New drug trial for duchenne MD halted early – what we know
NCT ID NCT06079736
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- PGN-EDO51 (a drug given by IV infusion to help produce a shorter but functional dystrophin protein)
- What this could lead to
- If successful, this could lead to a treatment that slows muscle decline in Duchenne muscular dystrophy patients who are candidates for exon 51 skipping.
- What could go wrong
- This is an early-phase, small trial (7 participants) focused on safety, not yet on effectiveness. The study was terminated, which raises uncertainty about its future.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H1G9, Canada
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CHU de Québec
Québec, Quebec, G1V4G2, Canada
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Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario, K1H8L1, Canada
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Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, E3B0C7, Canada
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The Hospital for Sick Children (SickKids)
Toronto, Ontario, M5G0A4, Canada
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a lower steroid dose preserve strength in young boys with DMD?
- Can a targeted infusion slow muscle decline in duchenne? a new trial aims to find out.
- Can a massive patient database unlock new treatments for muscular dystrophy?
- Umbilical cord stem cells aim to slow muscle loss in duchenne boys
- Experimental drug aims to preserve arm and lung function in boys with advanced duchenne
- Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy