therapy by Transplantation of Autologous CD34 Stem Cells Modified Ex Vivo Using Prime Editing PM359 in Participants with Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is an open-label single-arm multicenter Phase 12 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34 stem cells modified ex vivo PM359 in participants with autosomal recessive Chronic Granulomatous Disease CGD caused by mutations in the NCF1 Neutrophil Cytosolic Factor 1 gene
Detailed Description:
Chronic Granulomatous Disease CGD is a rare genetic disease affecting the white blood cells leading to failure of innate immunity against a variety of human pathogens and is also associated with autoimmune and inflammatory conditions Approximately 20-25 of people with CGD inherit a mutation commonly known as delGT in both copies of the NCF1 gene which encodes the p47phox protein
This study seeks to understand the safety and efficacy of a new gene editing technology known as Prime Editing in participants with autosomal recessive CGD caused by the delGT mutation in NCF1 Autologous CD34 cells are collected from the participant via mobilization and apheresis shipped to a central manufacturing facility and modified using Prime Editing to correct the delGT mutation causing p47phox CGD After manufacture the Prime Edited stem cells PM359 will be shipped to the study site where they will be infused back into the participant following a preparative procedure known as conditioning
The study will initially enroll adult participants aged 18 and plans to then move into adolescents aged 12 - 17 followed by children aged 6 - 11
This study seeks to understand the safety and efficacy of a new gene editing technology known as Prime Editing in participants with autosomal recessive CGD caused by the delGT mutation in NCF1 Autologous CD34 cells are collected from the participant via mobilization and apheresis shipped to a central manufacturing facility and modified using Prime Editing to correct the delGT mutation causing p47phox CGD After manufacture the Prime Edited stem cells PM359 will be shipped to the study site where they will be infused back into the participant following a preparative procedure known as conditioning
The study will initially enroll adult participants aged 18 and plans to then move into adolescents aged 12 - 17 followed by children aged 6 - 11
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None