A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

Part of paid clinical trials in Los Angeles, California.

Sponsor
Prime Medicine, Inc.
Study ID
NCT06559176
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • Chronic Granulomatous Disease
  • Granulomatous Disease, Chronic

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • PM359 — BIOLOGICAL
    Single dose of PM359 administered autologously by intravenous (I.V.) infusion following myeloablative conditioning with busulfan

Study Details

This is an open-label, single-arm, multicenter Phase 1/2 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.

Key Dates

Start date
Oct 17, 2024
Status verified
Mar 2026
Primary completion
Jan 31, 2030
Completion
Feb 28, 2030

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: PM359
    PM359 is an autologous CD34+ hematopoietic stem cell (HSC) suspension that is Prime Edited at the NCF1 locus resulting in expression of the p47phox protein.

Primary Outcome Measure

Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusion [ Time Frame: PM359 infusion through Month 12 after PM359 infusion ]

Locations (3)

FacilityCityStateZIPSite coordinators
University of California Los Angeles Medical CenterLos AngelesCalifornia90027-
NIH Clinical CenterBethesdaMaryland20892-
The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood CancersNashvilleTennessee37203-

Find similar trials in Los Angeles, CA

By research site
University of California Los Angeles Medical Center· Los Angeles, CANIH Clinical Center· Bethesda, MDThe Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers· Nashville, TN

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