Chronic Granulomatous DiseasePhase 1/2NCT06559176
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
A Phase 1/2 study testing PM359 in people with Chronic Granulomatous Disease and Granulomatous Disease, Chronic, run by Prime Medicine, Inc. at 5 sites in 3 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Chronic Granulomatous Disease, Granulomatous Disease, Chronic
- What is being tested?
- PM359
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 5 sitesin 3 countries
- Study dates
- 17 Oct 2024to Feb 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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:
- Contraindication to mobilization and apheresis, including severe allergic reaction to receipt of any medication or other drug substance required for mobilization and apheresis (e.g., G-CSF, plerixafor) or PM359 manufacture (e.g., DMSO).
- Contraindication to receipt of the conditioning agent, busulfan.
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- 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.
Trial locations and contacts
5 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 3 countries
United States3 sites
NIH Clinical Center
Bethesda, Maryland, 20892
University of California Los Angeles Medical Center
Los Angeles, California, 90027
The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, 37203
United Kingdom1 site
University College of London Hospital
London, England, NW1 2PG
About this study
Official title: A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing (PM359) in Participants With Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene
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.
Read the detailed description
Chronic Granulomatous Disease (CGD) is a rare genetic disease affecting the white blood cells, leading to failure of innate immunity against a variety of human pathogens and is also associated with autoimmune and inflammatory conditions. Approximately 20-25% of people with CGD inherit a mutation commonly known as "delGT" in both copies of the NCF1 gene, which encodes the p47phox protein.
This study seeks to understand the safety and efficacy of a new gene editing technology, known as Prime Editing, in participants with autosomal recessive CGD caused by the delGT mutation in NCF1. Autologous CD34+ cells are collected from the participant via mobilization and apheresis, shipped to a central manufacturing facility and modified using Prime Editing to 'correct' the delGT mutation causing p47phox CGD. After manufacture, the Prime Edited stem cells (PM359) will be shipped to the study site, where they will be infused back into the participant following a preparative procedure known as conditioning.
The study will initially enroll adult participants (aged ≥ 18) and plans to then move into adolescents aged 12 - 17, followed by children aged 6 - 11.
The study is currently enrolling a limited number of participants by invitation. Treating physicians or patients may contact cgdtrial@primemedicine.com to inquire about potential participation.
- Being tested
- PM359
- Keywords
- Granulomatous Disease, Chronic, Chronic Granulomatous Disease, Autosomal Recessive Chronic Granulomatous Disease, Genetic Therapy, Gene Therapy, Gene Editing, Genome Editing, Stem Cell Transplantation, Transplantation, Stem Cell, Hematopoietic Stem Cell Transplantation, Transplantation, Hematopoietic Stem Cell
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PM359Biological
Single dose of PM359 administered autologously by intravenous (I.V.) infusion following myeloablative conditioning with busulfan
| Study group | Role | What they receive |
|---|---|---|
| PM359 | Experimental | 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. Receives: Biological: PM359 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusion
Measured: PM359 infusion through Month 12 after PM359 infusion
Percentage of participants with sustained reconstitution of NADPH oxidase activity in neutrophils
Measured: At Month 6 and Month 12 after PM359 infusion, as compared to baseline
Secondary outcomes (16)
Frequency of all drug product-related AEs, ≥ Grade 3 AEs, and serious adverse events (SAEs)
Measured: Signing of ICF through Month 36 following PM359 infusion
Time to neutrophil engraftment
Measured: From PM359 infusion through engraftment, typically within 2-3 weeks but assessed up to 36 months
Transplant related mortality
Measured: From PM359 infusion, assessed at 100 Days and 1 Year post-PM359 infusion
Overall survival
Measured: From PM359 infusion, assessed at 1 Year and 3 Years post-PM359 infusion
Incidence of acute graft-versus-host disease (GvHD)
Measured: From PM359 infusion through Month 36
Incidence of graft failure or rejection
Measured: From PM359 infusion through Month 36
Durability of multi-lineage hematopoietic cell reconstitution
Measured: Assessed at 12, 24 and 36 months following PM359 infusion
Percentage of participants with clinical evidence of malignancy, pre-malignancy or myelodysplasia
Measured: From PM359 infusion through Month 36
Percentage of participants with sustained reconstitution of NADPH oxidase activity
Measured: Assessed at Month 6 and Month 12 after PM359 infusion
Percentage of participants with sustained reconstitution of NADPH oxidase activity
Measured: Assessed at Months 3, 18, 24 and 36 after PM359 infusion
Reconstitution of NADPH oxidase activity in peripheral granulocytes, as measured by DHR assay
Measured: Months 1, 2, 3, 6, 12, 18, 24, and 36 after PM359 infusion
Frequency of new or worsening severe bacterial or fungal infections requiring anti-microbial therapy, as compared to baseline
Measured: From Month 6 following PM359 infusion through Month 36
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
Measured: From Month 6 following PM359 infusion through Month 36
Frequency of autoimmune or inflammatory, non-infectious processes consistent with CGD, with symptom severity moderate or greater, as compared to baseline
Measured: From 1 Year following PM359 infusion through Month 36
Resolution of pre-existing active infections (defined as infection present at time of study enrollment and ongoing at time of PM359 infusion) following PM359 infusion
Measured: From PM359 infusion until resolution of active infection, assessed up to Month 36
Resolution of pre-existing autoimmune or inflammatory (non-infectious) processes (defined as autoimmune or inflammatory processes present at time of study enrollment and ongoing at time of PM359 infusion) following PM359 infusion
Measured: From PM359 infusion until resolution of autoimmune or inflammatory process, assessed up to Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2024
- Study start17 Oct 2024
- Primary completionJan 2030 (estimated)
- Study completionFeb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Prime Medicine, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06559176
- Sponsor's protocol ID
- Prime-0101
- Status last verified
- Mar 2026
- First submitted
- 11 Jul 2024
- Last updated
- 6 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: No plans to make IPD available to other researchers.
Publications
Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A, Liu DR. Search-and-replace genome editing without double-strand breaks or donor DNA. Nature. 2019 Dec;576(7785):149-157. doi: 10.1038/s41586-019-1711-4. Epub 2019 Oct 21.
PubMed 31634902Gori JL, Haddad E, Frangoul H, Kohn DB, Morris EC, Martin BN, Deary BA, Nickerson M, Scholz RL, Fernandez I, Leveille K, De Ravin SS, Kang EM, Pierzynski M, Estwick T, Littel P, Kuhns DB, Long Priel DA, Teira P, Turvey S, Arnold J, Evans MD, McManus M, Carpenter B, Waterman DP, Anzalone AV, Petrusich A, Osuna CE, O'Malley TT, Stewart-Ornstein J, Heath JM, Nehilla BJ, Asmal M, Malech HL. Prime Editing for p47phox-Deficient Chronic Granulomatous Disease. N Engl J Med. 2026 Mar 26;394(12):1195-1203. doi: 10.1056/NEJMoa2509807. Epub 2025 Dec 7.
PubMed 41358590
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing (PM359) in Participants With Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene
- ClinicalTrials.gov ID
- NCT06559176
- Acronym / sponsor ID
- Prime-0101
- Keywords
- Granulomatous Disease, Chronic, Chronic Granulomatous Disease, Autosomal Recessive Chronic Granulomatous Disease, Genetic Therapy, Gene Therapy, Gene Editing, Genome Editing, Stem Cell Transplantation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Prime Medicine, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Oct 2024 (actual)
- Primary completion
- Jan 2030 (estimated)
- Study completion
- Feb 2030 (estimated)
- First posted
- 19 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Chronic Granulomatous Disease (CGD) is a rare genetic disease affecting the white blood cells, leading to failure of innate immunity against a variety of human pathogens and is also associated with autoimmune and inflammatory conditions. Approximately 20-25% of people with CGD inherit a mutation commonly known as "delGT" in both copies of the NCF1 gene, which encodes the p47phox protein.
This study seeks to understand the safety and efficacy of a new gene editing technology, known as Prime Editing, in participants with autosomal recessive CGD caused by the delGT mutation in NCF1. Autologous CD34+ cells are collected from the participant via mobilization and apheresis, shipped to a central manufacturing facility and modified using Prime Editing to 'correct' the delGT mutation causing p47phox CGD. After manufacture, the Prime Edited stem cells (PM359) will be shipped to the study site, where they will be infused back into the participant following a preparative procedure known as conditioning.
The study will initially enroll adult participants (aged ≥ 18) and plans to then move into adolescents aged 12 - 17, followed by children aged 6 - 11.
The study is currently enrolling a limited number of participants by invitation. Treating physicians or patients may contact cgdtrial@primemedicine.com to inquire about potential participation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PM359 | Experimental | Biological: PM359PM359 is an autologous CD34+ hematopoietic stem cell (HSC) suspension that is Prime Edited at the NCF1 locus resulting in expression of the p47phox protein. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (12 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PM359Single dose of PM359 administered autologously by intravenous (I.V.) infusion following myeloablative conditioning with busulfan | Biological | PM359 |
Primary outcome
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
Percentage of participants with sustained reconstitution of NADPH oxidase activity in neutrophils
Time frame: At Month 6 and Month 12 after PM359 infusion, as compared to baseline
16 secondary outcomes
Frequency of all drug product-related AEs, ≥ Grade 3 AEs, and serious adverse events (SAEs)
Time frame: Signing of ICF through Month 36 following PM359 infusion
Time to neutrophil engraftment
Time frame: From PM359 infusion through engraftment, typically within 2-3 weeks but assessed up to 36 months
Transplant related mortality
Time frame: From PM359 infusion, assessed at 100 Days and 1 Year post-PM359 infusion
Overall survival
Time frame: From PM359 infusion, assessed at 1 Year and 3 Years post-PM359 infusion
Incidence of acute graft-versus-host disease (GvHD)
Time frame: From PM359 infusion through Month 36
Incidence of graft failure or rejection
Time frame: From PM359 infusion through Month 36
Durability of multi-lineage hematopoietic cell reconstitution
Time frame: Assessed at 12, 24 and 36 months following PM359 infusion
Percentage of participants with clinical evidence of malignancy, pre-malignancy or myelodysplasia
Time frame: From PM359 infusion through Month 36
Percentage of participants with sustained reconstitution of NADPH oxidase activity
Time frame: Assessed at Month 6 and Month 12 after PM359 infusion
Percentage of participants with sustained reconstitution of NADPH oxidase activity
Time frame: Assessed at Months 3, 18, 24 and 36 after PM359 infusion
Reconstitution of NADPH oxidase activity in peripheral granulocytes, as measured by DHR assay
Time frame: Months 1, 2, 3, 6, 12, 18, 24, and 36 after PM359 infusion
Frequency of new or worsening severe bacterial or fungal infections requiring anti-microbial therapy, as compared to baseline
Time frame: From Month 6 following PM359 infusion through Month 36
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
Time frame: From Month 6 following PM359 infusion through Month 36
Frequency of autoimmune or inflammatory, non-infectious processes consistent with CGD, with symptom severity moderate or greater, as compared to baseline
Time frame: From 1 Year following PM359 infusion through Month 36
Resolution of pre-existing active infections (defined as infection present at time of study enrollment and ongoing at time of PM359 infusion) following PM359 infusion
Time frame: From PM359 infusion until resolution of active infection, assessed up to Month 36
Resolution of pre-existing autoimmune or inflammatory (non-infectious) processes (defined as autoimmune or inflammatory processes present at time of study enrollment and ongoing at time of PM359 infusion) following PM359 infusion
Time frame: From PM359 infusion until resolution of autoimmune or inflammatory process, assessed up to Month 36
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2026.
- Background: PubMed 31634902Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A, Liu DR. Search-and-replace genome editing without double-strand breaks or donor DNA. Nature. 2019 Dec;576(7785):149-157. doi: 10.1038/s41586-019-1711-4. Epub 2019 Oct 21.
- Derived from this study: PubMed 41358590Gori JL, Haddad E, Frangoul H, Kohn DB, Morris EC, Martin BN, Deary BA, Nickerson M, Scholz RL, Fernandez I, Leveille K, De Ravin SS, Kang EM, Pierzynski M, Estwick T, Littel P, Kuhns DB, Long Priel DA, Teira P, Turvey S, Arnold J, Evans MD, McManus M, Carpenter B, Waterman DP, Anzalone AV, Petrusich A, Osuna CE, O'Malley TT, Stewart-Ornstein J, Heath JM, Nehilla BJ, Asmal M, Malech HL. Prime Editing for p47phox-Deficient Chronic Granulomatous Disease. N Engl J Med. 2026 Mar 26;394(12):1195-1203. doi: 10.1056/NEJMoa2509807. Epub 2025 Dec 7.
- Individual participant dataSharing planned: No. No plans to make IPD available to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PM359 in people with Chronic Granulomatous Disease and Granulomatous Disease, Chronic, run by Prime Medicine, Inc. at 5 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 6 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06559176, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2026.