Congenital Myotonic DystrophyNCT07119775
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
A study testing Tideglusib in people with Congenital Myotonic Dystrophy, run by AMO Pharma Limited.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Myotonic Dystrophy
- What is being tested?
- Tideglusib
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Child, Adult, Older adult
Trial locations and contacts
No sites are listed yet.
Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM contact
About this study
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.
- Conditions
- Congenital Myotonic Dystrophy
- Being tested
- Tideglusib
- Keywords
- Tideglusib, Congenital Myotonic Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Myotonia Atrophica, Myotonia Dystrophica, Myotonic Dystrophy, Congenital Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TideglusibDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Myotonic DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- AMO Pharma Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07119775
- Sponsor's protocol ID
- AMO-02 Expanded Access
- Status last verified
- Aug 2025
- First submitted
- 6 Aug 2025
- Last updated
- 13 Aug 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
- ClinicalTrials.gov ID
- NCT07119775
- Acronym / sponsor ID
- AMO-02 Expanded Access
- Conditions
- Congenital Myotonic Dystrophy
- Keywords
- Tideglusib, Congenital Myotonic Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Myotonia Atrophica, Myotonia Dystrophica, Myotonic Dystrophy, Congenital Steinert Disease, Steinert Myotonic Dystrophy
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- AMO Pharma LimitedIndustry (pharma, biotech, devices)
- First posted
- 13 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tideglusib | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM contact
Questions about this trial
What is this clinical trial studying?
A study testing Tideglusib in people with Congenital Myotonic Dystrophy, run by AMO Pharma Limited.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 13 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM (+44 (0)1270 270010, h.graystephens@amo-pharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2025.