Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

Phase 1

Recruiting

• Conditions: Chronic Granulomatous Disease (CGD); X-Linked Chronic Granulomatous Disease
• Interventions: Biological: Base-edited hematopoietic stem and progenitor cells; Drug: Busulfan; Drug: Palifermin; Drug: Filgrastim; Drug: Plerixafor

• Registration Number: NCT06325709
• Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Brief Summary

Background:
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Detailed Description

Study Description:
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Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2024-03-21
• Study First Submitted QC: 2024-03-21
• Study First Posted: 2024-03-22
• Study Start: 2024-04-17
• Status Verified: 2024-10-01
• Last Update Submitted: 2024-12-18
• Last Update Posted: 2024-12-19
• Primary Completion: 2032-12-31
• Study Completion: 2032-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 10

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single Arm Study	Base-edited hematopoietic stem and progenitor cells	-
Single Arm Study	Plerixafor	-
Single Arm Study	Busulfan	-
Single Arm Study	Filgrastim	-
Single Arm Study	Palifermin	-

Primary Outcome Measures

Name	Time	Method
To evaluate the efficacy of base-edited autologous CD34+ cells	Assessed 12 months post-infusion of base-edited cells	Efficacy of gene therapy as determined by percentages ofparticipants who have \>= 10 percent oxidase-positive granulocytes
To evaluate the safety of base-edited autologous CD34+ cells	Initiated from the time of the infusion of base-edited cells through 2 years post-infusion	Safety of gene therapy using base-edited autologous hematopoietic stem and progenitor cells as measured by study agent related adverse events and serious adverse events

Secondary Outcome Measures

Name	Time	Method
Evaluate efficacy in restoring NADPH oxidase function.	Assessed 12-24 months post-infusion of base-edited cells	Measure the frequency of DHR+ cells
Evaluate the efficiency of base-editing.	Assessed 12-24 months post-infusion of base-edited cells	Measure the percentages of gp91-expressing cells
Evaluate clinical efficacy	Assessed through study completion	Assess the frequency of infections and progression of co-morbidities of CGD
Evaluate the engraftment capability of base-edited hematopoietic stem progenitor cells.	Assessed 12-24 months post-infusion of base-edited cells	Measure the percentages of edited myeloid cells
Evaluate the efficiency in restoring gp91phox expression.	Assessed 12-24 months post-infusion of base-edited cells	Measure the frequency of gp91phox+ cells and the amount of gp91phox protein
Evaluate the stability of gene correction	Assessed through study completion	Compare frequencies of corrected alleles before infusion and at study completion

Trial Locations

Locations (1)

National Institutes of Health Clinical Center; 🇺🇸 Bethesda, Maryland, United States