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

NCT ID: NCT03433131

Last Updated: 2019-08-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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

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Conditions

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Narcolepsy Without Cataplexy Narcolepsy With Cataplexy

Interventions

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Pitolisant

Tablets

Intervention Type DRUG

Other Intervention Names

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Wakix

Eligibility Criteria

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Inclusion Criteria

1. 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.
2. 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.
3. Men or women, 18 years of age and over.
4. Patients with a diagnosis of narcolepsy with or without cataplexy according to the ICSD or DSM criteria at the time of diagnosis.
5. Patients should be free of prohibited treatments or have discontinued them for at least 7 days prior to the start of treatments.
6. Women of child-bearing potential must have a negative serum pregnancy test performed at the screening visit.
7. 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

1. The patient has severe hepatic impairment (Child-Pugh C).
2. The patient is a woman who is breastfeeding or plans to breastfeed during their participation.
3. 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.
4. 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).
5. The patient has a history of hypersensitivity or allergic reaction to pitolisant or any inactive ingredient of the formulation.
6. 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).
7. 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).
8. Significant abnormality in the physical examination or clinical laboratory results.
9. Prior severe adverse reactions to CNS stimulants.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Harmony Biosciences Management, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Michael Thorpy, MD

Role: PRINCIPAL_INVESTIGATOR

Albert Einstein College of Medicine

Locations

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Sleep Disorders Center of Alabama

Birmingham, Alabama, United States

Site Status

CPC

Cullman, Alabama, United States

Site Status

Xenoscience

Phoenix, Arizona, United States

Site Status

Xenoscience

Phoenix, Arizona, United States

Site Status

Foothills Neurology

Phoenix, Arizona, United States

Site Status

Mayo Clinic

Scottsdale, Arizona, United States

Site Status

Sleep Medicine Specialists of California

San Ramon, California, United States

Site Status

Santa Monica Sleep Disorders Center

Santa Monica, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Colorado Sleep Institute

Boulder, Colorado, United States

Site Status

Norwalk Hospital Sleep Disorders Center

Norwalk, Connecticut, United States

Site Status

Clinical Neurosciences fo Tampa Bay

Clearwater, Florida, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

Sleep Medicine Specialists of South Florida, PA

Miami, Florida, United States

Site Status

Child Lung, Asthma Sleep Specialists

Winter Park, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

NeuroTrials Research Center

Atlanta, Georgia, United States

Site Status

The Neurological Center of North Georgia

Gainesville, Georgia, United States

Site Status

Critical Healthcare Decisions

Savannah, Georgia, United States

Site Status

Northwestern Medical Group

Chicago, Illinois, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

NorthShore Uni HealthSys-Glenbrook Hospital

Glenview, Illinois, United States

Site Status

Northshore Sleep Medicine

Northbrook, Illinois, United States

Site Status

Indiana Internal Medicine Consultants

Greenwood, Indiana, United States

Site Status

The University of Kansas Health System

Fairway, Kansas, United States

Site Status

Norton Pulmonary Specialists

Louisville, Kentucky, United States

Site Status

Maine Medical Partners - Neurology

Scarborough, Maine, United States

Site Status

The Center for Sleep and Wake Disorders

Chevy Chase, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

The University of Michigan

Ann Arbor, Michigan, United States

Site Status

Henry Ford Health System

Novi, Michigan, United States

Site Status

Bronson Sleep Health

Portage, Michigan, United States

Site Status

Alliance Health Shelby Family Medicine

Shelby, Michigan, United States

Site Status

Fairview Health Services

Brooklyn Park, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

St. Lukes Hospital, Sleep Medicine

Chesterfield, Missouri, United States

Site Status

University of Missouri

Columbia, Missouri, United States

Site Status

Saint Luke's Hospital of Kansas City

Kansas City, Missouri, United States

Site Status

Norwell Health

New Hyde Park, New York, United States

Site Status

NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center

New York, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

Unity Sleep Disorders Center

Rochester, New York, United States

Site Status

University of Rochester Sleep Center

Rochester, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Excel Psychiatric Associates, PA

Huntersville, North Carolina, United States

Site Status

Raleigh Neurology Assoc., PA

Raleigh, North Carolina, United States

Site Status

Neurology & Neuroscience Associates, Inc.

Akron, Ohio, United States

Site Status

Intrepid Research, LLC

Cincinnati, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Ohio Sleep Medicine Institute

Dublin, Ohio, United States

Site Status

Oklahoma Heart Institute

Tulsa, Oklahoma, United States

Site Status

Oklahoma Heart Institute

Tulsa, Oklahoma, United States

Site Status

Legacy Medical Group Sleep Medicine

Portland, Oregon, United States

Site Status

Collegeville Family Practice

Berwyn, Pennsylvania, United States

Site Status

Paoli Sleep Center

Paoli, Pennsylvania, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Maria J. Sunseri, MD, LLC

Pittsburgh, Pennsylvania, United States

Site Status

Abington Neurological Associates

Willow Grove, Pennsylvania, United States

Site Status

Respitory Specialists

Wyomissing, Pennsylvania, United States

Site Status

Brown Medicine/Rhode Island Hospital

West Warwick, Rhode Island, United States

Site Status

Bogan Sleep Consultants

Columbia, South Carolina, United States

Site Status

Sleep Medicine Associates of Texas

Dallas, Texas, United States

Site Status

Houston Neurology & Sleep Center

Houston, Texas, United States

Site Status

Sleep Therapy & Research Center

San Antonio, Texas, United States

Site Status

Comprehensive Sleep Medicine Associates

Sugar Land, Texas, United States

Site Status

Chalottesville Neurology and Sleep Medicine

Charlottesville, Virginia, United States

Site Status

Children's Hospital of The King's Daughters

Norfolk, Virginia, United States

Site Status

Eastern Virginia Medical School

Norfolk, Virginia, United States

Site Status

Stultz Sleep and Behavioral Health

Barboursville, West Virginia, United States

Site Status

Medical College of Wisonsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Related Links

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http://thepeacenarcolepsyprogram.com

Program website to provide information to both prosective patients and physicians

Other Identifiers

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HBS-101-CL-001

Identifier Type: -

Identifier Source: org_study_id

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