BridgeMD
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
EU CTIS ID: 2024-515568-30-00
What this study is testing
This trial will characterize the efficacy of pitolisant (5-, 10-, 20-, 40 mg/d in Double Blind Period and 5-, 10-, 15-, 20-, 30-, 40 mg/d in Open Label Period) compared to placebo in showing an incremental improvement to the situation particularly in terms of a reduction of EDS as measured by the UNS, and also on the number of cataplexy episodes, if any.The clinical and biological tolerance of pitolisant will also be checked.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female children from 6 to less than 18 years of age (at V8) suffering from narcolepsy with or without cataplexy - meeting the International Classification of Sleep Disorders (ICSD-3) criteria (Narcolepsy type 1 and 2). Diagnosis confirmed with polysomnography and Multiple Sleep Latency Test for patients without cataplexy (if these examinations were not performed within the last 12 months) (Italy) Male and female children from 6 to less than 18 years of age (at V8) suffering from narcolepsy with or without cataplexy - meeting the International Classification of Sleep Disorders (ICSD-3) criteria (Narcolepsy type 1 and 2). Diagnosis confirmed with polysomnography (to be performed if this examination was not performed within the last 12 months) and Multiple Sleep Latency Test for patients without cataplexy (France)
- PDSS score ≥ 15 during baseline period (V1+V2) / 2.
- Patients should be free of non-authorized medication, in particular psychostimulant treatments as from the screening visit (V0) 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).
- Female pubescent patients shall use a birth control method, judged efficient by the investigator, throughout the study and during the month following treatment discontinuation.
You likely can't join if
- Any other conditions that can be considered the primary causes of EDS: such as sleep related breathing disorders as defined by a sleep Apnea Index ≥ 5 per hour or/and an Apnea/Hypopnea Index ≥ 10 per hour, chronic sleep deprivation, circadian sleep wake rhythm disorder or any other medical or neurological causes that could account for narcolepsy symptoms associated with EDS.
- Pregnancy (defined as positive β-HCG blood test), breast-feeding, or patients and unable to use an efficient method of birth control shall not be included in the study (for pubescent female only).
- Patients with severe hepatic Impairment (Child Pugh C) or with any other significant abnormality in the physical examination or clinical laboratory results.
- Psychiatric and neurological disorders, such as moderate or severe psychosis or dementia, depression, history of seizure disorder or other problem that, 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.
- Prior severe adverse reactions to CNS stimulants.
See the full eligibility criteria
Who can join
- Male and female children from 6 to less than 18 years of age (at V8) suffering from narcolepsy with or without cataplexy - meeting the International Classification of Sleep Disorders (ICSD-3) criteria (Narcolepsy type 1 and 2). Diagnosis confirmed with polysomnography and Multiple Sleep Latency Test for patients without cataplexy (if these examinations were not performed within the last 12 months) (Italy) Male and female children from 6 to less than 18 years of age (at V8) suffering from narcolepsy with or without cataplexy - meeting the International Classification of Sleep Disorders (ICSD-3) criteria (Narcolepsy type 1 and 2). Diagnosis confirmed with polysomnography (to be performed if this examination was not performed within the last 12 months) and Multiple Sleep Latency Test for patients without cataplexy (France)
- PDSS score ≥ 15 during baseline period (V1+V2) / 2.
- Patients should be free of non-authorized medication, in particular psychostimulant treatments as from the screening visit (V0) 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).
- Female pubescent patients shall use a birth control method, judged efficient by the investigator, throughout the study and during the month following treatment discontinuation.
- Patients should benefit from appropriate healthy insurance system (only applicable where mandatory e.g. in France).
What rules you out
- Any other conditions that can be considered the primary causes of EDS: such as sleep related breathing disorders as defined by a sleep Apnea Index ≥ 5 per hour or/and an Apnea/Hypopnea Index ≥ 10 per hour, chronic sleep deprivation, circadian sleep wake rhythm disorder or any other medical or neurological causes that could account for narcolepsy symptoms associated with EDS.
- Pregnancy (defined as positive β-HCG blood test), breast-feeding, or patients and unable to use an efficient method of birth control shall not be included in the study (for pubescent female only).
- Patients with severe hepatic Impairment (Child Pugh C) or with any other significant abnormality in the physical examination or clinical laboratory results.
- Psychiatric and neurological disorders, such as moderate or severe psychosis or dementia, depression, history of seizure disorder or other problem that, 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.
- Prior severe adverse reactions to CNS stimulants.
- Known hypersensitivity to the tested treatment including active substance and excipients.
- The inability to continue daily activities safely, without the use of treatment against EDS.
- Any patient presenting congenital galactosemia, glucose-galactose malabsorption or lactase deficiency due to the presence of lactose in investigational treatments.
- Patients participating in another study or being in a follow–up period for another study.
- Cannot be contacted in case of emergency
- Cataplectic patients treated by anticataplectics (SNRI, SSRI, sodium oxybate) which are not under a stable treatment since at least 4 weeks at the time of inclusion (V2).
- Patients treated for cataplexy or any other pathology, by tricyclic antidepressants (clomipramine, imipramine, mirtazapine, desmethylimipramine and protriptyline) are not authorized because they display histamine H1 receptor antagonist activity.
- The use of pitolisant within a 30-day period prior to initial screening visit (V0) for this trial.
- Current or recent (within one year) history of a substance abuse or dependence disorder including alcohol abuse as defined in Diagnostic and Statistical Manual of Mental Disorders (DSM-IV).
- Any significant abnormality of the electrocardiogram and particularly Fridericia’s QTc interval (QTcF = QT/3√ 60/HR) higher than 450 ms.
- Patients with severe depression (CDI ≥ 16).
- Patient with suicidal risk (C-SSRS).
- Positive urinary drug testing (test applicable to patients from 12 years)
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.