Trial · NCT04886518
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
NCT04886518, Phase 2, Completed, Harmony Biosciences Management, Inc.; results first posted 2025-04-25
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1. Trials, record NCT04886518. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04886518
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
- NCT number
- NCT04886518
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Conditions
- Myotonic Dystrophy 1, Excessive Daytime Sleepiness
- Interventions
- Pitolisant Oral Tablet, Placebo oral tablet
- First posted
- 2021-05-14
- Last updated
- 2026-09-04
- Results first posted
- 2025-04-25
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score | Baseline to Week 11 | The score of the DSS ranges from 0 to 15. A decrease in the DSS score represents an improvement in EDS. |
| Secondary | Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score | Baseline to Week 11 | The score of the FSS ranges from 0 to 63. A decrease in the FSS score represents an improvement in fatigue. |
| Secondary | Change in Psychomotor Function Based on Change in Cogstate Detection Test | Baseline to Week 11 | The Cogstate Detection Test is a computerized test. A faster speed represents an improvement in psychomotor test performance. |
| Secondary | Change in Attention Based on Change in Cogstate Identification Test | Baseline to Week 11 | The Cogstate Identification Test is a computerized test. A faster speed represents an improvement in attention test performance. |
| Secondary | Change in Working Memory Based on Change in Cogstate One Back Test | Baseline to Week 11 | The Cogstate One Back Test is a computerized test. A faster speed represents a better working memory test performance. |
| Secondary | Change in Burden of Disease Based on Change in Myotonic Dystrophy Health Index (MDHI) | Baseline to Week 11 | The MDHI score ranges from 0 to 100. A decrease in the MDHI score represents an improvement in overall burden of disease. |
| Secondary | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Epworth Sleepiness Scale (ESS) Score | Baseline to Week 11 | The score of the ESS ranges from 0 to 24. A decrease in the ESS score represents an improvement in EDS. |
| Secondary | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Clinical Global Impression of Severity (CGI-S) | Baseline to Week 11 | The CGI-S is a one-item scale that ranges from 0 to 4 with 0 being no EDS and 4 being severe EDS. The scale is completed by the site investigator/clinician. The higher the CGI-S score, the more severe the clinician's perception of the patient's symptoms of EDS. |
| Secondary | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Patient Global Impression of Severity (PGI-S) | Baseline to Week 11 | PGI-S is a one-item scale that ranges from 0 to 4 with 0 being no EDS and 4 being severe EDS. The scale is completed by the patient. The higher the PGI-S score, the more severe the patient's perception of excessive daytime sleepiness. |
| Secondary | Change in Sustained Attention Based on Sustained Attention to Response Task (SART) | Baseline to Week 11 | The Sustained Attention to Response Test provides a measure of vigilance and sustained attention. A number ranging from 1 to 9 is presented on screen. The subject must press a predetermined button (the YES external button) as soon as the number is presented, except when the number is a 3. The number 3 is the default number (no-go stimulus) for which a response should be inhibited, and the subject should not press the predetermined button. The no-go stimulus can be configured to be a number other than 3. Performance is measured by calculating the number of errors made during the test over the course of 225 trials. Performance is measured by calculating the error score, which is the sum of trials where a response was provided when a 3 was presented and trials where no response was provided when a number other than 3 was presented. A decrease in the SART score represents an improvement in sustained attention. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline DSS assessment. | score on a scale | MEAN |
| SECONDARY | Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score | Baseline to Week 11 | The analysis population includes all randomized participants who received at least one dose of study drug and have on baseline and at least one post-baseline FSS assessment. | score on a scale | MEAN |
| SECONDARY | Change in Psychomotor Function Based on Change in Cogstate Detection Test | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline Cogstate assessment. | log10 ms | MEAN |
| SECONDARY | Change in Attention Based on Change in Cogstate Identification Test | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline Cogstate assessment. | log10 ms | MEAN |
| SECONDARY | Change in Working Memory Based on Change in Cogstate One Back Test | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline cogstate assessment. | log10 ms | MEAN |
| SECONDARY | Change in Burden of Disease Based on Change in Myotonic Dystrophy Health Index (MDHI) | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline MDHI assessment. | score on a scale | MEAN |
| SECONDARY | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Epworth Sleepiness Scale (ESS) Score | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline ESS assessment. | score on a scale | MEAN |
| SECONDARY | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Clinical Global Impression of Severity (CGI-S) | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline CGI-S assessment. | score on a scale | MEAN |
| SECONDARY | Change in Excessive Daytime Sleepiness (EDS) Based on Change in Patient Global Impression of Severity (PGI-S) | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline PGI-S assessment. | score on a scale | MEAN |
| SECONDARY | Change in Sustained Attention Based on Sustained Attention to Response Task (SART) | Baseline to Week 11 | This analysis population includes all randomized participants who received at least one dose of study drug and have one baseline and at least one post-baseline Sustained Attention to Response Task assessment. | scores on a scale | MEAN |
Source ClinicalTrials.gov
Results by group30
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score | Higher Dose Pitolisant | -3.8 | Standard Deviation 1.98 | score on a scale | |
| Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score | Lower Dose Pitolisant | -1.8 | Standard Deviation 1.91 | score on a scale | |
| Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score | Placebo | 0.0 | Standard Deviation 2.29 | score on a scale | |
| Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score | Higher Dose Pitolisant | -0.86 | Standard Deviation 1.487 | score on a scale | |
| Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score | Lower Dose Pitolisant | -0.36 | Standard Deviation 0.367 | score on a scale | |
| Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score | Placebo | -0.13 | Standard Deviation 0.529 | score on a scale | |
| Change in Psychomotor Function Based on Change in Cogstate Detection Test | Higher Dose Pitolisant | 0.02 | Standard Deviation 0.058 | log10 ms | |
| Change in Psychomotor Function Based on Change in Cogstate Detection Test | Lower Dose Pitolisant | -0.05 | Standard Deviation 0.061 | log10 ms | |
| Change in Psychomotor Function Based on Change in Cogstate Detection Test | Placebo | 0.01 | Standard Deviation 0.037 | log10 ms | |
| Change in Attention Based on Change in Cogstate Identification Test | Higher Dose Pitolisant | 0.00 | Standard Deviation 0.035 | log10 ms |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Higher Dose Pitolisant | 10 | |
| Overall Study | STARTED | Lower Dose Pitolisant | 10 | |
| Overall Study | STARTED | Placebo | 10 | |
| Overall Study | COMPLETED | Higher Dose Pitolisant | 8 | |
| Overall Study | COMPLETED | Lower Dose Pitolisant | 9 | |
| Overall Study | COMPLETED | Placebo | 10 | |
| Overall Study | NOT COMPLETED | Higher Dose Pitolisant | 2 | |
| Overall Study | NOT COMPLETED | Lower Dose Pitolisant | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 0 |
Source ClinicalTrials.gov
Adverse events141
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 | Headache | Nervous system disorders | Higher Dose Pitolisant | 5 | 10 | |
| other | Headache | Nervous system disorders | Lower Dose Pitolisant | 1 | 10 | |
| other | Headache | Nervous system disorders | Placebo | 3 | 10 | |
| other | Insomnia | Psychiatric disorders | Higher Dose Pitolisant | 1 | 10 | |
| other | Insomnia | Psychiatric disorders | Lower Dose Pitolisant | 4 | 10 | |
| other | Insomnia | Psychiatric disorders | Placebo | 3 | 10 | |
| other | Nausea | Gastrointestinal disorders | Higher Dose Pitolisant | 3 | 10 | |
| other | Nausea | Gastrointestinal disorders | Lower Dose Pitolisant | 1 | 10 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 1 | 10 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Higher Dose Pitolisant | 2 | 10 |
1 to 10 of 141Source ClinicalTrials.gov
See all 141 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Higher Dose Pitolisant | 0 | 10 |
| Total, all-cause mortality | Lower Dose Pitolisant | 0 | 10 |
| Total, all-cause mortality | Placebo | 0 | 10 |
| Total, other adverse events | Higher Dose Pitolisant | 8 | 10 |
| Total, other adverse events | Lower Dose Pitolisant | 7 | 10 |
| Total, other adverse events | Placebo | 8 | 10 |
| Total, serious adverse events | Higher Dose Pitolisant | 0 | 10 |
| Total, serious adverse events | Lower Dose Pitolisant | 0 | 10 |
| Total, serious adverse events | Placebo | 0 | 10 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36790141 | DERIVED |
Source PubMed
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests2
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| 1-Back Task | Secondary outcome | ConductCognition |
| Sustained Attention to Response | Secondary outcome | ConductCognition |
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04886518
GET https://conductscience.org/api/trials/v1/NCT04886518/results?page=2