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
- Conditions
- Narcolepsy Without CataplexyNarcolepsy With Cataplexy
- Registration Number
- NCT03433131
- Lead Sponsor
- Harmony Biosciences, LLC
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
- 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.
- The patient has severe hepatic impairment (Child-Pugh C).
- 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.
- Prior severe adverse reactions to CNS stimulants.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (67)
St. Lukes Hospital, Sleep Medicine
🇺🇸Chesterfield, Missouri, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Northwestern Medical Group
🇺🇸Chicago, Illinois, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Sleep Medicine Specialists of South Florida, PA
🇺🇸Miami, Florida, United States
Intrepid Research, LLC
🇺🇸Cincinnati, Ohio, United States
CPC
🇺🇸Cullman, Alabama, United States
Sleep Medicine Specialists of California
🇺🇸San Ramon, California, United States
Norton Pulmonary Specialists
🇺🇸Louisville, Kentucky, United States
Santa Monica Sleep Disorders Center
🇺🇸Santa Monica, California, United States
Norwalk Hospital Sleep Disorders Center
🇺🇸Norwalk, Connecticut, United States
The Center for Sleep and Wake Disorders
🇺🇸Chevy Chase, Maryland, United States
Child Lung, Asthma Sleep Specialists
🇺🇸Winter Park, Florida, United States
Critical Healthcare Decisions
🇺🇸Savannah, Georgia, United States
NorthShore Uni HealthSys-Glenbrook Hospital
🇺🇸Glenview, Illinois, United States
Maine Medical Partners - Neurology
🇺🇸Scarborough, Maine, United States
The Neurological Center of North Georgia
🇺🇸Gainesville, Georgia, United States
The University of Kansas Health System
🇺🇸Fairway, Kansas, United States
Bronson Sleep Health
🇺🇸Portage, Michigan, United States
University of Missouri
🇺🇸Columbia, Missouri, United States
Indiana Internal Medicine Consultants
🇺🇸Greenwood, Indiana, United States
NeuroTrials Research Center
🇺🇸Atlanta, Georgia, United States
Northshore Sleep Medicine
🇺🇸Northbrook, Illinois, United States
The University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Henry Ford Health System
🇺🇸Novi, Michigan, United States
Alliance Health Shelby Family Medicine
🇺🇸Shelby, Michigan, United States
Norwell Health
🇺🇸New Hyde Park, New York, United States
Unity Sleep Disorders Center
🇺🇸Rochester, New York, United States
University of Rochester Sleep Center
🇺🇸Rochester, New York, United States
Raleigh Neurology Assoc., PA
🇺🇸Raleigh, North Carolina, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Excel Psychiatric Associates, PA
🇺🇸Huntersville, North Carolina, United States
NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center
🇺🇸New York, New York, United States
Columbia University
🇺🇸New York, New York, United States
Neurology & Neuroscience Associates, Inc.
🇺🇸Akron, Ohio, United States
Ohio Sleep Medicine Institute
🇺🇸Dublin, Ohio, United States
Maria J. Sunseri, MD, LLC
🇺🇸Pittsburgh, Pennsylvania, United States
Collegeville Family Practice
🇺🇸Berwyn, Pennsylvania, United States
Respitory Specialists
🇺🇸Wyomissing, Pennsylvania, United States
Bogan Sleep Consultants
🇺🇸Columbia, South Carolina, United States
Sleep Medicine Associates of Texas
🇺🇸Dallas, Texas, United States
Houston Neurology & Sleep Center
🇺🇸Houston, Texas, United States
Sleep Therapy & Research Center
🇺🇸San Antonio, Texas, United States
Stultz Sleep and Behavioral Health
🇺🇸Barboursville, West Virginia, United States
Saint Luke's Hospital of Kansas City
🇺🇸Kansas City, Missouri, United States
Oklahoma Heart Institute
🇺🇸Tulsa, Oklahoma, United States
Foothills Neurology
🇺🇸Phoenix, Arizona, United States
Sleep Disorders Center of Alabama
🇺🇸Birmingham, Alabama, United States
Medical College of Wisonsin
🇺🇸Milwaukee, Wisconsin, United States
Xenoscience
🇺🇸Phoenix, Arizona, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Legacy Medical Group Sleep Medicine
🇺🇸Portland, Oregon, United States
Paoli Sleep Center
🇺🇸Paoli, Pennsylvania, United States
Clinical Neurosciences fo Tampa Bay
🇺🇸Clearwater, Florida, United States
Colorado Sleep Institute
🇺🇸Boulder, Colorado, United States
Fairview Health Services
🇺🇸Brooklyn Park, Minnesota, United States
Abington Neurological Associates
🇺🇸Willow Grove, Pennsylvania, United States
Brown Medicine/Rhode Island Hospital
🇺🇸West Warwick, Rhode Island, United States
Chalottesville Neurology and Sleep Medicine
🇺🇸Charlottesville, Virginia, United States
Children's Hospital of The King's Daughters
🇺🇸Norfolk, Virginia, United States
Comprehensive Sleep Medicine Associates
🇺🇸Sugar Land, Texas, United States
Eastern Virginia Medical School
🇺🇸Norfolk, Virginia, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States