BridgeMD
Tests treatment safety and results for Duchenne Muscular Dystrophy (DMD)
Official title A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
ClinicalTrials.gov ID: NCT07037862
What this study is testing
What is ENTR-601-44?
ENTR-601-44 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy (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
- This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-44 is, learn about any side effects, and look at the potential positive effects of ENTR-601-44, compared to placebo.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 20, men only
You may be able to join if
- Principal 1. Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44...
- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
- Part A: 4-20 years of age, inclusive.
- Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
You likely can't join if
- Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
- Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's...
- Use of the following medications:
- Prior treatment with any exon skipping therapy at any time
- Prior treatment with any gene therapy at any time
- Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
See the full eligibility criteria
- Principal 1. Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.
- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
- Part A: 4-20 years of age, inclusive.
- Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
- Other protocol-defined criteria apply. Principal
- Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
- Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety.
- Use of the following medications:
- Prior treatment with any exon skipping therapy at any time
- Prior treatment with any gene therapy at any time
- Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
- Use of an immunosuppressants (other than oral corticosteroids for DMD conditions)
- Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat
- Laboratory abnormalities.
- Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
- Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) \>450 msec at Screening or prior to the first dose of...
- Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).
- Other protocol-defined criteria apply.
The study team makes the final eligibility decision.
Where it's taking place
- Ghent, Belgium
- Leuven, Belgium
- Liège, Belgium
- Milan, Italy
- Rome, Italy
- Barcelona, Spain
- Leeds, United Kingdom
- Liverpool, United Kingdom
- London, United Kingdom
- Manchester, United Kingdom
- Newcastle upon Tyne, United Kingdom
- Oxford, United Kingdom
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, 4 years to 20 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Ghent, Belgium; Leuven, Belgium; Liège, Belgium; Milan, Italy; Rome, Italy; Barcelona, Spain and 6 more location(s). 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.