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Higher dose pitolisant for Myotonic Dystrophy
Study Summary
This trial is testing a new drug to see if it's safe and effective in treating excessive daytime sleepiness in patients with Myotonic Dystrophy Type 1.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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- I haven't used CBD or THC for 28 days and won't use them during the study.I have had fainting spells that doctors couldn't explain.Your symptoms of excessive daytime sleepiness (EDS) are moderate to severe according to a screening assessment called Clinical Global Impression of Severity (CGI-S).You have a serious mental illness as your main medical condition.You have been assessed by a doctor and have been found to have moderate to severe excessive daytime sleepiness.You have had problems with drug or alcohol addiction within the past year.I understand and can follow the study's requirements.You understand the study and agree to participate by signing a written consent form.A family member has died suddenly or from heart rhythm problems.I haven't used CBD or THC for 28 days and agree not to use them during the trial.I have a genetic disorder other than DM1 that is not well-managed.I am taking medication that strongly affects how my body processes drugs.I've been on a stable dose of antidepressants for at least 12 weeks.My genetic test shows I have DM1 with a CTG repeat of 100 or more.I have had serious low potassium or magnesium levels that supplements can't fix.I have a history of heart rhythm problems that caused symptoms.I am between 18 and 65 years old.I have been on a stable dose of wake-promoting treatment for at least 2 months.I haven't changed my medication dose for 2 months and can stop certain medications if needed.I have a history of severe heart failure.I haven't started or changed any health therapies that could affect the study in the last 28 days.I am a poor metabolizer for the CYP2D6 enzyme.I am not pregnant, will not become pregnant, and agree to use birth control during the study.I haven't been in a drug/device study in the last 28 days or have completed the required washout period.I can walk on my own, with or without help from a device.I am between 18 and 65 years old.I have been genetically confirmed to have DM1 with a CTG repeat of 100 or more.I haven't changed my medication dose for 2 months, or I can stop taking it for its washout period before the study starts.I am not pregnant, can test to confirm, and will avoid pregnancy during the study.I have severe kidney disease or severe liver impairment.I use sleep aids no more than twice a week.I do not have a history of heart rhythm problems or a weak heart.You have a high risk of hurting yourself or committing suicide based on your medical history or routine psychiatric evaluation.I am scheduled for surgery during the study.I have been on a stable medication dose for 2 months and agree to keep it the same during the study.I am taking medication that affects my heart's rhythm.I have moderate to severe kidney or moderate liver problems.You drink more than six cups of coffee a day and are not willing to cut back to less than six cups a day during the study.I can walk on my own, with or without help from a device.You have a pacemaker or defibrillator implanted in your body.I have a sleep disorder that mainly causes my excessive daytime sleepiness.
- Group 1: Placebo
- Group 2: Higher dose pitolisant
- Group 3: Lower dose pitolisant
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
What indications are typically seen for the administration of Low dose pitolisant?
"Low dose pitolisant can help address the issues of sleeping too much during the day, narcolepsy with cataplexy, and general narcolepsy."
What safety considerations should be taken into account when administering low dose pitolisant?
"There is limited evidence to corroborate the safety of Low dose pitolisant, so it was given a rating of 2 on our scale. This assessment should be taken with caution since no studies have been conducted to assess its efficacy."
Am I eligible to join in the research of this trial?
"This clinical trial is recruiting 78 individuals aged 18 to 65 with acute excessive daytime sleepiness. The requirements are: providing voluntary, written informed consent; a confirmed diagnosis of DM1 through genetic testing (CTG repeat ≥100); being on a stable dose for at least 2 months prior or 5 half-lives washout before randomization; cannabidiol and tetrahydrocannabinol must be washed out for 28 days prior to randomisation; female patients must have negative pregnancy tests upon screening and baseline visits in addition to using effective birth control until 21 days after the last study drug administration; able to understand and comply with the protocol"
What is the maximum capacity of participants for this trial?
"For this research endeavour, a total of 78 volunteers that meet the established criteria are required. These individuals may join from either Wake Forest in Winston-Salem NC or University of Kansas Medical Centre situated in Kansas City KS."
Does this medical trial represent a groundbreaking advancement in the field?
"At present, Harmony Biosciences, LLC is sponsoring two active trials of Low dose pitolisant across 71 cities and 2 countries. The initial trial commenced in 2021 with 78 participants and successfully passed Phase 2 drug approval. Since its initiation there have been 82 separate studies conducted on this medication."
How many healthcare facilities are conducting this experiment?
"This medical trial is being conducted at various locations, such as Wake Forest in Winston-Salem, North carolina, UCI Center for Clinical Research in Irvine California and University of Kansas Medical Centre in Kansas City. There are an additional 19 sites taking part."
What other exploratory studies have been conducted regarding Low dose pitolisant?
"Pitolisant, a low dose drug, was first evaluated in 2021 at Ohio Sleep Medicine Institute. A total of 82 studies have been conducted and there are currently 2 trials actively seeking patients, with many being held in Winston-Salem, North carolina."
Is the eligibility criteria of this research study inclusive to those aged above forty-five?
"In accordance with the protocol, only people aged 18 to 65 are suitable for enrollment in this trial."
Is this medical research currently enrolling participants?
"Affirmative. According to the records available on clinicaltrials.gov, this medical trial which was first published on June 28th 2021 is actively looking for potential participants. The study requires 78 people from 19 distinct sites across the globe."
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