Pilot Study of Sleep Therapy and Biomarkers in Children With Autism Spectrum Disorders

The purpose of this study is to test the combined effect of melatonin and donepezil on improving sleep and behavior in children with Autism Spectrum Disorders. Melatonin is a natural neurohormone that helps regulate sleep and wake cycles. Donepezil is used to improve mental function for people with Alzheimer's disease. Children with Autism Spectrum Disorders are more likely to have problems sleeping than other children. This difficulty has been linked to daytime behavioral problems and family stress.

Study Overview

• Status: Terminated
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: Melatonin and Donepezil; Other: Placebo

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Stony Brook, New York, United States, 11794
      • Stony Brook University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Both male and female children
• Ages 4 to 17 1/2 years
• Diagnosis with Autism Spectrum Disorder on the basis of the clinical judgment of an autism specialist, preferably using the Autism Diagnostic Observation Schedule or the Autism Diagnostic Interview-Revised.

Exclusion Criteria:

• Abnormal electroencephalogram in the past month
• Cardiovascular problems
• Asthma
• Respiratory disease
• Peptic ulcer disease
• Renal or hepatic dysfunction (abnormal blood urea nitrogen/creatinine or 2 times elevated liver transaminases)
• Urinary tract obstruction
• Underweight (body mass index < 5th percentile compared to age and sex matched population)
• Other serious illness
• Use of any of the following drugs that might interact with study medications (anticholinergics, systemic corticosteroids, phenobarbital, peginterferon, beta-blockers or any drug that may cause arrhythmias). Drugs that induce or inhibit cytochrome P450 2D6 and cytochrome P450 3A4 (enzymes important for drug metabolism) will be allowed because any effects of such medications are likely to be lost in individual variability due to genetic polymorphisms.
• Use of medications affecting sleep with a half-life of 7 days or more. [Children on medications affecting sleep with a half-life of less than 7 days (including melatonin and donepezil) are eligible if they agree to discontinue use for the duration of the trial.]
• Pregnancy and lactation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Group A; Melatonin and donepezil (melatonin 3mg and donepezil 1.25-5mg based on age/weight each night) or placebo for 15 weeks	Drug: Melatonin and Donepezil; The duration depends on group assignment.; Other Names: Aricept; Other: Placebo; The duration depends on group assignment.
Other: Group B; Melatonin and donepezil (melatonin 3mg and donepezil 1.25-5mg based on age/weight each night) or placebo for 15 weeks	Drug: Melatonin and Donepezil; The duration depends on group assignment.; Other Names: Aricept; Other: Placebo; The duration depends on group assignment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lethargy/Social Withdrawal	Measured by subscale of the Aberrant Behavior Checklist	Every 5 weeks for a total of 5 times

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Stereotypic Behavior	Measured by subscale of the Aberrant Behavior Checklist	Every 5 weeks for a total of 5 times
Sleep quality	Measured by the Children's Sleep Habits Questionnaire	Every 5 weeks for a total of 5 times
Clinician Global improvement	Measured by the Clinical Global Impressions	Every 5 weeks for a total of 5 times
Parent Global improvement	Measured by the Parent Reported Global Impressions	Every 5 weeks for a total of 5 times

Collaborators and Investigators

• Sponsor: Stony Brook University
• Investigators: Principal Investigator: Jill Miller-Horn, MD, MS, Stony Brook University

Study record dates

Study Major Dates

• Study Start: June 1, 2015
• Primary Completion (Actual): December 31, 2020
• Study Completion (Actual): January 29, 2021

Study Registration Dates

• First Submitted: June 29, 2015
• First Submitted That Met QC Criteria: June 30, 2015
• First Posted (Estimate): July 1, 2015

Study Record Updates

• Last Update Posted (Actual): April 28, 2021
• Last Update Submitted That Met QC Criteria: April 26, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Keywords: Melatonin; Children; Autism Spectrum Disorder; Donepezil; Sleep disorder
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Neurodevelopmental Disorders; Disease; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Cholinergic Agents; Enzyme Inhibitors; Protective Agents; Antioxidants; Nootropic Agents; Cholinesterase Inhibitors; Melatonin; Donepezil
• Other Study ID Numbers: 65708

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No