Congenital Myotonic DystrophyPhase 2/3NCT03692312
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
A Phase 2/3 study testing Tideglusib in people with Congenital Myotonic Dystrophy, run by AMO Pharma Limited at 14 sites in 5 countries.
Trial summary
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1). Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Congenital Myotonic Dystrophy
- What is being tested?
- Tideglusib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 14 sitesin 5 countries
- Study dates
- 3 Mar 2021to 4 Apr 2023
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female children and adolescents aged ≥6 years and ≤16 years
- 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
Show all 14Show fewer
- Respiratory insufficiency
- Feeding difficulties
- Clubfoot or another musculoskeletal deformity
- Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
- 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
- Subject's caregiver must be willing and able to support participation for duration of study
- Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
Who cannot join
- Not able to walk; (full time wheel chair use)
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or change in medications/therapies within 4 weeks prior to Screening
- Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
- 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
- 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
- Hypersensitivity to tideglusib and its excipients including allergy to strawberry
Trial locations and contacts
14 sites in 5 countries.
14 sites in 5 countries
United States9 sites
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095
Stanford University
Palo Alto, California, 94304
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.
Richmond, Virginia, 23219
University of Rochester Medical Center
Rochester, New York, 14642
University of Utah Hospital
Salt Lake City, Utah, 84112
Canada2 sites
Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario, N6A4G5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
About this study
Official title: A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
Read the detailed description
This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.
- Conditions
- Congenital Myotonic Dystrophy
- Being tested
- Tideglusib, Placebo
- Keywords
- Tideglusib, AMO-02-MD-2-003, 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
Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily
PlaceboDrug
Matching placebo formulation
| Study group | Role | What they receive |
|---|---|---|
| Tideglusib | Experimental | Weight adjusted tideglusib, orally, once daily Receives: Drug: Tideglusib; Drug: Placebo |
| Placebo | Placebo comparator | Matching placebo, orally, once daily Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1.
The severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.
Measured: Baseline and week 20
Secondary outcomes (10)
Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores
The clinician administered CGI-I rates how much the subject's illness has improved or worsened relative to a baseline state.
A 7-point Likert type scale is used with ratings of 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.
Measured: Baseline and week 20
Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score
The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study. Caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long VAS with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). A score for each concern was to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 3 concerns (minimum = 0 cm, maximum = 30 cm). A higher score represents a worse outcome.
Measured: Baseline and week 20
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
The Caregiver-Completed Congenital DM1 Scale is a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 clinically relevant symptoms that the caregiver is asked to rate the severity of.
The symptoms are rated on a score from 0 to 4 based on overall severity where 0 = symptom not present or is no longer present during the relevant time frame, and 4 = very severe, symptom causes pronounced and consistent impairment and is highly disruptive with regard to daily life. A total CC-CDM1-RS score for each subject was calculated as the sum of the scores where 0 = min and 44 = max. A higher score represents a worse outcome.
Measured: Baseline and week 20
Clinical Global Impression - Severity Scale (CGI-S)
The Clinical Global Impression - Severity Scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis.
Subjects are assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
Measured: Baseline and week 20
10-meter Walk-run Test
The 10-meter walk/run test is a performance measure used to assess walking speed in seconds over a short distance. It can be used as an assessment of functional mobility.
Measured: 20 weeks
Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study.
Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.
Measured: Between Screening to End of Study, up to 28 weeks
Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study.
Abnormal laboratory findings (e.g. hematology, liver function, biochemistry, urinalysis) or other abnormal assessments (e.g. ECGs, vital signs) that are judged by the Investigator as clinically significant will be recorded as AEs or SAEs if they meet the definition of an AE. The Investigator will exercise his or her medical and scientific judgment in deciding whether an abnormal laboratory finding or other abnormal assessment is clinically significant.
Measured: Between Screening to End of Study, up to 28 weeks
CDM1-RS Independent Central Rater Score (CDM1-RS)
Change from baseline to end of treatment in the independent central rater CDM1-RS total score. CDM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity that are clinically relevant in CDM1.
The severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.
Measured: Baseline to week 20
CGI-I Independent Central Rater Score (CGI-I)
The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state on a seven point scale.
A 7-point Likert type scale is used with ratings of 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.
Measured: Baseline and week 20
Independent Rater Clinical Global Impression - Severity Scale (CGI-S)
CGI-S is a 7-point Likert type scale. An independent central rater rated the CGI-S scales for both the in-clinic and telehealth interviews.
Subjects are assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
Measured: Baseline and week 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2018
- Study start3 Mar 2021
- Primary completion4 Apr 2023
- Study completion4 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- AMO Pharma Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joseph P Horrigan, MD, Study director, AMO Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03692312
- Sponsor's protocol ID
- AMO-02-MD-2-003
- Other IDs
- 2016-004623-23, 2016
- Status last verified
- Sep 2025
- First submitted
- 16 Mar 2018
- Last updated
- 8 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)
- ClinicalTrials.gov ID
- NCT03692312
- Acronym / sponsor ID
- AMO-02-MD-2-003
- Conditions
- Congenital Myotonic Dystrophy
- Keywords
- Tideglusib, AMO-02-MD-2-003, Congenital Myotonic Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Myotonia Atrophica, Myotonia Dystrophica, Myotonic Dystrophy, Congenital
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- AMO Pharma LimitedIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2021 (actual)
- Primary completion
- 4 Apr 2023 (actual)
- Study completion
- 4 Apr 2023 (actual)
- First posted
- 2 Oct 2018 (actual)
- Results
- Posted 11 Sep 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
Detailed description
This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tideglusib | Experimental | Drug: Tideglusib, Drug: PlaceboWeight adjusted tideglusib, orally, once daily |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo, orally, once daily |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- Male or female children and adolescents aged ≥6 years and ≤16 years
- Diagnosis of Congenital DM1 (also known as Steinert's disease)
- Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
- Written, voluntary informed consent must be obtained before any study related procedures are conducted.
- Subject's caregiver must be willing and able to support participation for duration of study
Key exclusion criteria (8 listed)
- Not able to walk; (full time wheel chair use)
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or change in medications/therapies within 4 weeks prior to Screening
- Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
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 |
|---|---|---|
| TideglusibTideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily | Drug | Tideglusib |
| PlaceboMatching placebo formulation | Drug | Placebo, Tideglusib |
Primary outcome
Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Time frame: Baseline and week 20
The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1. The severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.
10 secondary outcomes
Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores
Time frame: Baseline and week 20
Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score
Time frame: Baseline and week 20
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
Time frame: Baseline and week 20
Clinical Global Impression - Severity Scale (CGI-S)
Time frame: Baseline and week 20
10-meter Walk-run Test
Time frame: 20 weeks
Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study.
Time frame: Between Screening to End of Study, up to 28 weeks
Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study.
Time frame: Between Screening to End of Study, up to 28 weeks
CDM1-RS Independent Central Rater Score (CDM1-RS)
Time frame: Baseline to week 20
CGI-I Independent Central Rater Score (CGI-I)
Time frame: Baseline and week 20
Independent Rater Clinical Global Impression - Severity Scale (CGI-S)
Time frame: Baseline and week 20
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Joseph P Horrigan, MD study director
AMO Pharma
14 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Tideglusib in people with Congenital Myotonic Dystrophy, run by AMO Pharma Limited at 14 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada, Australia, New Zealand, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Sep 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) (time frame: Baseline and week 20). It is what the study was designed to measure, not a finding.
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 8 Oct 2025.