Trial · NCT02611687

Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period

NCT02611687, Phase 3, Completed, Bioprojet; results first posted 2024-09-19

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period. Trials, record NCT02611687. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02611687

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
NCT number: NCT02611687
Status: Completed
Phases: Phase 3
Enrollment: 110 participants, actual
Lead sponsor: Bioprojet
Conditions: Narcolepsy With Cataplexy, Narcolepsy Without Cataplexy
Interventions: pitolisant, Placebo
First posted: 2015-11-23
Last updated: 2025-06-26
Other registries' IDs: eudract:2013-001506-29
Results first posted: 2024-09-19

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.	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.
Secondary	To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.	8 weeks	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.
Secondary	To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .	8 weeks	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.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.	8 weeks	Full Analysis Set	score on a scale	GEOMETRIC_LEAST_SQUARES_MEAN
SECONDARY	To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.	8 weeks	Full Analysis Set	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .	8 weeks	Full Analysis Set	score on a scale	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group6

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.	Pitolisant	-6.29	Standard Error 1.14	score on a scale
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.	Placebo	-2.60	Standard Error 1.35	score on a scale
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.	Pitolisant	-5.53	Standard Error 0.66	score on a scale
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.	Placebo	-2.11	Standard Error 0.89	score on a scale
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .	Pitolisant	-2.88	Standard Error 0.44	score on a scale
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .	Placebo	-1.12	Standard Error 0.64	score on a scale

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-blind Period (0 to 8 Weeks)	STARTED	Pitolisant	72
Double-blind Period (0 to 8 Weeks)	STARTED	Placebo	38
Double-blind Period (0 to 8 Weeks)	COMPLETED	Pitolisant	70
Double-blind Period (0 to 8 Weeks)	COMPLETED	Placebo	37
Double-blind Period (0 to 8 Weeks)	NOT COMPLETED	Pitolisant	2
Double-blind Period (0 to 8 Weeks)	NOT COMPLETED	Placebo	1
Open-label Period (0 to >11 Months)	STARTED	Pitolisant	70
Open-label Period (0 to >11 Months)	STARTED	Placebo	36
Open-label Period (0 to >11 Months)	COMPLETED	Pitolisant	39
Open-label Period (0 to >11 Months)	COMPLETED	Placebo	21

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events10

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Headache	Nervous system disorders	Pitolisant	14	73	24
other	Headache	Nervous system disorders	Placebo	3	37	4
other	Insomnia	Psychiatric disorders	Pitolisant	5	73	5
other	Insomnia	Psychiatric disorders	Placebo	1	37	1
other	Influenza	Infections and infestations	Pitolisant	2	73	2
other	Influenza	Infections and infestations	Placebo	2	37	2
other	Upper respiratory tract infection	Infections and infestations	Pitolisant	0	73	0
other	Upper respiratory tract infection	Infections and infestations	Placebo	3	37	5
other	Diarrhoea	Gastrointestinal disorders	Pitolisant	0	73	0
other	Diarrhoea	Gastrointestinal disorders	Placebo	2	37	2

Source ClinicalTrials.gov

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Pitolisant	0	73
Total, all-cause mortality	Placebo	0	37
Total, other adverse events	Pitolisant	15	73
Total, other adverse events	Placebo	11	37
Total, serious adverse events	Pitolisant	0	73
Total, serious adverse events	Placebo	0	37

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
36931805	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Pitolisant vs Placebo: deaths from any cause	Trial comparisons
Pitolisant vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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GET https://conductscience.org/api/trials/v1/NCT02611687/registered

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