Narcolepsy With CataplexyPhase 3NCT02611687
Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
A Phase 3 study testing pitolisant in people with Narcolepsy With Cataplexy and Narcolepsy Without Cataplexy, run by Bioprojet at 11 sites in 5 countries.
Trial summary
The purpose of this multicenter double blind study is to assess efficacy and safety of Pitolisant versus placebo in paediatric Narcoleptic patients with or without cataplexy. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Narcolepsy With Cataplexy, Narcolepsy Without Cataplexy
- What is being tested?
- pitolisant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 110 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 6 Jun 2016to 5 Jun 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male and female children from 6 to less than 18 years of age suffering from narcolepsy with or without cataplexy - ICSD-3 criteria (narcolepsy type 1 and 2).
- PDSS
- Patients should be free of non-authorized medication, in particular psychostimulant treatments as from the screening visit onwards.
- Parents - and patients old enough to understand who have expressed a willingness to participate in the study, who have signed and dated the informed consent form prior to beginning protocol required procedures.
- In the opinion of the investigator, the patient must have adequate support to comply with the entire study requirements as described in the protocol (e.g., transportation to and from trial site, self rating scales and diaries completion, drug compliance, scheduled visits, tests).
Who cannot join
- Any other conditions that can be considered the primary causes of EDS.
- Cataplectic patients treated by anticataplectics which are not under a stable treatment at the time of inclusion.
- Patients treated for cataplexy or any other pathology, by tricyclic antidepressants.
- Any significant abnormality of the electrocardiogram and particularly Fridericia's QTc interval.
- Patients with significant abnormality or clinical laboratory results.
- Psychiatric and neurological disorders in the investigator's opinion, would preclude the patient's participation and completion of this trial or comprise reliable representation of subjective symptoms.
- Active clinically significant illness, including unstable cardiovascular, endocrine, neoplastic, gastrointestinal, haematological, hepatic, immunologic, metabolic, neurological (other than narcolepsy/cataplexy), pulmonary, and/or renal disease which could interfere with the study conduct or counter-indicate the study treatments or place the patient at risk during the trial or compromise the study objectives.
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
Russia5 sites
Scientific-Research Medical Complex "Your Health"
Kazan'
I.M. Sechenov First Moscow State Medical University
Moscow
V.M. Behterev National Medical Research Psychiatry and Neurology Center
Saint Petersburg
Samara Regional Clinical Hospital
Samara
N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1
Simferopol
France3 sites
Hôpital Femme-Mère-Enfant
Bron
Hôpital Gui de Chauliac
Montpellier
Hôpital Robert Debré
Paris
About this study
Official title: Double Blind, Multicentre, Randomized, Placebo-controlled Trial to Evaluate Safety and Efficacy of Pitolisant in Children From 6 to Less Than 18 Years With Narcolepsy With/Without Cataplexy, Followed by a Prolonged Open-label Period
The purpose of this multicenter double blind study is to assess efficacy and safety of Pitolisant versus placebo in paediatric Narcoleptic patients with or without cataplexy.
- Being tested
- pitolisant, Placebo
- 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 BF2.649
Tablet
PlaceboDrug
Tablet
| Study group | Role | What they receive |
|---|---|---|
| pitolisant | Experimental | tablet, oral, once a day. Receives: Drug: pitolisant |
| placebo | Placebo comparator | tablet, oral, once a day. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.
Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS) from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures).
The UNS is an 11-item scale used to measure the intensity and frequency of symptoms of narcolepsy (EDS and cataplexy). The total score varies from 0 to 44, with higher scores denoting greater narcoleptic tendencies.
Measured: 8 weeks
Secondary outcomes (2)
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.
Changes in EDS measured by the Pediatric Daytime Sleepiness Scale (PDSS) from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures).
The PDSS total score can range from 0 and 32 and a score >13 is considered abnormal sleepiness.
Measured: 8 weeks
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .
Changes in EDS measured by Ullanlinna Narcolepsy Scale-Cataplexy Subscore (UNS-CTP) in patients with type 1 narcolepsy from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures).
This subscore is defined as the sum of the first 4 items of the UNS, UNS-CTP subscore ranges from 0 to 16, with higher scores indicating more severe symptoms.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2015 (estimated)
- Study start6 Jun 2016
- Primary completion3 Apr 2021
- Study completion5 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Narcolepsy With 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 110 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioprojet
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Giuseppe Plazzi, MD, Study chair, Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02611687
- Sponsor's protocol ID
- P11-06/BF2.649
- Other IDs
- 2013-001506-29
- Status last verified
- Jun 2025
- First submitted
- 16 Nov 2015
- Last updated
- 26 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Dauvilliers Y, Lecendreux M, Lammers GJ, Franco P, Poluektov M, Causse C, Lecomte I, Lecomte JM, Lehert P, Schwartz JC, Plazzi G. Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy: a double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2023 Apr;22(4):303-311. doi: 10.1016/S1474-4422(23)00036-4.
PubMed 36931805
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double Blind, Multicentre, Randomized, Placebo-controlled Trial to Evaluate Safety and Efficacy of Pitolisant in Children From 6 to Less Than 18 Years With Narcolepsy With/Without Cataplexy, Followed by a Prolonged Open-label Period
- ClinicalTrials.gov ID
- NCT02611687
- Acronym / sponsor ID
- P11-06/BF2.649
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 110 participants (actual)
- Lead sponsor
- BioprojetUniversity, hospital or other
- Study start
- 6 Jun 2016 (actual)
- Primary completion
- 3 Apr 2021 (actual)
- Study completion
- 5 Jun 2025 (actual)
- First posted
- 23 Nov 2015 (estimated)
- Results
- Posted 19 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this multicenter double blind study is to assess efficacy and safety of Pitolisant versus placebo in paediatric Narcoleptic patients with or without cataplexy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| pitolisant | Experimental | Drug: pitolisanttablet, oral, once a day. |
| placebo | Placebo comparator | Drug: Placebotablet, oral, once a day. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Male and female children from 6 to less than 18 years of age suffering from narcolepsy with or without cataplexy - ICSD-3 criteria (narcolepsy type 1 and 2).
- PDSS
- Patients should be free of non-authorized medication, in particular psychostimulant treatments as from the screening visit onwards.
- Parents - and patients old enough to understand who have expressed a willingness to participate in the study, who have signed and dated the informed consent form prior to beginning protocol required procedures.
- In the opinion of the investigator, the patient must have adequate support to comply with the entire study requirements as described in the protocol (e.g., transportation to and from trial site, self rating scales and diaries completion, drug compliance, scheduled visits, tests).
Key exclusion criteria (7 listed)
- Any other conditions that can be considered the primary causes of EDS.
- Cataplectic patients treated by anticataplectics which are not under a stable treatment at the time of inclusion.
- Patients treated for cataplexy or any other pathology, by tricyclic antidepressants.
- Any significant abnormality of the electrocardiogram and particularly Fridericia's QTc interval.
- Patients with significant abnormality or clinical laboratory results.
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: BF2.649Tablet | Drug | pitolisant |
| PlaceboTablet | Drug | placebo |
Primary outcome
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.
Time frame: 8 weeks
Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS) from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures). The UNS is an 11-item scale used to measure the intensity and frequency of symptoms of narcolepsy (EDS and cataplexy). The total score varies from 0 to 44, with higher scores denoting greater narcoleptic tendencies.
2 secondary outcomes
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.
Time frame: 8 weeks
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .
Time frame: 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36931805Dauvilliers Y, Lecendreux M, Lammers GJ, Franco P, Poluektov M, Causse C, Lecomte I, Lecomte JM, Lehert P, Schwartz JC, Plazzi G. Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy: a double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2023 Apr;22(4):303-311. doi: 10.1016/S1474-4422(23)00036-4.
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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.
Giuseppe Plazzi, MD study chair
Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna
11 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing pitolisant in people with Narcolepsy With Cataplexy and Narcolepsy Without Cataplexy, run by Bioprojet at 11 sites in 5 countries.
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 5 inclusion and 7 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: finished, as of the sponsor's last update on 26 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Russia, France, Finland, Italy, Netherlands. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Sep 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score. (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02611687, which also lists 1 linked publication.
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 26 Jun 2025.