← Back to Search

Gene Therapy

PM359 for Chronic Granulomatous Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Prime Medicine, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
* Autosomal recessive Chronic Granulomatous Disease due to the delGT mutation in NCF1 causing dysfunction of p47phox
* Treated and followed for at least the past 2 years in a specialized center
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from pm359 infusion, assessed at 1 year and 3 years post-pm359 infusion
Awards & highlights

Summary

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.

Who is the study for?
This trial is for individuals with autosomal recessive Chronic Granulomatous Disease (CGD) due to NCF1 gene mutations. Specific eligibility criteria are not provided, but typically include factors like age, disease severity, and overall health status.
What is being tested?
The study tests a new gene therapy called PM359. It involves editing the genes of a patient's own stem cells and then transplanting them back into the body to treat CGD.
What are the potential side effects?
Potential side effects are not detailed here, but gene therapy can sometimes cause immune reactions, bleeding or infection from the stem cell collection/transplantation process, and possible off-target genetic effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from pm359 infusion, assessed at 1 year and 3 years post-pm359 infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and from pm359 infusion, assessed at 1 year and 3 years post-pm359 infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Percentage of participants with sustained reconstitution of NADPH oxidase activity in neutrophils
Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusion
Secondary study objectives
Durability of multi-lineage hematopoietic cell reconstitution
Frequency of all drug product-related AEs, ≥ Grade 3 AEs, and serious adverse events (SAEs)
Frequency of any new or worsening moderate or greater CGD associated infection requiring anti-microbial therapy with confirmed microbiology demonstrating bacterial or fungal origin consistent with CGD-related pathology, as compared to baseline
+12 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: PM359Experimental Treatment1 Intervention
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.

Find a Location

Who is running the clinical trial?

Prime Medicine, Inc.Lead Sponsor
~8 spots leftby Sep 2029
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top