Experimental drug tideglusib tested for rare muscle disease

NCT ID NCT02858908

Completed Disease control Sponsor: AMO Pharma Limited Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

congenital myotonic dystrophy juvenile-onset Steinert myotonic dystrophy myotonic dystrophy myotonic dystrophy type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MYOTONIC DYSTROPHY 1

Contacts and locations

Locations

Newcastle-upon-Tyne Hospitals NHS Trust

Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom

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