Expanded Access Program to Provide Treatment With Pitolisant to Adult Patients in the U.S. With E… (NCT03433131) | Clinical Trial Compass
NO_LONGER_AVAILABLENot Applicable
Expanded Access Program to Provide Treatment With Pitolisant to Adult Patients in the U.S. With Excessive Daytime Sleepiness Associated With Narcolepsy With or Without Cataplexy
United States
Plain-language summary
This EAP will be open to provide access to treatment with pitolisant while a U.S. New Drug Application (NDA) is being prepared and submitted for review for marketing approval. This program will be open to adult patients in the U.S. with Excessive Daytime Sleepiness (EDS) associated with narcolepsy, with or without cataplexy. Pitolisant will be made available through treating physicians participating in the program.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. In the opinion of the treating physician, the patient is capable of understanding and complying with protocol and program requirements, oral drug administration and care instructions.
. The patient or, when applicable, the patient's legally acceptable representative, signs and dates a written, ICF and any required privacy authorization prior to the initiation of any program procedures.
. Men or women, 18 years of age and over.
. Patients with a diagnosis of narcolepsy with or without cataplexy according to the ICSD or DSM criteria at the time of diagnosis.
. Patients should be free of prohibited treatments or have discontinued them for at least 7 days prior to the start of treatments.
. Women of child-bearing potential must have a negative serum pregnancy test performed at the screening visit.
. Due to the effectiveness of hormonal contraceptives potentially being reduced when used with pitolisant, alternative or concomitant barrier methods of contraception are required for patients taking hormonal contraceptives (e.g., ethinyl estradiol) when taking pitolisant and for at least 21 days after discontinuation of pitolisant treatment.
Exclusion criteria
. The patient has severe hepatic impairment (Child-Pugh C).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. The patient is a woman who is breastfeeding or plans to breastfeed during their participation.
. The patient has, in the judgment of the treating physician, a history or current medical condition that could affect safety or poses an additional risk to the patient by their participation in the program.
. The patient is at high risk for suicide defined as a suicide attempt within the past year, significant risk determined by the investigator interview, or use of the Columbia Suicide Severity Rating Scale (C-SSRS).
. The patient has a history of hypersensitivity or allergic reaction to pitolisant or any inactive ingredient of the formulation.
. Current or recent (within one year) history of a substance abuse or dependence disorder including alcohol abuse as defined in Diagnostic and Statistical Manual of Mental Disorders (DSM-V).
. Patients with a known history of long QTc syndrome (e.g. syncope or arrhythmia) or any significant history of a serious abnormality of the ECG (e.g. recent myocardial infarction), or QTc Fridericia higher than 450 ms for male and 470 ms for female (electrocardiogram Fridericia's corrected QT interval = QT / 3√ RR).
. Significant abnormality in the physical examination or clinical laboratory results.