Back to resultsEfficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
Primary Purpose
Narcolepsy With Cataplexy, Narcolepsy Without Cataplexy
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
pitolisant
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Narcolepsy With Cataplexy
Eligibility Criteria
Inclusion Criteria:
- 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).
Exclusion Criteria:
- 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.
Sites / Locations
- Terveystalo Helsinki Uniklinikka
- Hôpital Femme-Mère-Enfant
- Hôpital Gui de Chauliac
- Hôpital Robert Debré
- Università di Bologna
- Polikliniek Heemstede Neurologist-Somnologist
- Scientific-Research Medical Complex "Your Health"
- I.M. Sechenov First Moscow State Medical University
- V.M. Behterev National Medical Research Psychiatry and Neurology Center
- Samara Regional Clinical Hospital
- N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
pitolisant
placebo
Arm Description
tablet, oral, once a day.
tablet, oral, once a day.
Outcomes
Primary Outcome Measures
To evaluate the efficacy of pitolisant in reducing residual Excessive Daytime Sleepiness (EDS).
Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS).
To evaluate the efficacy of pitolisant in reducing the number of cataplectic episodes (for patients with cataplexy)
Changes in the average number of cataplexy episodes per weeks (recorded in sleep diary).
To determine safety in children and adolescents - Safety assessment will be done on monitoring of adverse events, physical examination, vital signs, ECG (QTc) and Blood Laboratory tests modifications and the mood appraisal
Safety assessment will be done on monitoring of adverse events (Incidence of Treatment-Emergent Adverse Events), physical examination, vital signs (blood pressure and heart rate), ECG (QTc) and Blood Laboratory tests modifications (Haematology and Biochemistry) and the mood appraisal (Childhood Depression Inventory [CDI] and Columbia-Suicide Severity Rating Scale [C-SSRS]) throughout the study .
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02611687
Brief Title
Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 6, 2016 (Actual)
Primary Completion Date
April 3, 2021 (Actual)
Study Completion Date
December 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioprojet
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Narcolepsy With Cataplexy, Narcolepsy Without Cataplexy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
110 (Actual)
8. Arms, Groups, and Interventions
Arm Title
pitolisant
Arm Type
Experimental
Arm Description
tablet, oral, once a day.
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Description
tablet, oral, once a day.
Intervention Type
Drug
Intervention Name(s)
pitolisant
Other Intervention Name(s)
BF2.649
Intervention Description
Tablet
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Tablet
Primary Outcome Measure Information:
Title
To evaluate the efficacy of pitolisant in reducing residual Excessive Daytime Sleepiness (EDS).
Description
Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS).
Time Frame
8 weeks
Title
To evaluate the efficacy of pitolisant in reducing the number of cataplectic episodes (for patients with cataplexy)
Description
Changes in the average number of cataplexy episodes per weeks (recorded in sleep diary).
Time Frame
8 weeks
Title
To determine safety in children and adolescents - Safety assessment will be done on monitoring of adverse events, physical examination, vital signs, ECG (QTc) and Blood Laboratory tests modifications and the mood appraisal
Description
Safety assessment will be done on monitoring of adverse events (Incidence of Treatment-Emergent Adverse Events), physical examination, vital signs (blood pressure and heart rate), ECG (QTc) and Blood Laboratory tests modifications (Haematology and Biochemistry) and the mood appraisal (Childhood Depression Inventory [CDI] and Columbia-Suicide Severity Rating Scale [C-SSRS]) throughout the study .
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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).
Exclusion Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Giuseppe Plazzi, MD
Organizational Affiliation
Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna
Official's Role
Study Chair
Facility Information:
Facility Name
Terveystalo Helsinki Uniklinikka
City
Helsinki
Country
Finland
Facility Name
Hôpital Femme-Mère-Enfant
City
Bron
Country
France
Facility Name
Hôpital Gui de Chauliac
City
Montpellier
Country
France
Facility Name
Hôpital Robert Debré
City
Paris
Country
France
Facility Name
Università di Bologna
City
Bologna
Country
Italy
Facility Name
Polikliniek Heemstede Neurologist-Somnologist
City
Heemstede
Country
Netherlands
Facility Name
Scientific-Research Medical Complex "Your Health"
City
Kazan
Country
Russian Federation
Facility Name
I.M. Sechenov First Moscow State Medical University
City
Moscow
Country
Russian Federation
Facility Name
V.M. Behterev National Medical Research Psychiatry and Neurology Center
City
Saint Petersburg
Country
Russian Federation
Facility Name
Samara Regional Clinical Hospital
City
Samara
Country
Russian Federation
Facility Name
N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1
City
Simferopol
Country
Russian Federation
12. IPD Sharing Statement
Learn more about this trial
Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
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