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

August 22, 2019 updated by: Harmony Biosciences, LLC

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
No longer available

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35213
        • Sleep Disorders Center of Alabama
      • Cullman, Alabama, United States, 35058
        • CPC
    • Arizona
      • Phoenix, Arizona, United States, 85004
        • Xenoscience
      • Phoenix, Arizona, United States, 85004-1150
        • Xenoscience
      • Phoenix, Arizona, United States, 85048
        • Foothills Neurology
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic
    • California
      • San Ramon, California, United States, 94583
        • Sleep Medicine Specialists of California
      • Santa Monica, California, United States, 90404
        • Santa Monica Sleep Disorders Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
      • Boulder, Colorado, United States, 80301
        • Colorado Sleep Institute
    • Connecticut
      • Norwalk, Connecticut, United States, 06850
        • Norwalk Hospital Sleep Disorders Center
    • Florida
      • Clearwater, Florida, United States, 33756
        • Clinical Neurosciences fo Tampa Bay
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic
      • Miami, Florida, United States, 33126
        • Sleep Medicine Specialists of South Florida, PA
      • Winter Park, Florida, United States, 32789
        • Child Lung, Asthma Sleep Specialists
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Emory University
      • Atlanta, Georgia, United States, 30342
        • NeuroTrials Research Center
      • Gainesville, Georgia, United States, 30501
        • The Neurological Center of North Georgia
      • Savannah, Georgia, United States, 31419
        • Critical Healthcare Decisions
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern Medical Group
      • Chicago, Illinois, United States, 60637
        • Rush University Medical Center
      • Glenview, Illinois, United States, 60026
        • NorthShore Uni HealthSys-Glenbrook Hospital
      • Northbrook, Illinois, United States, 60062
        • Northshore Sleep Medicine
    • Indiana
      • Greenwood, Indiana, United States, 46143
        • Indiana Internal Medicine Consultants
    • Kansas
      • Fairway, Kansas, United States, 66205
        • The University of Kansas Health System
    • Kentucky
      • Louisville, Kentucky, United States, 40218
        • Norton Pulmonary Specialists
    • Maine
      • Scarborough, Maine, United States, 04074
        • Maine Medical Partners - Neurology
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
        • The Center for Sleep and Wake Disorders
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • The University of Michigan
      • Novi, Michigan, United States, 48377
        • Henry Ford Health System
      • Portage, Michigan, United States, 49024
        • Bronson Sleep Health
      • Shelby, Michigan, United States, 48317
        • Alliance Health Shelby Family Medicine
    • Minnesota
      • Brooklyn Park, Minnesota, United States, 55443
        • Fairview Health Services
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Missouri
      • Chesterfield, Missouri, United States, 63017
        • St. Lukes Hospital, Sleep Medicine
      • Columbia, Missouri, United States, 65212
        • University of Missouri
      • Kansas City, Missouri, United States, 64111
        • Saint Luke's Hospital of Kansas City
    • New York
      • Bronx, New York, United States, 10467
        • Montefiore Medical Center
      • New Hyde Park, New York, United States, 11042
        • Norwell Health
      • New York, New York, United States, 10032
        • Columbia University
      • New York, New York, United States, 10016
        • NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center
      • Rochester, New York, United States, 14618
        • Unity Sleep Disorders Center
      • Rochester, New York, United States, 35058
        • University of Rochester Sleep Center
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
      • Huntersville, North Carolina, United States, 28078
        • Excel Psychiatric Associates, PA
      • Raleigh, North Carolina, United States, 27607
        • Raleigh Neurology Assoc., PA
    • Ohio
      • Akron, Ohio, United States, 44320
        • Neurology & Neuroscience Associates, Inc.
      • Cincinnati, Ohio, United States, 45245
        • Intrepid Research, LLC
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
      • Dublin, Ohio, United States, 43017
        • Ohio Sleep Medicine Institute
    • Oklahoma
      • Tulsa, Oklahoma, United States, 74104
        • Oklahoma Heart Institute
      • Tulsa, Oklahoma, United States, 74133
        • Oklahoma Heart Institute
    • Oregon
      • Portland, Oregon, United States, 97210
        • Legacy Medical Group Sleep Medicine
    • Pennsylvania
      • Berwyn, Pennsylvania, United States, 19426
        • Collegeville Family Practice
      • Paoli, Pennsylvania, United States, 19301
        • Paoli Sleep Center
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson University
      • Pittsburgh, Pennsylvania, United States, 15224
        • Maria J. Sunseri, MD, LLC
      • Willow Grove, Pennsylvania, United States, 19090
        • Abington Neurological Associates
      • Wyomissing, Pennsylvania, United States, 19610
        • Respitory Specialists
    • Rhode Island
      • West Warwick, Rhode Island, United States, 02893
        • Brown Medicine/Rhode Island Hospital
    • South Carolina
      • Columbia, South Carolina, United States, 29201
        • Bogan Sleep Consultants
    • Texas
      • Dallas, Texas, United States, 75231
        • Sleep Medicine Associates of Texas
      • Houston, Texas, United States, 77063
        • Houston Neurology & Sleep Center
      • San Antonio, Texas, United States, 78229
        • Sleep Therapy & Research Center
      • Sugar Land, Texas, United States, 77478
        • Comprehensive Sleep Medicine Associates
    • Virginia
      • Charlottesville, Virginia, United States, 22911
        • Chalottesville Neurology and Sleep Medicine
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughters
      • Norfolk, Virginia, United States, 23507
        • Eastern Virginia Medical School
    • West Virginia
      • Barboursville, West Virginia, United States, 25504
        • Stultz Sleep and Behavioral Health
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisonsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Harmony Biosciences, LLC

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Michael Thorpy, MD, Albert Einstein College of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 7, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 7, 2018

First Posted (ACTUAL)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 14, 2018

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 28, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 22, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • HBS-101-CL-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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