Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2013-001506-29 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| pitolisant | Experimental | tablet, oral, once a day. |
|
| placebo | Placebo Comparator | tablet, oral, once a day. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| pitolisant | Drug | Tablet |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Giuseppe Plazzi, MD | Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Terveystalo Helsinki Uniklinikka | Helsinki | Finland | ||||
| Hôpital Femme-Mère-Enfant |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36931805 | Derived | 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. |
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Pitolisant | tablet, oral, once a day. pitolisant: Tablet |
| FG001 | Placebo | tablet, oral, once a day. Placebo: Tablet |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-blind Period (0 to 8 Weeks) |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 30, 2020 | Oct 12, 2023 |
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo |
| Drug |
Tablet |
|
| 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. | 8 weeks |
| Bron |
| France |
| Hôpital Gui de Chauliac | Montpellier | France |
| Hôpital Robert Debré | Paris | France |
| Università di Bologna | Bologna | Italy |
| Polikliniek Heemstede Neurologist-Somnologist | Heemstede | Netherlands |
| Scientific-Research Medical Complex "Your Health" | Kazan' | Russia |
| I.M. Sechenov First Moscow State Medical University | Moscow | Russia |
| V.M. Behterev National Medical Research Psychiatry and Neurology Center | Saint Petersburg | Russia |
| Samara Regional Clinical Hospital | Samara | Russia |
| N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1 | Simferopol | Russia |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open-label Period (0 to >11 Months) |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Pitolisant | tablet, oral, once a day. pitolisant: Tablet |
| BG001 | Placebo | tablet, oral, once a day. Placebo: Tablet |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Full Analysis Set | Posted | Geometric Least Squares Mean | Standard Error | score on a scale | 8 weeks |
|
|
| ||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set | Posted | Least Squares Mean | Standard Error | score on a scale | 8 weeks |
|
| |||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set | Posted | Least Squares Mean | Standard Error | score on a scale | 8 weeks |
|
|
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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pitolisant | tablet, oral, once a day. pitolisant: Tablet | 0 | 73 | 0 | 73 | 15 | 73 |
| EG001 | Placebo | tablet, oral, once a day. Placebo: Tablet | 0 | 37 | 0 | 37 | 11 | 37 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Project Manager | Bioprojet Pharma | +33 (0)1 47 03 66 23 | a.viatte@bioprojet.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 15, 2021 | Oct 12, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009290 | Narcolepsy |
| ID | Term |
|---|---|
| D006970 | Disorders of Excessive Somnolence |
| D020919 | Sleep Disorders, Intrinsic |
| D020920 | Dyssomnias |
| D012893 | Sleep Wake Disorders |
| D009422 | Nervous System Diseases |
| D001523 | Mental Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| C516975 | pitolisant |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Finland |
|
| Italy |
|
| France |
|
| Russia |
|
|
|