Experimental drug tideglusib tested for rare muscle disease
NCT ID NCT02858908
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This phase 2 study tested the safety and effectiveness of tideglusib, an experimental drug, in 16 adolescents and adults with congenital or juvenile-onset myotonic dystrophy type 1. Participants received either 400 mg or 1000 mg of tideglusib daily. The study measured side effects and how the drug moves through the body. Results are not yet available.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Tideglusib (an experimental drug taken by mouth)
What this could lead to
If successful, this could point toward a treatment that helps control symptoms of myotonic dystrophy in adolescents and adults.
What could go wrong
This is a small, early-phase trial with only 16 participants, so results may not apply to everyone. The drug may not prove effective or could have side effects.
Disclaimer
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This is a summary of
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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Locations
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Newcastle-upon-Tyne Hospitals NHS Trust
Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom