Trial of Zolpidem for Sleep in Children With Autism

NCT05540574 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: IRB-672211P50HD109861
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to examine the effect of zolpidem on sleep in children and adolescents with Autism Spectrum Disorder (ASD). Zolpidem is a nonbenzodiazepine GABAa receptor agonist drug that acts as a hypnotic. To accomplish this, the investigators will use a randomized double-blind placebo-controlled crossover 8-week study design to examine the effect of zolpidem on sleep physiology as assessed by polysomnography (PSG), actigraphy, circadian rhythm, and clinical measures.

Conditions

Autism; Autism Spectrum Disorder

Keywords

zolpidem; sleep; clinical trial; autism

Outcome Measures

Primary Outcomes (1)

• Change from baseline in sleep architecture as measured by polysomnography (PSG) and/or actigraphy, examples include sleep latency and non-rapid eye movement (NREM)

  Baseline, Week 4 and Week 8

Secondary Outcomes (1)

• Change from baseline in sleep efficiency as measured by actigraphy

  Baseline, Week 4 and Week 8

Other Outcomes (11)

• Change from baseline on Children's Sleep Habits Questionnaire (CSHQ) subscale score

  Baseline, Week 4 and Week 8

• Change from baseline on Aberrant Behavior Checklist, Second Edition (ABC-2) subscale scores

  Baseline, Week 4 and Week 8

• Change from baseline on Parent Sleep Habits Questionnaire Parent (PSHQ) scores

  Baseline, Week 4 and Week 8

Study Arms (2)

Zolpidem, then Placebo

EXPERIMENTAL

Participants will first receive Zolpidem for a 4-week period. A 5 mg dose of Zolpidem will be given at bedtime for one week and then will increase to 10 mg if needed and if well tolerated. Participants will then receive Placebo (fake tablet) for a 4-week period. A 5 mg dose of matching Placebo will be given at bedtime for one week and then will increase to 10 mg if needed and if well tolerated.

Drug: Zolpidem; Drug: Placebo

Placebo, then Zolpidem

EXPERIMENTAL

Participants will first receive Placebo (fake tablet) for a 4-week period. A 5 mg dose of matching Placebo will be given at bedtime for one week and then will increase to 10 mg if needed and if well tolerated. Participants will then receive Zolpidem for a 4-week period. A 5 mg dose of Zolpidem will be given at bedtime for one week and then will increase to 10 mg if needed and if well tolerated.

Drug: Zolpidem; Drug: Placebo

Interventions

Zolpidem DRUG

5mg (and up to10mg) Diphenhydramine given orally

Also known as: Ambien

Placebo, then Zolpidem; Zolpidem, then Placebo

Placebo DRUG

Matching Placebo given orally

Placebo, then Zolpidem; Zolpidem, then Placebo

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participants will meet the following
• Outpatients between 8 and 17 years of age with only 12- 17 years of age at time of consent during year 1
• Diagnostic and Statistical Manual, 5th edition (DSM-5) criteria for Autism Spectrum Disorder (ASD) on the basis of clinical evaluation, confirmed with the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule, 2nd Ed (ADOS-2) or the Childhood Autism Rating Scale, Second Edition (CARS-2)
• Males and females
• Availability of polysomnography (PSG) and/or actigraphy data
• Sleep disturbances as assessed using Children's Sleep Habits Questionnaire (CSHQ) with a score of 41 or higher and sleep efficiency of 85% or less and/or total sleep time less than 7 hours and/or wake after sleep onset of more than 30 minutes as measured by polysomnography (PSG) or actigraphy.
• care provider who can reliably bring participant to clinic visits, provide trustworthy ratings, and interacts with participant on a regular basis
• stable medications for at least 2 weeks, with the exception of Prozac which is required to be stable for at least 4 weeks
• no planned changes in psychosocial and biomedical interventions during the trial
• willingness to provide additional saliva samples and participate in key study procedures (i.e., safety measurements every visit, PSG at weeks 4 and 8, and wear the actigraphy watch for 2 weeks before the beginning of trial as well as during the 8 weeks of the trial)
• requirement of dual protection contraception use in females who are sexually active and are of childbearing potential. Dual use contraceptive methods involve the use of both a hormonal method (oral contraceptives, long-acting reversible contraceptives, etc.) and a barrier method (condoms).

You may not qualify if:

• Participants will be excluded if one or more of the following is met
• active suicidal ideation or DSM-5 diagnosis of severe depression, substance use disorder, schizophrenia, schizoaffective disorder, or psychotic disorder
• unstable medical problems: migraine, asthma, seizure disorder, significant physical illness (e.g., anaphylaxis, serious liver, renal, or cardiac pathology) and hepatic insufficiency
• evidence of a metabolic, or infectious etiology for the participant's autism on the basis of medical history, neurologic history, and available tests for inborn errors of metabolism;
• pregnant or sexually active females not using a reliable method of contraception (urinary tests for pregnancy will be employed in this study)
• individuals taking benzodiazepines, antiepileptic medications when prescribed for seizure disorder/epilepsy, antidepressants, melatonin and centrally-acting antihistamines
• history of hypersensitivity to zolpidem
• history of severe side effects from zolpidem
• history of adequate trial of zolpidem
• current use of any medications known to interact with zolpidem such as medications inhibiting CYP3A4 and CYP1A2
• history of complex sleep-related behaviors
• individuals using alcohol, marijuana and other substances.

Sponsors & Collaborators

• Stanford University: lead
• National Institutes of Health (NIH): collaborator

Study Sites (1)

Stanford University

Stanford, California, 94305-5719, United States

RECRUITING

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Interventions

Zolpidem

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Antonio Y. Hardan, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ryan Villacrucis

CONTACT

(650) 736-1235; acesleepstudy@stanford.edu

Robin Libove, BS

CONTACT

(650) 736-1235; rlibove@stanford.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: BASIC SCIENCE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Psychiatry and Behavioral Sciences

Study Record Dates

• First Submitted: September 12, 2022
• First Posted: September 14, 2022
• Study Start: August 9, 2023
• Primary Completion (Estimated): April 30, 2028
• Study Completion (Estimated): April 30, 2028
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

US (1)