Status and phase
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Study type
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Identifiers
About
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-45 and placebo are both called study treatments. The study has 2 parts: Part A: to evaluate if ENTR-601-45 is safe and to determine the best dose of ENTR-601-45 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-45 at the dose determined in Part A.
Participants will:
Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
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 :
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 study drug on Day 1.
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.
Primary purpose
Allocation
Interventional model
Masking
24 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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