Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMDPhase 1/2NCT07429240
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
A Phase 1/2 study testing PBGENE-DMD (IV) in people with Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD, run by Precision BioSciences, Inc. at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 7 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
- What is being tested?
- PBGENE-DMD (IV)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- 24 Apr 2026to Dec 2029
- 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 2 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
- Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive])
- Clinical phenotype consistent with DMD in the opinion of the Investigator
- Ability to complete age-appropriate motor testing assessments requirements.
- Participants aged 2 to < 4 years at the time of screening must:
- Be able to walk at least 10 meters independently (without assistive devices).
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- 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:
- Be able to walk at least 100 meters independently (without assistive devices).
- Have an NSAA total score between 16 and 29, inclusive.
- Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.
- 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.
- The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial.
Who cannot join
- Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.
- Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.
- 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.
- 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.
- Concurrent enrollment in another clinical trial, unless it is observational (non-interventional).
- A positive test for antibodies to AAV9
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- A participant has any condition that would contraindicate treatment with immunosuppression.
- Participants with pathogenic mutations in exons 1-44 and/or exons 56-79.
- Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF <50% on screening echocardiogram.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Kristin Hecke, MacleanKD@archildrens.org
Aravindhan Veerapandiyan, MD
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Natalie Goedeker, 314-362-4919, NeuromusclePediatricResearch@wustl.edu
Craig Zaidman, MD
About this study
Official title: 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)
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.
Read the detailed description
This is a Phase 1/2a, open-label, multicenter trial designed to evaluate the safety, tolerability, and primary efficacy of a single IV dose of PBGENE-DMD in male participants with DMD presenting with mutations that may be amenable to treatment with PBGENE-DMD. A structured, multimodal, short-term immunomodulatory regimen will be administered around the time of dosing to mitigate the risk of potential immune-mediated responses.
The trial consists of two parts: Part 1 is to confirm a safe and well-tolerated single dose of PBGENE-DMD that may be further evaluated in Part 2 (expansion).
A total of up to 18 participants may be enrolled in this trial. Total duration of trial participation for each participant: approximately 130 weeks.
- Being tested
- PBGENE-DMD (IV)
- Keywords
- Duchenne muscular dystrophy, DMD, X-linked Muscle wasting disease, Muscular dystrophy, Progressive muscle weakness, Gene editing, AAV serotype 9, AAV-9, Exon 45-55 excision
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBGENE-DMD (IV)Biological
Participants will receive a single dose of PBGENE-DMD
| Study group | Role | What they receive |
|---|---|---|
| Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort | Experimental | The trial is planned to enroll participants into 2 parts as follows:
* Part 1 (Initial Safety) A total of up to 6 participants may be enrolled.
* Part 2 (Expansion) Up to 12 participants Receives: Biological: PBGENE-DMD (IV) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment
Measured: From Dosing through Week 104
Secondary outcomes (1)
Dystrophin expression in skeletal muscle
Measurement of Biologic activity of PBGENE-DMD through dystrophin expression in skeletal muscle
Measured: Week 12, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2026
- Study start24 Apr 2026
- Primary completionNov 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMDStudies 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
- Model description
- A single dose of PBGENE-DMD administered Intravenously (IV) following screening and pretreatment. Total duration of trial intervention for each participant: approximately 130 weeks
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Precision BioSciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07429240
- Sponsor's protocol ID
- PBGENE-DMD-01
- Status last verified
- Oct 2026
- First submitted
- 17 Feb 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07429240
- Acronym / sponsor ID
- PBGENE-DMD-01
- Keywords
- Duchenne muscular dystrophy, DMD, X-linked Muscle wasting disease, Muscular dystrophy, Progressive muscle weakness, Gene editing, AAV serotype 9, AAV-9
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Precision BioSciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Apr 2026 (actual)
- Primary completion
- Nov 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 24 Feb 2026 (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.
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.
Detailed description
This is a Phase 1/2a, open-label, multicenter trial designed to evaluate the safety, tolerability, and primary efficacy of a single IV dose of PBGENE-DMD in male participants with DMD presenting with mutations that may be amenable to treatment with PBGENE-DMD. A structured, multimodal, short-term immunomodulatory regimen will be administered around the time of dosing to mitigate the risk of potential immune-mediated responses.
The trial consists of two parts: Part 1 is to confirm a safe and well-tolerated single dose of PBGENE-DMD that may be further evaluated in Part 2 (expansion).
A total of up to 18 participants may be enrolled in this trial. Total duration of trial participation for each participant: approximately 130 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A single dose of PBGENE-DMD administered Intravenously (IV) following screening and pretreatment. Total duration of trial intervention for each participant: approximately 130 weeks
| Arm | Type | Receives |
|---|---|---|
| Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort | Experimental | Biological: PBGENE-DMD (IV)The trial is planned to enroll participants into 2 parts as follows: * Part 1 (Initial Safety) A total of up to 6 participants may be enrolled. * Part 2 (Expansion) Up to 12 participants |
Who may qualify
- Ages
- 2 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (13 listed)
- Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
- Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive])
- Clinical phenotype consistent with DMD in the opinion of the Investigator
- Ability to complete age-appropriate motor testing assessments requirements.
- Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.
Key exclusion criteria (9 listed)
- Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.
- Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.
- 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.
- 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.
- Concurrent enrollment in another clinical trial, unless it is observational (non-interventional).
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 |
|---|---|---|
| PBGENE-DMD (IV)Participants will receive a single dose of PBGENE-DMD | Biological | Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort |
Primary outcome
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events
Time frame: From Dosing through Week 104
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment
1 secondary outcome
Dystrophin expression in skeletal muscle
Time frame: Week 12, Week 52
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 21 Oct 2026.
- Individual participant dataSharing planned: No
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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PBGENE-DMD (IV) in people with Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD, run by Precision BioSciences, Inc. at 2 sites in United States.
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 13 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
Contact Precision BioSciences Clin Ops ((800) 593-0346, function-DMD@precisionbiosciences.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events (time frame: From Dosing through Week 104). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Oct 2026.