Clinical trial NCT03433131: 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
Official title: An Open Label Expanded Access Program Intended to Provide Treatment With HBS-101 (Pitolisant) to Adult Patients in the U.S. With Excessive Daytime Sleepiness Associated With Narcolepsy With or Without Cataplexy
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Narcolepsy Without Cataplexy
Narcolepsy With Cataplexy
Lead sponsor: Harmony Biosciences Management, Inc.
Sponsor class: INDUSTRY
First posted: 2018-02-14
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

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.


Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Sleep Disorders Center of Alabama
state: Alabama
city: Cullman
country: United States
facility: CPC
state: Alabama
city: Phoenix
country: United States
facility: Xenoscience
state: Arizona
city: Phoenix
country: United States
facility: Xenoscience
state: Arizona
city: Phoenix
country: United States
facility: Foothills Neurology
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: San Ramon
country: United States
facility: Sleep Medicine Specialists of California
state: California
city: Santa Monica
country: United States
facility: Santa Monica Sleep Disorders Center
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Boulder
country: United States
facility: Colorado Sleep Institute
state: Colorado
city: Norwalk
country: United States
facility: Norwalk Hospital Sleep Disorders Center
state: Connecticut
city: Clearwater
country: United States
facility: Clinical Neurosciences fo Tampa Bay
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Miami
country: United States
facility: Sleep Medicine Specialists of South Florida, PA
state: Florida
city: Winter Park
country: United States
facility: Child Lung, Asthma Sleep Specialists
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Atlanta
country: United States
facility: NeuroTrials Research Center
state: Georgia
city: Gainesville
country: United States
facility: The Neurological Center of North Georgia
state: Georgia
city: Savannah
country: United States
facility: Critical Healthcare Decisions
state: Georgia
city: Chicago
country: United States
facility: Northwestern Medical Group
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Glenview
country: United States
facility: NorthShore Uni HealthSys-Glenbrook Hospital
state: Illinois
city: Northbrook
country: United States
facility: Northshore Sleep Medicine
state: Illinois
city: Greenwood
country: United States
facility: Indiana Internal Medicine Consultants
state: Indiana
city: Fairway
country: United States
facility: The University of Kansas Health System
state: Kansas
city: Louisville
country: United States
facility: Norton Pulmonary Specialists
state: Kentucky
city: Scarborough
country: United States
facility: Maine Medical Partners - Neurology
state: Maine
city: Chevy Chase
country: United States
facility: The Center for Sleep and Wake Disorders
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: The University of Michigan
state: Michigan
city: Novi
country: United States
facility: Henry Ford Health System
state: Michigan
city: Portage
country: United States
facility: Bronson Sleep Health
state: Michigan
city: Shelby
country: United States
facility: Alliance Health Shelby Family Medicine
state: Michigan
city: Brooklyn Park
country: United States
facility: Fairview Health Services
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Chesterfield
country: United States
facility: St. Lukes Hospital, Sleep Medicine
state: Missouri
city: Columbia
country: United States
facility: University of Missouri
state: Missouri
city: Kansas City
country: United States
facility: Saint Luke's Hospital of Kansas City
state: Missouri
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: New Hyde Park
country: United States
facility: Norwell Health
state: New York
city: New York
country: United States
facility: NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center
state: New York
city: New York
country: United States
facility: Columbia University
state: New York
city: Rochester
country: United States
facility: Unity Sleep Disorders Center
state: New York
city: Rochester
country: United States
facility: University of Rochester Sleep Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Huntersville
country: United States
facility: Excel Psychiatric Associates, PA
state: North Carolina
city: Raleigh
country: United States
facility: Raleigh Neurology Assoc., PA
state: North Carolina
city: Akron
country: United States
facility: Neurology & Neuroscience Associates, Inc.
state: Ohio
city: Cincinnati
country: United States
facility: Intrepid Research, LLC
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Dublin
country: United States
facility: Ohio Sleep Medicine Institute
state: Ohio
city: Tulsa
country: United States
facility: Oklahoma Heart Institute
state: Oklahoma
city: Tulsa
country: United States
facility: Oklahoma Heart Institute
state: Oklahoma
city: Portland
country: United States
facility: Legacy Medical Group Sleep Medicine
state: Oregon
city: Berwyn
country: United States
facility: Collegeville Family Practice
state: Pennsylvania
city: Paoli
country: United States
facility: Paoli Sleep Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Maria J. Sunseri, MD, LLC
state: Pennsylvania
city: Willow Grove
country: United States
facility: Abington Neurological Associates
state: Pennsylvania
city: Wyomissing
country: United States
facility: Respitory Specialists
state: Pennsylvania
city: West Warwick
country: United States
facility: Brown Medicine/Rhode Island Hospital
state: Rhode Island
city: Columbia
country: United States
facility: Bogan Sleep Consultants
state: South Carolina
city: Dallas
country: United States
facility: Sleep Medicine Associates of Texas
state: Texas
city: Houston
country: United States
facility: Houston Neurology & Sleep Center
state: Texas
city: San Antonio
country: United States
facility: Sleep Therapy & Research Center
state: Texas
city: Sugar Land
country: United States
facility: Comprehensive Sleep Medicine Associates
state: Texas
city: Charlottesville
country: United States
facility: Chalottesville Neurology and Sleep Medicine
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of The King's Daughters
state: Virginia
city: Norfolk
country: United States
facility: Eastern Virginia Medical School
state: Virginia
city: Barboursville
country: United States
facility: Stultz Sleep and Behavioral Health
state: West Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisonsin
state: Wisconsin
Study officials:
affiliation: Albert Einstein College of Medicine
name: Michael Thorpy, MD
role: PRINCIPAL_INVESTIGATOR
References: