Narcolepsy Without CataplexyNCT03433131
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
A study testing Pitolisant in people with Narcolepsy Without Cataplexy and Narcolepsy With Cataplexy, run by Harmony Biosciences Management, Inc. at 71 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Narcolepsy Without Cataplexy, Narcolepsy With Cataplexy
- What is being tested?
- Pitolisant
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 71 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
71 sites in 1 country.
71 sites in 1 country
United States71 sites
Neurology & Neuroscience Associates, Inc.
Akron, Ohio, 44320
The University of Michigan
Ann Arbor, Michigan, 48109
Emory University
Atlanta, Georgia, 30329
NeuroTrials Research Center
Atlanta, Georgia, 30342
Children's Hospital Colorado
Aurora, Colorado, 80045
Stultz Sleep and Behavioral Health
Barboursville, West Virginia, 25504
Collegeville Family Practice
Berwyn, Pennsylvania, 19426
Sleep Disorders Center of Alabama
Birmingham, Alabama, 35213
Boston Children's Hospital
Boston, Massachusetts, 02115
Colorado Sleep Institute
Boulder, Colorado, 80301
Fairview Health Services
Brooklyn Park, Minnesota, 55443
Chalottesville Neurology and Sleep Medicine
Charlottesville, Virginia, 22911
St. Lukes Hospital, Sleep Medicine
Chesterfield, Missouri, 63017
The Center for Sleep and Wake Disorders
Chevy Chase, Maryland, 20815
Northwestern Medical Group
Chicago, Illinois, 60611
Rush University Medical Center
Chicago, Illinois, 60637
Intrepid Research, LLC
Cincinnati, Ohio, 45245
Clinical Neurosciences fo Tampa Bay
Clearwater, Florida, 33756
Cleveland Clinic
Cleveland, Ohio, 44195
University of Missouri
Columbia, Missouri, 65212
Bogan Sleep Consultants
Columbia, South Carolina, 29201
CPC
Cullman, Alabama, 35058
Sleep Medicine Associates of Texas
Dallas, Texas, 75231
Ohio Sleep Medicine Institute
Dublin, Ohio, 43017
Duke University Medical Center
Durham, North Carolina, 27710
The University of Kansas Health System
Fairway, Kansas, 66205
The Neurological Center of North Georgia
Gainesville, Georgia, 30501
NorthShore Uni HealthSys-Glenbrook Hospital
Glenview, Illinois, 60026
Indiana Internal Medicine Consultants
Greenwood, Indiana, 46143
Houston Neurology & Sleep Center
Houston, Texas, 77063
Excel Psychiatric Associates, PA
Huntersville, North Carolina, 28078
Mayo Clinic
Jacksonville, Florida, 32224
Saint Luke's Hospital of Kansas City
Kansas City, Missouri, 64111
Norton Pulmonary Specialists
Louisville, Kentucky, 40218
Sleep Medicine Specialists of South Florida, PA
Miami, Florida, 33126
Medical College of Wisonsin
Milwaukee, Wisconsin, 53226
Norwell Health
New Hyde Park, New York, 11042
NYU-FGP Comphrenhensive Epilepsy Center-Sleep Center
New York, New York, 10016
Columbia University
New York, New York, 10032
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Eastern Virginia Medical School
Norfolk, Virginia, 23507
Northshore Sleep Medicine
Northbrook, Illinois, 60062
Norwalk Hospital Sleep Disorders Center
Norwalk, Connecticut, 06850
Henry Ford Health System
Novi, Michigan, 48377
Paoli Sleep Center
Paoli, Pennsylvania, 19301
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
Xenoscience
Phoenix, Arizona, 85004-1150
Xenoscience
Phoenix, Arizona, 85004
Foothills Neurology
Phoenix, Arizona, 85048
Maria J. Sunseri, MD, LLC
Pittsburgh, Pennsylvania, 15224
Bronson Sleep Health
Portage, Michigan, 49024
Legacy Medical Group Sleep Medicine
Portland, Oregon, 97210
Raleigh Neurology Assoc., PA
Raleigh, North Carolina, 27607
Mayo Clinic
Rochester, Minnesota, 55905
Unity Sleep Disorders Center
Rochester, New York, 14618
University of Rochester Sleep Center
Rochester, New York, 35058
Sleep Therapy & Research Center
San Antonio, Texas, 78229
Sleep Medicine Specialists of California
San Ramon, California, 94583
Santa Monica Sleep Disorders Center
Santa Monica, California, 90404
Critical Healthcare Decisions
Savannah, Georgia, 31419
Maine Medical Partners - Neurology
Scarborough, Maine, 04074
Mayo Clinic
Scottsdale, Arizona, 85259
Alliance Health Shelby Family Medicine
Shelby, Michigan, 48317
Comprehensive Sleep Medicine Associates
Sugar Land, Texas, 77478
Montefiore Medical Center
The Bronx, New York, 10467
Oklahoma Heart Institute
Tulsa, Oklahoma, 74104
Oklahoma Heart Institute
Tulsa, Oklahoma, 74133
Brown Medicine/Rhode Island Hospital
West Warwick, Rhode Island, 02893
Abington Neurological Associates
Willow Grove, Pennsylvania, 19090
Child Lung, Asthma Sleep Specialists
Winter Park, Florida, 32789
Respitory Specialists
Wyomissing, Pennsylvania, 19610
About this study
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
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.
- Being tested
- Pitolisant
- Medical subject headings
- Narcolepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PitolisantDrug
Also called Wakix
Tablets
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Narcolepsy Without CataplexyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Thorpy, MD, Principal investigator, Albert Einstein College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03433131
- Sponsor's protocol ID
- HBS-101-CL-001
- Status last verified
- Aug 2019
- First submitted
- 7 Feb 2018
- Last updated
- 28 Aug 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03433131
- Acronym / sponsor ID
- HBS-101-CL-001
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Harmony Biosciences Management, Inc.Industry (pharma, biotech, devices)
- First posted
- 14 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PitolisantAlso: WakixTablets | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2019.
- Program website to provide information to both prosective patients and physiciansLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Thorpy, MD principal investigator
Albert Einstein College of Medicine
71 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Pitolisant in people with Narcolepsy Without Cataplexy and Narcolepsy With Cataplexy, run by Harmony Biosciences Management, Inc. at 71 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 71 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2019.