St Louis, MONCT07429240Now EnrollingIRB Ready

Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD Clinical Trial in St Louis, MO

Access cutting-edge duchenne muscular dystrophy with mutations amenable to pbgene-dmd treatment through this clinical trial at a research site in St Louis. Study-provided care at no cost to qualified participants.

Sponsored by Precision BioSciences, Inc.

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Expert Care in St Louis

Access duchenne muscular dystrophy with mutations amenable to pbgene-dmd specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related duchenne muscular dystrophy with mutations amenable to pbgene-dmd treatment provided free

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Check if you qualify for this duchenne muscular dystrophy with mutations amenable to pbgene-dmd clinical trial in St Louis, MO

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Why Participate?

  • No-Cost Study Care

  • Local to St Louis

    Convenient for MO residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit St Louis site if eligible
  4. 4Begin participation

About This Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD Study in St Louis

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.

Sponsor: Precision BioSciences, Inc.

Who Can Participate

Inclusion Criteria

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).
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.

Exclusion Criteria

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
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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in St Louis?

Yes, this clinical trial (NCT07429240) has an active research site in St Louis, MO that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD Treatment Options in St Louis, MO

If you're searching for duchenne muscular dystrophy with mutations amenable to pbgene-dmd treatment options in St Louis, MO, this clinical trial (NCT07429240) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our St Louis research site is actively enrolling participants for this clinical trial. You'll receive care from experienced duchenne muscular dystrophy with mutations amenable to pbgene-dmd specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all duchenne muscular dystrophy with mutations amenable to pbgene-dmd clinical trials near you to find additional studies recruiting in your area.

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