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

Sponsor

Harmony Biosciences, LLC (Industry)

Overall Status

No longer available

CT.gov ID

NCT03433131

Collaborator

(none)

Locations

Study Details

Study Description

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	Phase
Narcolepsy Without Cataplexy Narcolepsy With Cataplexy	Drug: Pitolisant

Study Design

Study Type:

Expanded Access

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

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Inclusion Criteria:

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 Criteria:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Sleep Disorders Center of Alabama	Birmingham	Alabama	United States	35213
2	CPC	Cullman	Alabama	United States	35058
3	Xenoscience	Phoenix	Arizona	United States	85004-1150
4	Xenoscience	Phoenix	Arizona	United States	85004
5	Foothills Neurology	Phoenix	Arizona	United States	85048
6	Mayo Clinic	Scottsdale	Arizona	United States	85259
7	Sleep Medicine Specialists of California	San Ramon	California	United States	94583
8	Santa Monica Sleep Disorders Center	Santa Monica	California	United States	90404
9	Children's Hospital Colorado	Aurora	Colorado	United States	80045
10	Colorado Sleep Institute	Boulder	Colorado	United States	80301
11	Norwalk Hospital Sleep Disorders Center	Norwalk	Connecticut	United States	06850
12	Clinical Neurosciences fo Tampa Bay	Clearwater	Florida	United States	33756
13	Mayo Clinic	Jacksonville	Florida	United States	32224
14	Sleep Medicine Specialists of South Florida, PA	Miami	Florida	United States	33126
15	Child Lung, Asthma Sleep Specialists	Winter Park	Florida	United States	32789
16	Emory University	Atlanta	Georgia	United States	30329
17	NeuroTrials Research Center	Atlanta	Georgia	United States	30342
18	The Neurological Center of North Georgia	Gainesville	Georgia	United States	30501
19	Critical Healthcare Decisions	Savannah	Georgia	United States	31419
20	Northwestern Medical Group	Chicago	Illinois	United States	60611
21	Rush University Medical Center	Chicago	Illinois	United States	60637
22	NorthShore Uni HealthSys-Glenbrook Hospital	Glenview	Illinois	United States	60026
23	Northshore Sleep Medicine	Northbrook	Illinois	United States	60062
24	Indiana Internal Medicine Consultants	Greenwood	Indiana	United States	46143
25	The University of Kansas Health System	Fairway	Kansas	United States	66205
26	Norton Pulmonary Specialists	Louisville	Kentucky	United States	40218
27	Maine Medical Partners - Neurology	Scarborough	Maine	United States	04074
28	The Center for Sleep and Wake Disorders	Chevy Chase	Maryland	United States	20815
29	Boston Children's Hospital	Boston	Massachusetts	United States	02115
30	The University of Michigan	Ann Arbor	Michigan	United States	48109
31	Henry Ford Health System	Novi	Michigan	United States	48377
32	Bronson Sleep Health	Portage	Michigan	United States	49024
33	Alliance Health Shelby Family Medicine	Shelby	Michigan	United States	48317
34	Fairview Health Services	Brooklyn Park	Minnesota	United States	55443
35	Mayo Clinic	Rochester	Minnesota	United States	55905
36	St. Lukes Hospital, Sleep Medicine	Chesterfield	Missouri	United States	63017
37	University of Missouri	Columbia	Missouri	United States	65212
38	Saint Luke's Hospital of Kansas City	Kansas City	Missouri	United States	64111
39	Montefiore Medical Center	Bronx	New York	United States	10467
40	Norwell Health	New Hyde Park	New York	United States	11042
41	NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center	New York	New York	United States	10016
42	Columbia University	New York	New York	United States	10032
43	Unity Sleep Disorders Center	Rochester	New York	United States	14618
44	University of Rochester Sleep Center	Rochester	New York	United States	35058
45	Duke University Medical Center	Durham	North Carolina	United States	27710
46	Excel Psychiatric Associates, PA	Huntersville	North Carolina	United States	28078
47	Raleigh Neurology Assoc., PA	Raleigh	North Carolina	United States	27607
48	Neurology & Neuroscience Associates, Inc.	Akron	Ohio	United States	44320
49	Intrepid Research, LLC	Cincinnati	Ohio	United States	45245
50	Cleveland Clinic	Cleveland	Ohio	United States	44195
51	Ohio Sleep Medicine Institute	Dublin	Ohio	United States	43017
52	Oklahoma Heart Institute	Tulsa	Oklahoma	United States	74104
53	Oklahoma Heart Institute	Tulsa	Oklahoma	United States	74133
54	Legacy Medical Group Sleep Medicine	Portland	Oregon	United States	97210
55	Collegeville Family Practice	Berwyn	Pennsylvania	United States	19426
56	Paoli Sleep Center	Paoli	Pennsylvania	United States	19301
57	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
58	Maria J. Sunseri, MD, LLC	Pittsburgh	Pennsylvania	United States	15224
59	Abington Neurological Associates	Willow Grove	Pennsylvania	United States	19090
60	Respitory Specialists	Wyomissing	Pennsylvania	United States	19610
61	Brown Medicine/Rhode Island Hospital	West Warwick	Rhode Island	United States	02893
62	Bogan Sleep Consultants	Columbia	South Carolina	United States	29201
63	Sleep Medicine Associates of Texas	Dallas	Texas	United States	75231
64	Houston Neurology & Sleep Center	Houston	Texas	United States	77063
65	Sleep Therapy & Research Center	San Antonio	Texas	United States	78229
66	Comprehensive Sleep Medicine Associates	Sugar Land	Texas	United States	77478
67	Chalottesville Neurology and Sleep Medicine	Charlottesville	Virginia	United States	22911
68	Children's Hospital of The King's Daughters	Norfolk	Virginia	United States	23507
69	Eastern Virginia Medical School	Norfolk	Virginia	United States	23507
70	Stultz Sleep and Behavioral Health	Barboursville	West Virginia	United States	25504
71	Medical College of Wisonsin	Milwaukee	Wisconsin	United States	53226