Gene therapy SPOT-03 enters human testing for duchenne muscular dystrophy
NCT ID NCT07188012
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD), aged 2 to 8. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it increases dystrophin protein levels in muscles, which could help slow the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SPOT-03 (gene therapy)
What this could lead to
If successful, this could point toward a treatment that helps boys with Duchenne muscular dystrophy produce dystrophin protein, potentially slowing muscle damage.
What could go wrong
This is a very early, small trial (9 participants) focused on safety, not effectiveness. Gene therapies can have side effects, and it may not work as hoped.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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