NCT07119775|Unknown
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
1 other identifier
AMO-02 Expanded Access
Study Type
expanded_access
Target
N/A
Locations
0 countries
Sites
N/A
Timeline
RegisteredAug 2025
Brief Summary
This treatment plan is limited to a single patient with Congenital Myotonic Dystrophy, who is ineligible or otherwise unable to participate in ongoing clinical trials.
Trial Health
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 6, 2025
Completed7 days until next milestone
First Posted
Study publicly available on registry
August 13, 2025
CompletedLast Updated
August 13, 2025
Status Verified
August 1, 2025
First QC Date
August 6, 2025
Last Update Submit
August 6, 2025
Conditions
Keywords
TideglusibCongenital Myotonic DystrophyMyotonic DystrophyDystrophia MyotonicaMyotonia AtrophicaMyotonia DystrophicaMyotonic Dystrophy, Congenital Steinert DiseaseSteinert Myotonic DystrophySteinert's Disease
Interventions
TideglusibDRUG
Sponsors & Collaborators
MeSH Terms
Conditions
Myotonic Dystrophy
Interventions
tideglusib
Condition Hierarchy (Ancestors)
Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities
Central Study Contacts
Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM
CONTACT
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 6, 2025
First Posted
August 13, 2025
Last Updated
August 13, 2025
Record last verified: 2025-08