Narcolepsy With Cataplexy Clinical Trial
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
| NCT number | NCT03433131 |
| Other study ID # | HBS-101-CL-001 |
| Secondary ID | |
| Status | No longer available |
| Phase | |
| First received | |
| Last updated |
| Verified date | August 2019 |
| Source | Harmony Biosciences, LLC |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Expanded Access |
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.
| Status | No longer available |
| Enrollment | 0 |
| Est. completion date | |
| Est. primary completion date | |
| Accepts healthy volunteers | |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
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 / 3v RR). 8. Significant abnormality in the physical examination or clinical laboratory results. 9. Prior severe adverse reactions to CNS stimulants. |
| Country | Name | City | State |
|---|---|---|---|
| United States | Neurology & Neuroscience Associates, Inc. | Akron | Ohio |
| United States | The University of Michigan | Ann Arbor | Michigan |
| United States | Emory University | Atlanta | Georgia |
| United States | NeuroTrials Research Center | Atlanta | Georgia |
| United States | Children's Hospital Colorado | Aurora | Colorado |
| United States | Stultz Sleep and Behavioral Health | Barboursville | West Virginia |
| United States | Collegeville Family Practice | Berwyn | Pennsylvania |
| United States | Sleep Disorders Center of Alabama | Birmingham | Alabama |
| United States | Boston Children's Hospital | Boston | Massachusetts |
| United States | Colorado Sleep Institute | Boulder | Colorado |
| United States | Montefiore Medical Center | Bronx | New York |
| United States | Fairview Health Services | Brooklyn Park | Minnesota |
| United States | Chalottesville Neurology and Sleep Medicine | Charlottesville | Virginia |
| United States | St. Lukes Hospital, Sleep Medicine | Chesterfield | Missouri |
| United States | The Center for Sleep and Wake Disorders | Chevy Chase | Maryland |
| United States | Northwestern Medical Group | Chicago | Illinois |
| United States | Rush University Medical Center | Chicago | Illinois |
| United States | Intrepid Research, LLC | Cincinnati | Ohio |
| United States | Clinical Neurosciences fo Tampa Bay | Clearwater | Florida |
| United States | Cleveland Clinic | Cleveland | Ohio |
| United States | Bogan Sleep Consultants | Columbia | South Carolina |
| United States | University of Missouri | Columbia | Missouri |
| United States | CPC | Cullman | Alabama |
| United States | Sleep Medicine Associates of Texas | Dallas | Texas |
| United States | Ohio Sleep Medicine Institute | Dublin | Ohio |
| United States | Duke University Medical Center | Durham | North Carolina |
| United States | The University of Kansas Health System | Fairway | Kansas |
| United States | The Neurological Center of North Georgia | Gainesville | Georgia |
| United States | NorthShore Uni HealthSys-Glenbrook Hospital | Glenview | Illinois |
| United States | Indiana Internal Medicine Consultants | Greenwood | Indiana |
| United States | Houston Neurology & Sleep Center | Houston | Texas |
| United States | Excel Psychiatric Associates, PA | Huntersville | North Carolina |
| United States | Mayo Clinic | Jacksonville | Florida |
| United States | Saint Luke's Hospital of Kansas City | Kansas City | Missouri |
| United States | Norton Pulmonary Specialists | Louisville | Kentucky |
| United States | Sleep Medicine Specialists of South Florida, PA | Miami | Florida |
| United States | Medical College of Wisonsin | Milwaukee | Wisconsin |
| United States | Norwell Health | New Hyde Park | New York |
| United States | Columbia University | New York | New York |
| United States | NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center | New York | New York |
| United States | Children's Hospital of The King's Daughters | Norfolk | Virginia |
| United States | Eastern Virginia Medical School | Norfolk | Virginia |
| United States | Northshore Sleep Medicine | Northbrook | Illinois |
| United States | Norwalk Hospital Sleep Disorders Center | Norwalk | Connecticut |
| United States | Henry Ford Health System | Novi | Michigan |
| United States | Paoli Sleep Center | Paoli | Pennsylvania |
| United States | Thomas Jefferson University | Philadelphia | Pennsylvania |
| United States | Foothills Neurology | Phoenix | Arizona |
| United States | Xenoscience | Phoenix | Arizona |
| United States | Xenoscience | Phoenix | Arizona |
| United States | Maria J. Sunseri, MD, LLC | Pittsburgh | Pennsylvania |
| United States | Bronson Sleep Health | Portage | Michigan |
| United States | Legacy Medical Group Sleep Medicine | Portland | Oregon |
| United States | Raleigh Neurology Assoc., PA | Raleigh | North Carolina |
| United States | Mayo Clinic | Rochester | Minnesota |
| United States | Unity Sleep Disorders Center | Rochester | New York |
| United States | University of Rochester Sleep Center | Rochester | New York |
| United States | Sleep Therapy & Research Center | San Antonio | Texas |
| United States | Sleep Medicine Specialists of California | San Ramon | California |
| United States | Santa Monica Sleep Disorders Center | Santa Monica | California |
| United States | Critical Healthcare Decisions | Savannah | Georgia |
| United States | Maine Medical Partners - Neurology | Scarborough | Maine |
| United States | Mayo Clinic | Scottsdale | Arizona |
| United States | Alliance Health Shelby Family Medicine | Shelby | Michigan |
| United States | Comprehensive Sleep Medicine Associates | Sugar Land | Texas |
| United States | Oklahoma Heart Institute | Tulsa | Oklahoma |
| United States | Oklahoma Heart Institute | Tulsa | Oklahoma |
| United States | Brown Medicine/Rhode Island Hospital | West Warwick | Rhode Island |
| United States | Abington Neurological Associates | Willow Grove | Pennsylvania |
| United States | Child Lung, Asthma Sleep Specialists | Winter Park | Florida |
| United States | Respitory Specialists | Wyomissing | Pennsylvania |
| Lead Sponsor | Collaborator |
|---|---|
| Harmony Biosciences, LLC |
United States,
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