Can plasma exchange clear the way for gene therapy in duchenne?
NCT ID NCT06597656
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early study tested whether a blood-cleaning procedure called plasmapheresis could allow boys with Duchenne muscular dystrophy who have antibodies against the gene therapy carrier to still receive the treatment. Only 3 boys were enrolled before the study was stopped early. The goal was to see if the gene therapy could safely produce dystrophin protein in muscle.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- delandistrogene moxeparvovec (gene therapy)
- What this could lead to
- If successful, this approach could allow more boys with Duchenne to receive gene therapy even if they have pre-existing antibodies.
- What could go wrong
- This was a very small, early-phase trial that was terminated early, so results are limited. The added step of plasmapheresis carries its own risks and may not overcome the immune barrier.
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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
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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University of Florida, College of Medicine
Gainesville, Florida, 32610, United States
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Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110, United States
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