Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug shows promise for rare muscle disease in kids

NCT ID NCT03692312

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called tideglusib against a placebo in 56 children aged 6 to 16 with congenital myotonic dystrophy, a rare genetic muscle disorder. The goal was to see if tideglusib could reduce symptoms like muscle weakness and breathing problems. The trial was completed, but results are not yet publicly available.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

56 people

The number who actually took part.

Started

Mar 2021

Finished

Apr 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 16 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Male or female children and adolescents aged ≥6 years and ≤16 years 2. Diagnosis of Congenital DM1 (also known as Steinert's disease) * Diagnosis must be genetically confirmed * One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth: * Hypotonia * Generalized weakness * Respiratory insufficiency * Feeding difficulties * Clubfoot or another musculoskeletal deformity 3. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed) 4. Written, voluntary informed consent must be obtained before any study related procedures are conducted. * Where a parent or LAR provides consent, there must also be assent from the subject 5. Subject's caregiver must be willing and able to support participation for duration of study 6. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol Exclusion Criteria: 1. Not able to walk; (full time wheel chair use) 2. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m² 3. New or change in medications/therapies within 4 weeks prior to Screening 4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline 5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin) 6. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months 7. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment 8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Congenital myotonic dystrophy are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

congenital myotonic dystrophy myotonic dystrophy

As listed by the trial registrant

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

Congenital myotonic dystrophy

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital London Health Sciences Centre (LHSC)

    London, Ontario, N6A4G5, Canada

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • New Zealand Clinical Research (NZCR)

    Auckland, 1010, New Zealand

  • Newcastle University

    Newcastle upon Tyne, NE2 4HH, United Kingdom

  • Stanford University

    Palo Alto, California, 94304, United States

  • The Bright Alliance

    Randwick, New South Wales, 2031, Australia

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84112, United States

  • Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.

    Richmond, Virginia, 23219, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.