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Gene Therapy

Gene Therapy 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
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.
Autosomal recessive Chronic Granulomatous Disease due to the delGT mutation in NCF1 causing dysfunction of p47phox
Must not have
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)
Ongoing inflammatory condition that is ≥ CTCAE v5.0 Grade 3 despite high-dose steroids (≥ 0.5 mg/kg/day of prednisone and/or equivalent)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This trial is testing a new gene therapy for people with a specific genetic disorder called Chronic Granulomatous Disease. The therapy involves modifying the patient's own stem cells outside the body and then transplanting

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
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I have had a severe infection or an ongoing health issue due to CGD.
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I have Chronic Granulomatous Disease with a specific genetic mutation.
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I have been under care at a specialized center for over 2 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am under 16 and have a fully matched family donor for my treatment.
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I have a severe ongoing inflammation not controlled by high-dose steroids.
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I currently have a bacterial or fungal infection in my blood.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

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.

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Who is running the clinical trial?

Prime Medicine, Inc.Lead Sponsor
~8 spots leftby Sep 2029
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