Trial · NCT03692312
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
NCT03692312, Phase 2, Phase 3, Completed, AMO Pharma Limited; results first posted 2025-09-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy. Trials, record NCT03692312. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03692312
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
- NCT number
- NCT03692312
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 56 participants, actual
- Lead sponsor
- AMO Pharma Limited
- Conditions
- Congenital Myotonic Dystrophy
- Interventions
- Tideglusib, Placebo
- First posted
- 2018-10-02
- Last updated
- 2025-10-08
- Other registries' IDs
- eudract:2016-004623-23
- Results first posted
- 2025-09-11
- Limitations and caveats
- None reported
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) | 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. |
| Secondary | Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores | Baseline and week 20 | 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. |
| Secondary | Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score | Baseline and week 20 | 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. |
| Secondary | Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) | Baseline and week 20 | 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. |
| Secondary | Clinical Global Impression - Severity Scale (CGI-S) | Baseline and week 20 | 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. |
| Secondary | 10-meter Walk-run Test | 20 weeks | 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. |
| Secondary | Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study. | Between Screening to End of Study, up to 28 weeks | Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician. |
| Secondary | Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study. | Between Screening to End of Study, up to 28 weeks | 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. |
| Secondary | CDM1-RS Independent Central Rater Score (CDM1-RS) | Baseline to week 20 | 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. |
| Secondary | CGI-I Independent Central Rater Score (CGI-I) | Baseline and week 20 | 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. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) | Baseline and week 20 | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores | Baseline and week 20 | A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score | Baseline and week 20 | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) | Baseline and week 20 | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Clinical Global Impression - Severity Scale (CGI-S) | Baseline and week 20 | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | 10-meter Walk-run Test | 20 weeks | Seconds | LEAST_SQUARES_MEAN | |
| SECONDARY | Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study. | Between Screening to End of Study, up to 28 weeks | A participant assigned to placebo was found to have some tideglusib levels (due to inadvertent exposure between siblings in opposing arms), and was therefore, included in the safety analysis and full analysis sets for tideglusib. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study. | Between Screening to End of Study, up to 28 weeks | A participant assigned to placebo was found to have some tideglusib levels (due to inadvertent exposure between siblings in opposing arms), and was therefore, included in the safety analysis and full analysis sets for tideglusib. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | CDM1-RS Independent Central Rater Score (CDM1-RS) | Baseline to week 20 | A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | CGI-I Independent Central Rater Score (CGI-I) | Baseline and week 20 | A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment. | score on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group73
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) | Tideglusib | -1.65 | Standard Error 0.620 | score on a scale | |
| Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) | Placebo | -3.40 | Standard Error 0.618 | score on a scale | |
| Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores | Tideglusib | 3.11 | Standard Error 0.190 | score on a scale | |
| Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores | Placebo | 2.77 | Standard Error 0.189 | score on a scale | |
| Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score | Tideglusib | -1.74 | Standard Error 0.780 | score on a scale | |
| Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score | Placebo | -6.47 | Standard Error 0.780 | score on a scale | |
| Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) | Tideglusib | -0.18 | Standard Error 0.770 | score on a scale | |
| Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) | Placebo | -3.36 | Standard Error 0.782 | score on a scale | |
| Clinical Global Impression - Severity Scale (CGI-S) | Tideglusib | -0.20 | Standard Error 0.076 | score on a scale | |
| Clinical Global Impression - Severity Scale (CGI-S) | Placebo | -0.12 | Standard Error 0.076 | score on a scale |
1 to 10 of 73Source ClinicalTrials.gov
See all 73 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tideglusib | 27 | |
| Overall Study | STARTED | Placebo | 26 | |
| Overall Study | COMPLETED | Tideglusib | 25 | |
| Overall Study | COMPLETED | Placebo | 25 | |
| Overall Study | NOT COMPLETED | Tideglusib | 2 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events32
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nasopharyngytitis | Infections and infestations | Tideglusib | 3 | 28 | 4 |
| other | Nasopharyngytitis | Infections and infestations | Placebo | 3 | 25 | 3 |
| other | Upper respiratory tract infection | Infections and infestations | Tideglusib | 3 | 28 | 4 |
| other | Upper respiratory tract infection | Infections and infestations | Placebo | 2 | 25 | 2 |
| other | COVID-19 | Infections and infestations | Tideglusib | 2 | 28 | 2 |
| other | COVID-19 | Infections and infestations | Placebo | 1 | 25 | 1 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Tideglusib | 4 | 28 | 4 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Placebo | 0 | 25 | 0 |
| other | Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Tideglusib | 3 | 28 | 3 |
| other | Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Placebo | 1 | 25 | 1 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tideglusib | 0 | 28 |
| Total, all-cause mortality | Placebo | 0 | 25 |
| Total, other adverse events | Tideglusib | 20 | 28 |
| Total, other adverse events | Placebo | 19 | 25 |
| Total, serious adverse events | Tideglusib | 1 | 28 |
| Total, serious adverse events | Placebo | 0 | 25 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Tideglusib vs Placebo: deaths from any cause | Trial comparisons |
| Tideglusib vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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