Trial Outcomes & Findings for Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period (NCT NCT02611687)

Last Updated: 2025-06-26

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

110 participants

Primary outcome timeframe

8 weeks

Results posted on

2025-06-26

Participant Flow

Participant milestones

Participant milestones
Measure	Pitolisant tablet, oral, once a day. pitolisant: Tablet	Placebo tablet, oral, once a day. Placebo: Tablet
Double-blind Period (0 to 8 Weeks) STARTED	72	38
Double-blind Period (0 to 8 Weeks) COMPLETED	70	37
Double-blind Period (0 to 8 Weeks) NOT COMPLETED	2	1
Open-label Period (0 to >11 Months) STARTED	70	36
Open-label Period (0 to >11 Months) COMPLETED	39	21
Open-label Period (0 to >11 Months) NOT COMPLETED	31	15

Reasons for withdrawal

Reasons for withdrawal
Measure	Pitolisant tablet, oral, once a day. pitolisant: Tablet	Placebo tablet, oral, once a day. Placebo: Tablet
Double-blind Period (0 to 8 Weeks) Withdrawal by Subject	2	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pitolisant n=72 Participants tablet, oral, once a day. pitolisant: Tablet	Placebo n=38 Participants tablet, oral, once a day. Placebo: Tablet	Total n=110 Participants Total of all reporting groups
Age, Categorical <=18 years	72 Participants n=5 Participants	38 Participants n=7 Participants	110 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	13.0 years STANDARD_DEVIATION 3.0 • n=5 Participants	12.5 years STANDARD_DEVIATION 3.0 • n=7 Participants	12.9 years STANDARD_DEVIATION 3.0 • n=5 Participants
Sex: Female, Male Female	35 Participants n=5 Participants	14 Participants n=7 Participants	49 Participants n=5 Participants
Sex: Female, Male Male	37 Participants n=5 Participants	24 Participants n=7 Participants	61 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	72 Participants n=5 Participants	38 Participants n=7 Participants	110 Participants n=5 Participants
Region of Enrollment Netherlands	3 participants n=5 Participants	2 participants n=7 Participants	5 participants n=5 Participants
Region of Enrollment Finland	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Italy	17 participants n=5 Participants	9 participants n=7 Participants	26 participants n=5 Participants
Region of Enrollment France	19 participants n=5 Participants	9 participants n=7 Participants	28 participants n=5 Participants
Region of Enrollment Russia	32 participants n=5 Participants	17 participants n=7 Participants	49 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 weeks

Population: Full Analysis Set

Outcome measures

Outcome measures
Measure	Pitolisant n=72 Participants tablet, oral, once a day. pitolisant: Tablet	Placebo n=38 Participants tablet, oral, once a day. Placebo: Tablet
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.	-6.29 score on a scale Standard Error 1.14	-2.60 score on a scale Standard Error 1.35

SECONDARY outcome

Timeframe: 8 weeks

Population: Full Analysis Set

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.

Outcome measures

Outcome measures
Measure	Pitolisant n=72 Participants tablet, oral, once a day. pitolisant: Tablet	Placebo n=38 Participants tablet, oral, once a day. Placebo: Tablet
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.	-5.53 score on a scale Standard Error 0.66	-2.11 score on a scale Standard Error 0.89

SECONDARY outcome

Timeframe: 8 weeks

Population: Full Analysis Set

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.

Outcome measures

Outcome measures
Measure	Pitolisant n=61 Participants tablet, oral, once a day. pitolisant: Tablet	Placebo n=29 Participants tablet, oral, once a day. Placebo: Tablet
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .	-2.88 score on a scale Standard Error 0.44	-1.12 score on a scale Standard Error 0.64

Adverse Events

Pitolisant

Serious events: 0 serious events

Other events: 15 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Pitolisant n=73 participants at risk tablet, oral, once a day. pitolisant: Tablet	Placebo n=37 participants at risk tablet, oral, once a day. Placebo: Tablet
Nervous system disorders Headache	19.2% 14/73 • Number of events 24 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.	8.1% 3/37 • Number of events 4 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.
Psychiatric disorders Insomnia	6.8% 5/73 • Number of events 5 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.	2.7% 1/37 • Number of events 1 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.
Infections and infestations Influenza	2.7% 2/73 • Number of events 2 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.	5.4% 2/37 • Number of events 2 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.
Infections and infestations Upper respiratory tract infection	0.00% 0/73 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.	8.1% 3/37 • Number of events 5 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.
Gastrointestinal disorders Diarrhoea	0.00% 0/73 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.	5.4% 2/37 • Number of events 2 • 8 to 13 weeks (up to 4 weeks prior randomization, 8 weeks double-blind period and 1 week single-blind period) Other Adverse Events, reported with frequency threshold of 5%, are the most common Treatment Emergent Adverse Events. The open-label period of the study is ongoing, results of the open-label period will be available after completion of the study. Some patients did not receive the correct planned treatment, the safety analyses were performed by actual treatment group: 73 patients in the pitolisant group and 37 patients in the placebo group.

Additional Information

Clinical Project Manager

Bioprojet Pharma

Phone: +33 (0)1 47 03 66 23

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER