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

Harmony Biosciences

Status

Conditions: Narcolepsy Without Cataplexy; Narcolepsy With Cataplexy

Treatments: Drug: Pitolisant

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT03433131

HBS-101-CL-001

Details and patient eligibility

About

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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.