New drug hopes to strengthen muscles in rare genetic disorder
NCT ID NCT07560020
First seen Jun 27, 2026 · Last updated Jul 16, 2026 · Updated 2 times
Summary
This study tests a medicine called surlorian in 28 adults with a rare genetic muscle disease (RYR1-related myopathy) that causes weakness. The goal is to see if surlorian improves muscle strength and function, like standing from a chair or walking. Participants will be randomly assigned to receive either surlorian or a placebo, and neither they nor the doctors will know which they get.
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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
Email: •••••@•••••
Locations
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AP-HM- Hôpital de La Timone
NOT_YET_RECRUITINGMarseille, Bouches-du-Rhône, 13385, France
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Charité - Campus Berlin Buch
RECRUITINGBerlin, State of Berlin, 13125, Germany
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Hospital Universitario Vall d'Hebron - PPDS
NOT_YET_RECRUITINGBarcelona, Barcelona, 8035, Spain
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Hospital Universitario de Donostia
NOT_YET_RECRUITINGSan Sebastián, Guipúzcoa, 20014, Spain
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Institut de Myologie - Hôpital de La Pitié-Salpétrière
NOT_YET_RECRUITINGParis, 73013, France
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Radboud Universitair Medisch Centrum
NOT_YET_RECRUITINGNijmegen, Gelderland, 6500 HB, Netherlands
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The Robert Jones and Agnes Hunt Orthopaedic Hospital
NOT_YET_RECRUITINGOswestry, Shropshire, SY10 7AG, United Kingdom
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University College Hospital - PPDS
RECRUITINGLondon, WC1N 3BG, United Kingdom
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Universitätsklinikum Ulm
NOT_YET_RECRUITINGUlm, Baden-Wurttemberg, 89081, Germany