- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT07119775
- Original Trial
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
August 6, 2025 updated by: AMO Pharma Limited
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.
Study Overview
Study Type
Expanded Access
Expanded Access Type
- Individual Patients
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
N/A
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
August 6, 2025
First Submitted That Met QC Criteria
August 6, 2025
First Posted (Actual)
August 13, 2025
Study Record Updates
Last Update Posted (Actual)
August 13, 2025
Last Update Submitted That Met QC Criteria
August 6, 2025
Last Verified
August 1, 2025
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Musculoskeletal Diseases
- Nervous System Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Heredodegenerative Disorders, Nervous System
- Muscular Disorders, Atrophic
- Muscular Dystrophies
- Myotonic Disorders
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities
- Myotonic Dystrophy
- tideglusib
Other Study ID Numbers
- AMO-02 Expanded Access
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Congenital Myotonic Dystrophy
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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMuscular Dystrophy | Myotonic Dystrophy Type 2 | Myotonic Dystrophy Type 1 | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Myotonic Muscular DystrophyUnited States
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Fundació Institut Germans Trias i PujolGermans Trias i Pujol Hospital; Hospital Donostia; Hospital de Basurto; Hospital... and other collaboratorsRecruitingMyotonic Dystrophy Type 1 | Myotonic Dystrophy 1 | DM1 | Steinert Disease | Myotonic Dystrophy, CongenitalSpain
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Myotonic Dystrophy FoundationRecruitingMyotonic Dystrophy | Myotonic Dystrophy 1 | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Steinert Disease | Dystrophia Myotonica 1 | Myotonic Dystrophy 2 | Dystrophia Myotonica | Dystrophia Myotonica 2 | Myotonia Dystrophica | Myotonic Dystrophy, Congenital | Myotonic... and other conditionsUnited States
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Avidity Biosciences, Inc.CompletedMyotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert DiseaseUnited States
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Hanns LochmullerUniversity of OttawaRecruitingMyotonic Dystrophy Type 1 | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy, Type 1 (DM1) | Myotonic Dystrophy, CongenitalCanada
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Avidity Biosciences, Inc.Active, not recruitingMyotonic Dystrophy | Myotonic Disorders | Myotonia | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert Disease | SteinertUnited States, Denmark, Japan, Canada, Netherlands, United Kingdom, France, Italy, Germany, Spain
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Virginia Commonwealth UniversityUniversity of California, San Diego; National Institute of Neurological Disorders... and other collaboratorsRecruitingMyotonic Dystrophy | Congenital Myotonic Dystrophy | Childhood Myotonic DystrophyUnited States
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Avidity Biosciences, Inc.CompletedNervous System Diseases | Genetic Diseases, Inborn | Musculoskeletal Diseases | Muscular Diseases | Neuromuscular Diseases | Neurodegenerative Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Heredodegenerative Disorders, Nervous System | Myotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy... and other conditionsUnited States
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University of RochesterRecruitingMyotonic Dystrophy Type 1 | Congenital DM1 | Juvenile DM1 | Childhood DM1United States
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PepGen IncRecruitingMyotonic Dystrophy 1Canada
Clinical Trials on Tideglusib
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AMO Pharma LimitedCompletedMyotonic Dystrophy 1United Kingdom
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Hamilton Health Sciences CorporationCanadian Institutes of Health Research (CIHR); Population Health Research Institute and other collaboratorsRecruitingArrhythmogenic Right Ventricular Cardiomyopathy | Arrhythmogenic CardiomyopathyCanada
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AMO Pharma LimitedCompletedCongenital Myotonic DystrophyUnited States, Canada, New Zealand, Australia, United Kingdom
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University of ZurichUniversity Hospital, Geneva; University of Lausanne Hospitals; University of... and other collaboratorsNot yet recruitingAmyotrophic Lateral SclerosisSwitzerland
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Holland Bloorview Kids Rehabilitation HospitalMcMaster University; Unity Health Toronto; University of Toronto; Western University...CompletedAutism Spectrum DisordersCanada
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AMO Pharma LimitedRecruitingCongenital Myotonic DystrophyUnited States, Canada, Australia, New Zealand
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Noscira SACompletedAlzheimer´s DiseaseGermany
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Noscira SAi3 ResearchCompletedProgressive Supranuclear PalsyUnited States, Germany, Spain, United Kingdom
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Noscira SAICON Clinical ResearchCompletedAlzheimer's DiseaseBelgium, Finland, France, Germany, Spain, United Kingdom
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Hannover Medical SchoolGilead Sciences; HepNet Study House, German Liverfoundation; German Center for...CompletedHepatitis C | Acute Hepatitis CGermany