PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

Q: Who can participate in NCT07429240?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

Q: How do I contact the trial site for NCT07429240?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Q: Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Q: Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

A Phase 1/2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD) (NCT07429240) is a Phase 1 / Phase 2 interventional studying Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD, sponsored by Precision BioSciences, Inc.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 18 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Males, 2 to 7 years of age, inclusive, at the time of willing to sign a consent form/assent 2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\]) 3. Clinical phenotype consistent with DMD in the opinion of the Investigator 4. Ability to complete age-appropriate motor testing assessments requirements. Participants aged 2 to \< 4 years at the time of screening must: 1. Be able to walk at least 10 meters independently (without assistive devices). 2. Be able to rise from the floor without physical assistance (use of a Gowers' maneuver is acceptable). Participants aged 4 to 7 years at the time of screening must: 3. Be able to walk at least 100 meters independently (without assistive devices). 4. Have an NSAA total score between 16 and 29, inclusive. 5. Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule. 6. The participant's parent(s)/LAR(s) are willing and able to provide written willing to sign a consent form prior to the initiation of any trial-specific procedures; where applicable, the participant must provide written or verbal assent in accordance with local regulations. 7. The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial. Who Should NOT Join This Trial: 1. Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time. 2. Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1. 3. Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: 1. Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent 2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\]) 3. Clinical phenotype consistent with DMD in the opinion of the Investigator 4. Ability to complete age-appropriate motor testing assessments requirements. Participants aged 2 to \< 4 years at the time of screening must: 1. Be able to walk at least 10 meters independently (without assistive devices). 2. Be able to rise from the floor without physical assistance (use of a Gowers' maneuver is acceptable). Participants aged 4 to 7 years at the time of screening must: 3. Be able to walk at least 100 meters independently (without assistive devices). 4. Have an NSAA total score between 16 and 29, inclusive. 5. Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule. 6. The participant's parent(s)/LAR(s) are willing and able to provide written informed consent prior to the initiation of any trial-specific procedures; where applicable, the participant must provide written or verbal assent in accordance with local regulations. 7. The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial. Exclusion Criteria: 1. Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time. 2. Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1. 3. Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration. 4. Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose. 5. Concurrent enrollment in another clinical trial, unless it is observational (non-interventional). 6. A positive test for antibodies to AAV9 7. A participant has any condition that would contraindicate treatment with immunosuppression. 8. Participants with pathogenic mutations in exons 1-44 and/or exons 56-79. 9. Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF \<50% on screening echocardiogram.

Treatments Being Tested

BIOLOGICAL

PBGENE-DMD (IV)

Participants will receive a single dose of PBGENE-DMD

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Arkansas Children's Hospital

Little Rock, Arkansas, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07429240), the sponsor (Precision BioSciences, Inc.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07429240 clinical trial studying?

Who can participate in NCT07429240?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07429240?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07429240. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07429240. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.