Plain-English translation of NCT02740972 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Duchenne muscular dystrophy research guide →Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
Researchers studied , an experimental medication designed to help the body produce working muscle protein in boys with Duchenne muscular dystrophy. The trial tested two different dose levels of this treatment to find the safest and most effective amount. Participants received weekly infusions of either the medication or a placebo (inactive treatment) for 24 weeks while doctors carefully monitored their muscle strength and safety.
Duchenne muscular dystrophy is a serious genetic condition that causes progressive muscle weakness in boys. This trial aimed to test whether the medication could help slow muscle loss and improve function in young patients with a specific type of genetic mutation that might respond to this new approach.
Participants received weekly intravenous infusions (medicine delivered through an IV) of either the medication or placebo for the first four weeks, then continued with the active medication for 20 more weeks (total of 24 weeks). They underwent regular study visits that included physical function tests like walking tests and climbing stairs, blood and urine tests, heart monitoring, and muscle biopsies (small tissue samples taken from the arm) at the beginning and end of the study. All participants also had blood samples taken at several visits to measure how the body processes the medication.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
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