A study to find out which dose of daridorexant is safe and effective to treat insomnia in children and adolescents 10 to <18 years old

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Idorsia Pharmaceuticals Ltd.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

5 Dec 2022 → 5 Feb 2026

Decision date (initial)

2024-11-04

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Idorsia Pharmaceuticals Ltd

External identifiers

EU CT number

2024-513885-20-00

EudraCT number

2021-003867-87

ClinicalTrials.gov

NCT05423717

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Dose response, Efficacy

To characterize the dose-response relationship of daridorexant on objective total sleep time (TST) using polysomnography (PSG) in pediatric subjects with insomnia disorder.

Secondary objectives 1

1. To assess the safety and tolerability of daridorexant in pediatric subjects with insomnia disorder.

Conditions and MedDRA coding

Insomnia Disorder

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-002121-PIP03-19

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 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.
2. 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 (IRBs) / independent ethics committees (IECs).
3. Male or female subjects aged ≥ 10 and < 18 years at the time of signing the ICF.
4. Chronic insomnia disorder in accordance with International Classification of Sleep Disorders (ICSD), 3rd edition or insomnia disorder in accordance with 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.
5. Sleep Disturbance Scale for Children (SDSC) score >16 on the Difficulty Initiating or Maintaining Sleep domain at Screening.
6. Adolescent of Child-Bearing Potential (AoCBP): 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.
7. Inclusion criteria applicable only to a subset of children with insomnia and comorbid neurodevelopmental disorder: - Must have a documented history of neurodevelopmental disorder (NDD; including autism spectrum disorder [ASD] or attention deficit hyperactivity disorder [ADHD]) according to DSM-5 criteria, as confirmed by review of medical records, at Screening. Use of CNS stimulants is allowed if started at least 4 weeks prior to Screening, stable and expected to remain stable during the study until EOT. CNS stimulants are recommended to be taken in the morning.

Exclusion criteria 7

1. Body weight < 25 kg.
2. Daytime napping ≥ 1 h per day on at least 3 weekdays per week during the 3 months prior to Screening.
3. 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.
4. 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.
5. 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 4or 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.
6. Any acute or unstable significant medical condition(e.g., seizure disorder, bipolar disorder, schizophrenia), hematology/biochemistry test results, ECG 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.
7. Cognitive behavior therapy (CBT) 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline to Day 1 in TST (in min) as measured by polysomnography (PSG). PSG will be performed on 2 nights during the screening period and on Day 1 of the treatment period (total time frame: 3 days). Baseline is defined as the mean of the 2 PSG nights during the screening period.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Idorsia Pharmaceuticals Ltd.

Sponsor organisation

Idorsia Pharmaceuticals Ltd.

Address

Hegenheimermattweg 91

City

Allschwil

Postcode

4123

Country

Switzerland

Scientific contact point

Organisation

Idorsia Pharmaceuticals Ltd.

Contact name

Idorsia Clinical Trials Information

Public contact point

Organisation

Idorsia Pharmaceuticals Ltd.

Contact name

Idorsia Clinical Trials Information

Third parties 15

Locations

4 EU/EEA countries · 22 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 138 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

10 submissions — initial application plus substantial / non-substantial modifications