Clinical trial NCT05423717: Dose-finding Study Assessing the Efficacy, Safety, and Pharmacokinetics of Daridorexant in Subjects Aged 10 to < 18 Years With Insomnia Disorder
Official title: Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Polysomnography, Dose-finding Study Assessing the Efficacy, Safety, and Pharmacokinetics of Multiple-dose Oral Administration of Daridorexant in Pediatric Subjects Aged 10 to < 18 Years With Insomnia Disorder
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Insomnia
Lead sponsor: Idorsia Pharmaceuticals Ltd.
Sponsor class: INDUSTRY
First posted: 2022-06-21
Enrollment: 150
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study assesses the efficacy, safety, and pharmacokinetics of multiple-dose oral administration of daridorexant in pediatric subjects aged 10 to \< 18 years with insomnia disorder.


Eligibility criteria: Inclusion Criteria:

* Signed and dated informed consent form (ICF) from the caregiver, i.e., parent/legal guardian prior to any study mandated procedure, or as per local regulation.
* Written assent must be obtained from subjects of the appropriate age who can give assent, as determined by the caregiver and local regulation or institutional review boards / independent ethics committees.
* Male or female subjects aged ≥ 10 and \< 18 years at the time of signing the ICF.
* Chronic insomnia disorder in accordance with International Classification of Sleep Disorders, 3rd edition (ICSD-3) or insomnia disorder in accordance with Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) criteria at Screening, as supported by statements from the child and/or the caregiver:

  1. Difficulty initiating or maintaining sleep, or early morning awakening with inability to return to sleep,
  2. Sleep difficulty has been present for at least 3 months prior to Screening,
  3. Sleep difficulty occurs at least 3 nights per week,
  4. Persistence of sleep difficulty, despite adequate sleep hygiene or non-pharmacological therapy,
  5. The sleep problem occurs despite adequate age appropriate time and opportunity for sleep,
  6. The sleep problem is not due to the direct pharmacological effects of any concomitant medication (e.g., amphetamines, selective serotonin reuptake inhibitors) as per investigator judgment,
  7. Self-report or caregiver report of poor sleep quality and/or quantity impacting the daytime performance of the subject,
* Sleep Disturbance Scale for Children score \> 16 on the Difficulty Initiating or Maintaining Sleep domain at Screening.
* Adolescent of Child-Bearing Potential:

  1. Negative serum pregnancy test at Screening and a negative urine pregnancy test at Randomization.
  2. Agreement to undertake urine pregnancy tests during the study, as per the schedule of activities and up to 5 days after study treatment discontinuation.
  3. Agreement to use an acceptable effective method of contraception from Screening up to 5 days after study treatment discontinuation.
* Inclusion criteria applicable only to a subset of children with insomnia and comorbid neurodevelopmental disorder (NDD): Must have a documented history of NDD (including autism spectrum disorder or attention deficit hyperactivity disorder) according to DSM-5 criteria, as confirmed by review of medical records, at Screening. Use of central nervous system (CNS) stimulants is allowed if started at least 4 weeks prior to Screening, is stable, and is expected to remain stable during the study until End-of-Treatment. CNS stimulants are recommended to be taken in the morning.

Exclusion Criteria:

* Body weight \< 25 kg.
* Daytime napping ≥ 1 h per day on at least 3 weekdays per week during the 3 months prior to Screening.
* Any lifetime history of sleep-related breathing disorders such as obstructive sleep apnea, based on the subject's medical records. Note: a subject whose breathing disorder has been treated by tonsillectomy/ adenoidectomy remains eligible.
* Any other diagnosed sleep-wake disorder as defined in DSM-5 or ICSD-3 (e.g., restless legs syndrome, circadian rhythm sleep wake disorder, parasomnias, narcolepsy) at Screening.
* Any of the following conditions related to suicidality:

  1. Any suicidal ideation with intent, with or without a plan at Screening, i.e., answering "Yes" to questions 4 or 5 on the suicidal ideation section of the lifetime (Visit 1) and visit (Visit 2) version of the Columbia Suicide Severity Rating Scale© (C-SSRS©). Participants who answer "yes" to any of these questions must be referred to the investigator for follow-up evaluation.
  2. History of suicide attempt on the suicidal behavior section of the lifetime version of the C-SSRS© at Visit 1.
* Any acute or unstable significant medical condition (e.g., seizure disorder, bipolar disorder, schizophrenia), hematology/biochemistry test results, and/or electrocardiogram results deviating from the normal ranges to a clinically relevant extent that would preclude participation in the study or could prevent the subject from complying with study requirements, as per investigator judgement.
* Cognitive behavior therapy for any indication is allowed only if it has been started at least 1 month prior to Visit 2 and is kept stable throughout the study.

Primary outcomes:
measure: Change from baseline to Day 1 in Total Sleep Time (TST) as measured by polysomnography (PSG).
timeFrame: PSG will be performed on 2 nights during the screening period and on Day 1 of the treatment period (total duration: 3 days).
Secondary outcomes:
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Banner - University Medical Center Tucson
state: Arizona
city: Fayetteville
country: United States
facility: Preferred Research Partners NWA, LLC
state: Arkansas
city: Little Rock
country: United States
facility: Preferred Research Partners, Inc.
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Upland
country: United States
facility: Pacific Clinical Research Management Group LLC
state: California
city: Brandon
country: United States
facility: Teradan Clinical Trials
state: Florida
city: Miami
country: United States
facility: D&H National Research Centers, Inc.
state: Florida
city: Miami
country: United States
facility: Hope Research Network
state: Florida
city: Tampa
country: United States
facility: University of South Florida - Tampa General Hospital (TGH)
state: Florida
city: Weston
country: United States
facility: Encore Medical Research of Weston
state: Florida
city: Winter Park
country: United States
facility: Florida Pediatric Research Institute
state: Florida
city: Atlanta
country: United States
facility: NeuroTrials Research, Inc.
state: Georgia
city: Decatur
country: United States
facility: CenExel iResearch, LLC
state: Georgia
city: Stockbridge
country: United States
facility: Clinical Research Institute
state: Georgia
city: Kalamazoo
country: United States
facility: Western Michigan Homer Stryker M.D. School of Medicine
state: Michigan
city: Charlotte
country: United States
facility: OnSite Clinical Solutions, LLC
state: North Carolina
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital of University Hospitals
state: Ohio
city: Columbia
country: United States
facility: Bogan Sleep Consultants, LLC
state: South Carolina
city: Richmond
country: United States
facility: National Clinical Research, Inc
state: Virginia
city: Edegem
country: Belgium
facility: University Hospital Antwerp (Department Pediatrics)
city: Gent
country: Belgium
facility: University Hospital Gent, Pediatric Sleep Center
city: Kozloduy
country: Bulgaria
facility: MC Zdrave 1 Ltd.
city: Plovdiv
country: Bulgaria
facility: University Hospital (UMHAT) Sveti Georgi
city: Razgrad
country: Bulgaria
facility: MC ReSpiro Ltd.
city: Sofia
country: Bulgaria
facility: MC Avitsena Ltd.
city: Sofia
country: Bulgaria
facility: MC Sun I Zdrave ("Sleep and health") Ltd.
city: Sofia
country: Bulgaria
facility: MC Kalimat
city: Sofia
country: Bulgaria
facility: MC Inspiro
city: Varna
country: Bulgaria
facility: MC Sanamedik Ltd.
city: Berlin
country: Germany
facility: Advanced Sleep Research Berlin GmbH
city: Berlin
country: Germany
facility: Charité -Universitätsmedizin Berlin
city: Datteln
country: Germany
facility: Vestische Caritas-Kliniken GmbH, Vestische Kinder- und Jugendklinik Datteln
city: Schwerin
country: Germany
facility: SOMNIBENE Institut für Medizinische Forschung und Schlafmedizin Schwerin GmbH
city: Bari
country: Italy
facility: S.C. Neuropsichiatria Infantile
city: Bologna
country: Italy
facility: IRCCS Ospedale Bellaria
city: Florence
country: Italy
facility: Meyer Children's Hospital
city: Genova
country: Italy
facility: Institute Giannina Gaslini
city: Milano
country: Italy
facility: Ospedale San Paolo
city: Monserrato
country: Italy
facility: Centro di Medicina del Sonno
city: Roma
country: Italy
facility: Sapienza University
city: Barcelona
country: Spain
facility: Centro Médico Teknon - Medicina del Sueño
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital de La Santa Creu I Sant Pau
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Madrid
country: Spain
facility: Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz
city: Móstoles
country: Spain
facility: HM Puerta del Sur, Unidad del Sueño
city: Valencia
country: Spain
facility: Hospital Quironsalud Valencia - Sleep Unit
city: Vitoria-Gasteiz
country: Spain
facility: Hospital Universitario Araba
city: Basel
country: Switzerland
facility: Universitäts-Kinderspital beider Basel (UKBB)
city: Lugano
country: Switzerland
facility: Ospedale Regionale di Lugano Civico - Neurocentro della Svizzera Italiana, Sleep Medicine Unit
Study officials:
affiliation: Idorsia Pharmaceuticals Ltd.
name: Clinical Trials
role: STUDY_DIRECTOR
References: