BridgeMD
Tests treatment safety and results for Duchenne Muscular Dystrophy With Mutations Amenable to...
Official title PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
ClinicalTrials.gov ID: NCT07429240
What this study is testing
What is PBGENE-DMD (IV)?
PBGENE-DMD (IV) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy with mutations amenable to pbgene-dmd.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 7, men only
You may be able to join if
- 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).
You likely can't join if
- 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...
- Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within...
- Concurrent enrollment in another clinical trial, unless it is observational (non-treatment).
- A positive test for antibodies to AAV9
See the full eligibility 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...
- The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial.
- 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...
- 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-treatment).
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- St Louis, Missouri, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years to 7 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; St Louis, Missouri, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.