A Randomized, Double-blind, Placebo-controlled Study of Intranasal Oxytocin for Bone Health in Children With Autism Spectrum Disorder

2023P000307

This is a randomized, double blind, placebo-controlled study of the effects of intranasal oxytocin on bone health in children with autism spectrum disorder, ages 6-18 years old. Subjects will be randomized to receive intranasal oxytocin or placebo (30 IU, 2 times daily) for 12 months in the double-blind phase, followed by a 6-month open label phase during which all study subjects will receive intranasal oxytocin (30 IU, 2 times daily). Study visits include screening to determine eligibility, followed by study visits at baseline, week 2, and months 6, 12, 18 and phone calls every two weeks for the first two months and monthly thereafter for the duration of the study. Study assessments include history and physical examinations, anthropometric measurements, electrocardiogram (EKG), adverse event monitoring, laboratory tests for chemistries, hormones and biomarkers for bone metabolism, questionnaires regarding diet and exercise, and imaging to assess body composition, bone density and structure.

2023-08-01

2025-10-31

2026-10-31

Recruiting

Early phase I

96

Inclusion Criteria: 1. Ages 6 to 18 years old at Randomization 2. BMI between the 10th-85th percentiles 3. Expert clinical diagnosis of ASD confirmed using the Diagnostic and Statistical Manual of Mental Disorders (DSM) -5 Checklist and a Social Communication Questionnaire (SCQ)-Lifetime 4. Availability of parent/guardian to provide informed consent 5. If cognitively able, the subject must be able to provide informed assent/consent Exclusion Criteria: 1. Fragile X, tuberous sclerosis, and other single gene defects that are syndromic 2. Other conditions that may contribute to low bone density (e.g., hyperprolactinemia, hypogonadism) 3. Medications that may impact bone such as specific anti-seizure medications, oral glucocorticoids, combined hormonal contraception 4. Hyponatremia 5. Creatinine or liver enzymes more than twice the upper limit of the normal range 6. Changes in doses of antipsychotics that can cause hyperprolactinemia within 2 months of the baseline visit 7. Substance use disorder within the last 6 months 8. History of known coronary artery disease, heart failure, reduced ejection fraction, hypertrophic cardiomyopathy, ventricular arrhythmias, or prolonged QT 9. Active seizures within 6 months preceding the Screening visit or the Baseline visit 10. Subjects who are pregnant, lactating, or who refuse contraception if sexually active 11. Subjects who have had previous treatment with OXT (within 2 months of Randomization) 12. Subjects who are not able to cooperate with medication administration, blood drawing, or imaging procedures despite behavior training 13. Caregivers who are unable to speak English, be consistently present at study visits to report on symptoms or, per the judgement of the data collection team, are unable to comply with the protocol

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2023-10-18