NCT06559176
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
Enrolling by Invitation
PHASE1Ages 6+InterventionalTreatmentPrime Medicine, Inc.
~12 participants
Updated 2026-04-06 on ClinicalTrials.gov
What's tested:PM359
At a glance
Recruiting sites
0 of 5 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusion
Measured over PM359 infusion through Month 12 after PM359 infusion
+1 more outcome measured
Conditions
Where it's being run
5 sites across 5 statesCalifornia1
Maryland1
Tennessee1
Quebec1
England1
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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
Willingness to participate in this study as well as a long-term follow-up study with the understanding that the total participation is 15 years
At least 1 prior severe CGD-related infection OR an ongoing severe CGD-related infection requiring therapy or that is refractory to standard therapy; OR an autoimmune or inflammatory condition related to CGD that is active or requiring therapy to maintain remission.
Exclusion
For participants younger than 16 years of age: known, willing, and available 10/10 (A,B,C,DR,DQ) HLA-matched related donor (10/10 MRD)
Active bacteremia or fungemia
Ongoing inflammatory condition that is ≥ CTCAE v5.0 Grade 3 despite high-dose steroids (≥ 0.5 mg/kg/day of prednisone and/or equivalent).
Any contraindication which in the opinion of the transplant physician would make the participant ineligible to undergo autologous HSCT, including, but not limited to:
Positive for presence of human immunodeficiency virus (HIV)-1 or HIV-2, or active infection with hepatitis B virus (HBV), or hepatitis C virus (HCV).
Inadequate organ function, including known chronic advanced end-organ damage which in the opinion of the investigator would put the participant at risk for undergoing HSCT
Prior or current malignancy or myeloproliferative disorder (excluding Stage 1 or lower, fully treated/excised malignant and pre-malignant disease of the skin, cervix or colon).
Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful study completion, including Participant/Parent/Guardian unable or unwilling to comply with the protocol requirements.
Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile participants. Females of childbearing potential and non-sterile male participants who are or may become sexually active with female partners of childbearing potential are required to use highly effective contraception from Screening through at least 12 months after drug product infusion.
Participation in another clinical study with an investigational drug within 30 days of Screening or at least 5 times the half-life of the investigational drug (whichever is longer), or any prior receipt of gene therapy or hematopoietic stem cell transplant.
What this trial measures
- Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusionPM359 infusion through Month 12 after PM359 infusion
- Percentage of participants with sustained reconstitution of NADPH oxidase activity in neutrophilsAt Month 6 and Month 12 after PM359 infusion, as compared to baseline