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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

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ClinicalTrials.gov Identifier: NCT03433131
Expanded Access Status : Available
First Posted : February 14, 2018
Last Update Posted : December 6, 2018
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Narcolepsy Without Cataplexy Narcolepsy With Cataplexy Drug: Pitolisant

Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
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



Intervention Details:
  • Drug: Pitolisant
    Tablets
    Other Name: Wakix

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03433131


Contacts
Contact: Eric D Bauer, BS 312-777-3744 ebauer@harmonybiosciences.com

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Sponsors and Collaborators
Harmony Biosciences, LLC
Investigators
Principal Investigator: Michael Thorpy, MD Albert Einstein College of Medicine, Inc.

Additional Information:
Responsible Party: Harmony Biosciences, LLC
ClinicalTrials.gov Identifier: NCT03433131     History of Changes
Other Study ID Numbers: HBS-101-CL-001
First Posted: February 14, 2018    Key Record Dates
Last Update Posted: December 6, 2018
Last Verified: December 2018

Additional relevant MeSH terms:
Narcolepsy
Cataplexy
Disorders of Excessive Somnolence
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Wake Disorders
Nervous System Diseases
Mental Disorders