Myotonic Dystrophy 1Phase 2NCT04886518
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
A Phase 2 study testing Pitolisant Oral Tablet in people with Myotonic Dystrophy 1 and Excessive Daytime Sleepiness, run by Harmony Biosciences Management, Inc. at 13 sites in 2 countries.
Trial summary
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myotonic Dystrophy 1, Excessive Daytime Sleepiness
- What is being tested?
- Pitolisant Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 13 sitesin 2 countries
- Study dates
- 28 Jun 2021to 31 Oct 2024
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Is able to provide voluntary, written informed consent.
- Has a diagnosis of DM1 confirmed by genetic testing (cytosine-thymine-guanine [CTG] repeat of ≥100) from the Screening Visit.
- Male or female patients ages 18 to 65 years at the time of enrollment.
- Has a Clinical Global Impression of Severity (CGI-S) assessment of moderate or severe for overall severity of EDS at Screening.
- If on a wake-promoting treatment that could affect EDS (including stimulants, modafinil, and armodafinil):
- Must be on a stable dose for at least 2 months prior to Screening and agree to continue the stable dose for the duration of the Double-Blind Treatment Phase of the study (dose adjustments will be permitted in the OLE Phase).
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- If not on a stable dose for 2 months prior to Screening, washout for 5 half-lives prior to randomization and agree to remain off these treatments for the duration of the Double-Blind Treatment Phase of the study.
- Washout of cannabidiol and tetrahydrocannabinol for 28 days prior to randomization and agree to remain off for the duration of the Double-Blind Treatment Phase of the study.
- Able to walk independently with or without an assistive device (e.g., cane, walker, orthoses allowed).
- A patient who is a female of child-bearing potential (FCBP) must have a negative serum pregnancy test at the Screening Visit and negative urine pregnancy test at the Baseline Visit and agree to remain abstinent or use an effective method of non-hormonal contraception to prevent pregnancy for the duration of the study and for 21 days after final dose of study drug.
- In the opinion of the Investigator, the patient is capable of understanding and complying with the protocol and administration of oral study drug.
Who cannot join
- Has a diagnosis of another genetic or chromosomal disorder that is distinct from DM1 and that is not being managed adequately in the opinion of the Investigator.
- Experiences <6 hours on average of sleep per night based on their sleep diary during Screening (patients need to record at least 7 of 10 consecutive nights including 2 nights that fall on a weekend in their sleep diary during Screening).
- Consistently consumes >600 mg of caffeine per day and is unable/unwilling to reduce caffeine intake to <600 mg per day for the duration of the Double-Blind Treatment Phase of the study; caffeine intake should remain consistent during Screening and throughout the Double-Blind Treatment Phase of the study.
- Does not agree to discontinue any prohibited medication or substances listed in the protocol.
- Is currently breastfeeding or planning to breastfeed over the course of the study. Lactating women must agree not to breastfeed for the duration of the study (Double-Blind Treatment Phase and OLE Phase) and for 21 days after final dose of study drug.
- Participation in an interventional research study involving another investigational medication or device in the 28 days prior to enrollment; patients who undergo a washout of an investigational medication of at least 5 half-lives can be enrolled in the Double-Blind Treatment Phase of the study. Patients considering participation in another interventional research study in the OLE Phase must consult with the Investigator who will consult with the Medical Monitor.
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- Has a primary diagnosis of severe psychiatric illness.
- Patients taking antidepressants who have not been on a stable dose of their antidepressant for at least 12 weeks prior to Screening; for patients on a stable dose of their antidepressant for at least 12 weeks prior to Screening, must agree to continue their stable dose for the duration of the Double-Blind Treatment Phase of the study. Dose adjustments will be permitted in the OLE Phase. In the Double-Blind Treatment Phase of the study, antidepressants that are strong CYP2D6 inhibitors are exclusionary.
- Has a history of sleep-disordered breathing or another underlying sleep disorder that in the opinion of the Investigator is a main contributory factor to the patient's EDS.
- Has a diagnosis of end-stage renal disease (ESRD; estimated glomerular filtration rate [eGFR] of <15 mL/minute/1.73 m2) or severe hepatic impairment (Child-Pugh C).
- Has a diagnosis of moderate or severe renal impairment (eGFR ≥15 to ≤59 mL/minute/1.73 m2) or moderate hepatic impairment (Child-Pugh B) at Screening or during the Double-Blind Treatment Phase.
- Has a family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member (i.e., first degree relative such as parent, sibling, or offspring).
- Has a history of unexplained syncope.
- Has a history of long corrected QT interval (QTc) syndrome or corrected QT interval using Fridericia's formula (QTcF) >450 msec for males or >470 msec for females (QTcF = QT / 3√ RR) sustained atrial fibrillation (AF) or left ventricular ejection fraction <50%.
- Has a history of documented symptomatic arrhythmias (e.g., ECG, Holter monitor).
- Electrocardiogram abnormalities during a 10-second, 12-lead ECG at Screening of first degree atrioventricular block (AVB; PR interval >220 msec), QRS >120 msec, heart rate (HR) <50 beats per minute (bpm), marked T-wave abnormalities, more than single atrial premature complexes (APCs) or premature ventricular contractions (PVCs), left bundle branch block, or Brugada pattern type 1.
- Note: Patients with 1st degree AVB with a PR interval >220 msec, who are treated prophylactically with an allowable implanted device are not excluded from the study.
- Based on Holter monitor, any episode of 3rd degree AVB, any prolonged episode of second degree AVB (>2 episodes during waking hours, >6 episodes during sleep), any prolonged episode of 2nd degree AVB (>10 seconds), any asystole longer than 3.5 seconds, any run of ventricular tachycardia (VT) >6 beats, frequent runs of non-sustained VT (>5/24 hour), >400 PVCs/24 hours, AF or paroxysmal AF, or frequent or complex atrial arrhythmias.
- Has history of New York Heart Association (NYHA) class III or class IV heart failure.
- Has an implanted defibrillator or implanted biventricular pacemaker. Note: Patients with implanted univentricular pacemakers that are used prophylactically to prevent or treat bradycardia or heart block may be included.
- Is receiving a medication known to prolong the QT interval.
- Has a history of clinically significant hypokalemia or hypomagnesemia that cannot be adequately controlled by supplementation.
- Has serum potassium or magnesium levels that are outside of the normal reference ranges and considered clinically significant at Screening. Patients with mild hyperkalemia that, in the opinion of the Investigator, does not pose an arrhythmia threat may be included.
- Is receiving a concomitant medication that is known to be a strong cytochrome P450 (CYP) 2D6 inhibitor, a strong CYP3A4 inducer; or a centrally acting histamine 1 receptor (H1R) antagonist (sedating antihistamine).
- Note: Patients who undergo a washout of these medications of at least 5 half-lives may be enrolled in the Double-Blind Treatment Phase of the study.
- Note: Use of strong CYP2D6 inhibitors and strong CYP3A4 inducers is allowed during the OLE Phase; however, adjustment of pitolisant dose is required. Although not prohibited during the OLE Phase of the study, use of centrally acting or sedating H1R antagonists should be avoided.
- Is a known CYP2D6 poor metabolizer (PM).
- Regular use (more than twice per week) of any sleep-promoting treatments that could affect EDS and not willing to limit use to no more than twice per week during Screening and for the duration of the Double-Blind Treatment Phase of the study (use of sleep-promoting agents are not allowed within one day prior to study-related assessments).
- Has abnormal laboratory values at Screening that are clinically significant as determined by the Investigator.
- Has initiated any new or change in allied health therapies or interventions that can interfere with the study outcomes within 28 days prior to randomization and that are prohibited during the Double-Blind Treatment Phase of the study, based on the Investigator's judgment.
- Has a current or recent (within 1 year) history of a substance use disorder or dependence disorder, including alcohol and caffeine use disorders as defined in the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-V).
- Has planned surgery during the Double-Blind Treatment Phase of the study; planned surgery is permitted during the OLE Phase.
- Has a significant risk of committing suicide or suicidality based on history, routine psychiatric examination, Investigator's judgment, or who has an answer of "yes" on any question other than questions 1 to 3 on the Columbia-Suicide Severity Rating Scale.
- Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to an unstable or uncontrolled medical condition or one that might interfere with the conduct of the study, confound interpretation of study results, pose a health risk to the patient, or compromise the integrity of the study.
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States11 sites
Rare Disease Research
Atlanta, Georgia, 30329
University of Colorado School of Medicine
Aurora, Colorado, 80045
Kennedy Krieger Institute Center for Genetic Muscle Disorders
Baltimore, Maryland, 21205
University of North Carolina
Chapel Hill, North Carolina, 27599
Indiana University
Indianapolis, Indiana, 46202
UCI Center for Clinical Research
Irvine, California, 92697
University of Minnesota
Minneapolis, Minnesota, 55455
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Rochester Medical Center
Rochester, New York, 14642
University of South Florida
Tampa, Florida, 33612
Wake Forest
Winston-Salem, North Carolina, 27157
Canada2 sites
Hôpital de Chicoutimi
Chicoutimi, Quebec
The Ottawa Hospital
Ottawa, Ontario
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
Read the detailed description
The study will consist of a Screening Period, an 11-week Double-Blind Treatment Phase (including a 3-week Titration Period and an 8-week Stable Dose Period), and an optional Open Label Extension (OLE) Phase. The OLE Phase will last approximately one year for each patient or until the Sponsor elects to terminate the study.
Approximately 30 patients ages 18 to 65 years who meet all eligibility criteria will be randomized at the Baseline Visit in a 1:1:1 ratio to lower dose pitolisant, higher dose pitolisant, or matching placebo. In the Double-Blind Treatment Phase, patients will be titrated to their randomized stable dose of study drug during the 3-week Titration Period.
After completion of the 3-week Titration Period, patients will continue to take study drug at their randomized stable dose once daily in the morning upon wakening for an additional 8 weeks of blinded treatment (Stable Dose Period). The duration of the Double-Blind Treatment Phase will be 11 weeks.
Following the 11-week Double-Blind Treatment Phase, eligible patients will be given the opportunity to participate in an optional OLE Phase. During the OLE Phase, all eligible patients will receive treatment with open-label pitolisant. Patients will first undergo a 3-week Titration Period to a maximum target dose, after which they will continue to take their dose of pitolisant once daily in the morning upon wakening until the end of the study. The patient's dose of pitolisant may be adjusted during the OLE phase.
- Conditions
- Myotonic Dystrophy 1, Excessive Daytime Sleepiness
- Being tested
- Pitolisant Oral Tablet, Placebo oral tablet
- Medical subject headings
- Myotonic Dystrophy, Disorders of Excessive Somnolence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pitolisant Oral TabletDrug
Pitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 mm in diameter. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant.
Pitolisant 17.8 mg tablets: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant.
Placebo oral tabletDrug
Matching placebo tablets will be provided for each strength of active pitolisant film-coated tablets.
| Study group | Role | What they receive |
|---|---|---|
| Higher dose pitolisant | Active comparator | Double-Blind Treatment Phase:
Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 35.6 mg pitolisant administered once daily in the morning. Receives: Drug: Pitolisant Oral Tablet |
| Lower dose pitolisant | Active comparator | Double-Blind Treatment Phase:
Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning Receives: Drug: Pitolisant Oral Tablet |
| Placebo | Placebo comparator | Double-Blind Treatment Phase:
Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Receives: Drug: Placebo oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score
The score of the DSS ranges from 0 to 15. A decrease in the DSS score represents an improvement in EDS.
Measured: Baseline to Week 11
Secondary outcomes (9)
Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score
The score of the FSS ranges from 0 to 63. A decrease in the FSS score represents an improvement in fatigue.
Measured: Baseline to Week 11
Change in Psychomotor Function Based on Change in Cogstate Detection Test
The Cogstate Detection Test is a computerized test. A faster speed represents an improvement in psychomotor test performance.
Measured: Baseline to Week 11
Change in Attention Based on Change in Cogstate Identification Test
The Cogstate Identification Test is a computerized test. A faster speed represents an improvement in attention test performance.
Measured: Baseline to Week 11
Change in Working Memory Based on Change in Cogstate One Back Test
The Cogstate One Back Test is a computerized test. A faster speed represents a better working memory test performance.
Measured: Baseline to Week 11
Change in Burden of Disease Based on Change in Myotonic Dystrophy Health Index (MDHI)
The MDHI score ranges from 0 to 100. A decrease in the MDHI score represents an improvement in overall burden of disease.
Measured: Baseline to Week 11
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Epworth Sleepiness Scale (ESS) Score
The score of the ESS ranges from 0 to 24. A decrease in the ESS score represents an improvement in EDS.
Measured: Baseline to Week 11
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Clinical Global Impression of Severity (CGI-S)
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.
Measured: Baseline to Week 11
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Patient Global Impression of Severity (PGI-S)
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.
Measured: Baseline to Week 11
Change in Sustained Attention Based on Sustained Attention to Response Task (SART)
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.
Measured: Baseline to Week 11
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2021
- Study start28 Jun 2021
- Primary completion6 Oct 2023
- Study completion31 Oct 2024
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myotonic Dystrophy 1Studies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04886518
- Sponsor's protocol ID
- HBS-101-CL-005
- Status last verified
- Aug 2026
- First submitted
- 10 May 2021
- Last updated
- 4 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
D'Ambrosio ES, Chuang K, David WS, Amato AA, Gonzalez-Perez P. Frequency and type of cancers in myotonic dystrophy: A retrospective cross-sectional study. Muscle Nerve. 2023 Aug;68(2):142-148. doi: 10.1002/mus.27801. Epub 2023 Mar 27.
PubMed 36790141
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension
- ClinicalTrials.gov ID
- NCT04886518
- Acronym / sponsor ID
- HBS-101-CL-005
- Conditions
- Myotonic Dystrophy 1, Excessive Daytime Sleepiness
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Harmony Biosciences Management, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Jun 2021 (actual)
- Primary completion
- 6 Oct 2023 (actual)
- Study completion
- 31 Oct 2024 (actual)
- First posted
- 14 May 2021 (actual)
- Results
- Posted 25 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
Detailed description
The study will consist of a Screening Period, an 11-week Double-Blind Treatment Phase (including a 3-week Titration Period and an 8-week Stable Dose Period), and an optional Open Label Extension (OLE) Phase. The OLE Phase will last approximately one year for each patient or until the Sponsor elects to terminate the study.
Approximately 30 patients ages 18 to 65 years who meet all eligibility criteria will be randomized at the Baseline Visit in a 1:1:1 ratio to lower dose pitolisant, higher dose pitolisant, or matching placebo. In the Double-Blind Treatment Phase, patients will be titrated to their randomized stable dose of study drug during the 3-week Titration Period.
After completion of the 3-week Titration Period, patients will continue to take study drug at their randomized stable dose once daily in the morning upon wakening for an additional 8 weeks of blinded treatment (Stable Dose Period). The duration of the Double-Blind Treatment Phase will be 11 weeks.
Following the 11-week Double-Blind Treatment Phase, eligible patients will be given the opportunity to participate in an optional OLE Phase. During the OLE Phase, all eligible patients will receive treatment with open-label pitolisant. Patients will first undergo a 3-week Titration Period to a maximum target dose, after which they will continue to take their dose of pitolisant once daily in the morning upon wakening until the end of the study. The patient's dose of pitolisant may be adjusted during the OLE phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Higher dose pitolisant | Active comparator | Drug: Pitolisant Oral TabletDouble-Blind Treatment Phase: Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 35.6 mg pitolisant administered once daily in the morning. |
| Lower dose pitolisant | Active comparator | Drug: Pitolisant Oral TabletDouble-Blind Treatment Phase: Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning |
| Placebo | Placebo comparator | Drug: Placebo oral tabletDouble-Blind Treatment Phase: Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Is able to provide voluntary, written informed consent.
- Has a diagnosis of DM1 confirmed by genetic testing (cytosine-thymine-guanine [CTG] repeat of ≥100) from the Screening Visit.
- Male or female patients ages 18 to 65 years at the time of enrollment.
- Has a Clinical Global Impression of Severity (CGI-S) assessment of moderate or severe for overall severity of EDS at Screening.
- If on a wake-promoting treatment that could affect EDS (including stimulants, modafinil, and armodafinil):
Key exclusion criteria (34 listed)
- Has a diagnosis of another genetic or chromosomal disorder that is distinct from DM1 and that is not being managed adequately in the opinion of the Investigator.
- Experiences <6 hours on average of sleep per night based on their sleep diary during Screening (patients need to record at least 7 of 10 consecutive nights including 2 nights that fall on a weekend in their sleep diary during Screening).
- Consistently consumes >600 mg of caffeine per day and is unable/unwilling to reduce caffeine intake to <600 mg per day for the duration of the Double-Blind Treatment Phase of the study; caffeine intake should remain consistent during Screening and throughout the Double-Blind Treatment Phase of the study.
- Does not agree to discontinue any prohibited medication or substances listed in the protocol.
- Is currently breastfeeding or planning to breastfeed over the course of the study. Lactating women must agree not to breastfeed for the duration of the study (Double-Blind Treatment Phase and OLE Phase) and for 21 days after final dose of study drug.
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 |
|---|---|---|
| Pitolisant Oral TabletPitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 mm in diameter. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant. Pitolisant 17.8 mg tablets: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant. | Drug | Higher dose pitolisant, Lower dose pitolisant |
| Placebo oral tabletMatching placebo tablets will be provided for each strength of active pitolisant film-coated tablets. | Drug | Placebo |
Primary outcome
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score
Time frame: 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.
9 secondary outcomes
Change in Fatigue Based on Change in Fatigue Severity Scale (FSS) Score
Time frame: Baseline to Week 11
Change in Psychomotor Function Based on Change in Cogstate Detection Test
Time frame: Baseline to Week 11
Change in Attention Based on Change in Cogstate Identification Test
Time frame: Baseline to Week 11
Change in Working Memory Based on Change in Cogstate One Back Test
Time frame: Baseline to Week 11
Change in Burden of Disease Based on Change in Myotonic Dystrophy Health Index (MDHI)
Time frame: Baseline to Week 11
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Epworth Sleepiness Scale (ESS) Score
Time frame: Baseline to Week 11
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Clinical Global Impression of Severity (CGI-S)
Time frame: Baseline to Week 11
Change in Excessive Daytime Sleepiness (EDS) Based on Change in Patient Global Impression of Severity (PGI-S)
Time frame: Baseline to Week 11
Change in Sustained Attention Based on Sustained Attention to Response Task (SART)
Time frame: Baseline to Week 11
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36790141D'Ambrosio ES, Chuang K, David WS, Amato AA, Gonzalez-Perez P. Frequency and type of cancers in myotonic dystrophy: A retrospective cross-sectional study. Muscle Nerve. 2023 Aug;68(2):142-148. doi: 10.1002/mus.27801. Epub 2023 Mar 27.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pitolisant Oral Tablet in people with Myotonic Dystrophy 1 and Excessive Daytime Sleepiness, run by Harmony Biosciences Management, Inc. at 13 sites in 2 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 11 inclusion and 34 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 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Canada. 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 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.
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 25 Apr 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 Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score (time frame: Baseline to Week 11). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04886518, which also lists 1 linked publication.
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 4 Sep 2026.