A Phase 3 interventional study of pitolisant and Placebo in Narcolepsy With Cataplexy and Narcolepsy Without Cataplexy, sponsored by Bioprojet. Completed at 11 sites in 5 countries. Open to participants aged 6 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-06-26.
Sponsored by Bioprojet · Phase 3, Interventional, and Treatment
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.
157 studies on the registry are indexed under Narcolepsy; 37 are open to participants now.
This study's enrollment of 110 is above the median of 78 across 114 interventional studies indexed under Narcolepsy.
Browse Narcolepsy studies →Bioprojet is the lead sponsor of 29 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
tablet, oral, once a day.
Drug: pitolisant
tablet, oral, once a day.
Drug: Placebo
Tablet
Also known as: BF2.649
Tablet
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.
Time frame: 8 weeks
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.
Time frame: 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.
Time frame: 8 weeks
| Milestone | Pitolisant | Placebo |
|---|---|---|
| Started | 72 | 38 |
| Completed | 70 | 37 |
| Not completed | 2 | 1 |
| Withdrew: Withdrawal by subject | 2 | 1 |
| Milestone | Pitolisant | Placebo |
|---|---|---|
| Started | 70 | 36 |
| Completed | 39 | 21 |
| Not completed | 31 | 15 |
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.
| score on a scale | Pitolisant | Placebo |
|---|---|---|
| To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score. | -6.29 ± 1.14 | -2.60 ± 1.35 |
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.
| score on a scale | Pitolisant | Placebo |
|---|---|---|
| To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale. | -5.53 ± 0.66 | -2.11 ± 0.89 |
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.
| score on a scale | Pitolisant | Placebo |
|---|---|---|
| To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore . | -2.88 ± 0.44 | -1.12 ± 0.64 |
Collected over 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pitolisant | 0/73 (0%) | 0/73 (0%) | 15/73 (20.5%) |
| Placebo | 0/37 (0%) | 0/37 (0%) | 11/37 (29.7%) |
| Event | Pitolisant | Placebo |
|---|---|---|
| HeadacheNervous system disorders | 14/73 | 3/37 |
| Upper respiratory tract infectionInfections and infestations | 0/73 | 3/37 |
| InsomniaPsychiatric disorders | 5/73 | 1/37 |
| InfluenzaInfections and infestations | 2/73 | 2/37 |
| DiarrhoeaGastrointestinal disorders | 0/73 | 2/37 |
| Age, Categorical(Participants) | Pitolisant | Placebo | Total |
|---|---|---|---|
| <=18 years | 72 | 38 | 110 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Age, Continuous(years) | Pitolisant | Placebo | Total |
|---|---|---|---|
| Mean | 13.0 ± 3.0 | 12.5 ± 3.0 | 12.9 ± 3.0 |
| Sex: Female, Male(Participants) | Pitolisant | Placebo | Total |
|---|---|---|---|
| Female | 35 | 14 | 49 |
| Male | 37 | 24 | 61 |
| Race (NIH/OMB)(Participants) | Pitolisant | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 0 | 0 | 0 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 72 | 38 | 110 |
| Region of Enrollment(participants) | Pitolisant | Placebo | Total |
|---|---|---|---|
| Netherlands | 3 | 2 | 5 |
| Finland | 1 | 1 | 2 |
| Italy | 17 | 9 | 26 |
| France | 19 | 9 | 28 |
| Russia | 32 | 17 | 49 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Pitolisant
Bioprojet