Plain-English translation of NCT07038824 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing a new medicine called designed to treat Duchenne muscular dystrophy (DMD), a rare genetic muscle disease. The study has two parts: first, researchers will test how safe the medication is and find the right dose, and then they will see whether it actually helps slow muscle weakness. You would receive the treatment through IV infusions every 6 weeks and have regular check-ups including blood tests, muscle biopsies, and strength tests.
Duchenne muscular dystrophy is a serious genetic condition that causes progressive muscle weakness, and there are limited treatment options available. This trial is trying to see whether this new medication can help preserve muscle function in patients whose genetic mutation makes them eligible for this type of therapy.
You likely qualify if…
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You would visit the clinic multiple times over several weeks to receive slow IV infusions of either the study medication or placebo (a non-active treatment that looks the same) every 6 weeks. At each visit, you would have blood and urine tests, physical exams, muscle strength tests, and questionnaires; you would also have muscle biopsies at the start and end of the study so researchers can see if the medication is changing your muscle tissue. You can continue taking your regular DMD medications during the study, and there is a chance to move into an extended open-label phase where everyone receives the active treatment.
AI-generated summary from trial data · Sep 24, 2026 · Not medical advice
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