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A Study of Prime Editing (PM359) for p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD)

This study is testing a new gene editing treatment called PM359 for people with a specific type of Chronic Granulomatous Disease (CGD). CGD is a rare genetic disease that affects white blood cells, making it harder for your body to fight infections. This study focuses on CGD caused by a specific genetic change (delGT mutation in the NCF1 gene). PM359 works by taking your own blood stem cells, correcting the genetic error using "Prime Editing" technology, and then giving them back to you. Researchers want to see if PM359 is safe and if it helps your white blood cells work better. Success means fewer side effects and improved immune cell function. The study is currently enrolling a small number of participants, starting with adults, then adolescents, and finally children aged 6-11.

Study design
This is an open-label, single-arm study, meaning all 12 participants will receive the PM359 treatment, and everyone involved will know what treatment is being given.
What's involved
You would have your own cells collected, modified, and then infused back into you after a preparation procedure. You would also participate in a long-term follow-up study for a total of 15 years.
Compensation
Not stated in the trial record.
Follow-up
Your safety will be monitored for 12 months after the PM359 infusion. The activity of your immune cells will be checked at 6 and 12 months after the infusion.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

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+InterventionalTreatment
Prime 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

Chronic Granulomatous Disease
Granulomatous Disease, Chronic

Explore related trials

Autosomal Recessive Chronic Granulomatous DiseaseNCF1 GeneNeutrophil Cytosol Factor 1

NCT06559176

Where you'd take part

This study runs at 5 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • CHU - Sainte Justine Hospital

    Montreal, Quebec, Canadano site contact published

  • NIH Clinical Center

    Bethesda, Marylandno site contact published

  • The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers

    Nashville, Tennesseeno site contact published

  • University College of London Hospital

    London, England, United Kingdomno site contact published

  • University of California Los Angeles Medical Center

    Los Angeles, Californiano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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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