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. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-44 and placebo are both called study treatments.
The study has 2 parts:
* Part A
* A Double-Blind Period, to evaluate if ENTR-601-44 is safe and to determine the best dose of ENTR-601-44 for Part B. * Following the Double-Blind period, participants will roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated. * Part B
* To further evaluate the effect and safety of ENTR-601-44 at the dose determined in Part A.
Participants will:
* Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B * Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug.
Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.
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.
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.
Prior treatment with any exon skipping therapy at any time
Prior treatment with any gene therapy at any time
Parallel
ENTR-601-44
Drugintravenous infusion
ENTR-601-44 - matching placebo
Drugintravenous infusion
Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and Open Label (OL) Period)
Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.
Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and Open Label (OL) Period)
Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A)
Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)
Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy at End of Study (Part A)
Click on the lead sponsor to view all of their trials.
