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

Status:

NO_LONGER_AVAILABLE

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

Lead Sponsor:

Harmony Biosciences Management, Inc.

Conditions:

Narcolepsy Without Cataplexy

Narcolepsy With Cataplexy

Eligibility:

All Genders

18+ years

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 ope...

Eligibility Criteria

Inclusion

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.

Exclusion

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.

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT03433131

Last Update

August 28 2019

Active Locations (71)

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Page 1 of 18 (71 locations)

Sleep Disorders Center of Alabama

Birmingham, Alabama, United States, 35213

CPC

Cullman, Alabama, United States, 35058

Xenoscience

Phoenix, Arizona, United States, 85004-1150