Oxytocin Effects on Bone in Children With Autism Spectrum Disorder

NCT05754073 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 2023P000307
• Study Type: interventional
• Target: 96
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Bone Health; Autism Spectrum Disorder

Keywords

Autism spectrum disorder; Bone density; Oxytocin; Peri-pubertal children

Outcome Measures

Primary Outcomes (1)

• Difference between IN OXT vs placebo in 12-month change in whole body less head BMD Z-scores.

  12 months

Secondary Outcomes (5)

• Difference between IN OXT vs placebo in 12-month change in areal BMD Z-score at the femoral neck.

  12 months

• Difference between IN OXT vs placebo in 12-month change in radial and tibial cortical area and radial trabecular thickness.

  12 months

• Difference between IN OXT vs placebo in 12-month change in radial and tibial failure load.

  12 months

• Difference between IN OXT vs placebo in 12-month change in bone turnover markers, cortisol.

  12 months

• Difference between IN OXT vs placebo in 12-month change in lean mass and muscle area

  12 months

Study Arms (2)

1. Intranasal Oxytocin

EXPERIMENTAL

Intranasal oxytocin spray (30 IU twice daily) for 12 months in the double-blinded phase followed by intranasal oxytocin spray (30 IU twice daily) for 6-months in the open-label phase

Drug: 1. Intranasal oxytocin spray; Drug: 3. Intranasal Oxytocin spray

2. Placebo

PLACEBO COMPARATOR

Intranasal placebo spray (30 IU twice daily (total 60 IU per day) for 12 months followed by intranasal oxytocin spray (30 IU twice daily) for 6-months in the open-label phase

Drug: 2. Intranasal placebo spray; Drug: 3. Intranasal Oxytocin spray

Interventions

1. Intranasal oxytocin spray DRUG

30 IU, twice daily for 12 months in the experimental arm in double-blinded phase

1. Intranasal Oxytocin

2. Intranasal placebo spray DRUG

30 IU, twice daily for 12 months in the placebo comparator arm in double-blinded phase

2. Placebo

3. Intranasal Oxytocin spray DRUG

30 IU, twice daily for 6 months in both experimental and placebo comparator arm in open-label phase

1. Intranasal Oxytocin; 2. Placebo

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Ages 6 to 18 years old at Randomization
• BMI greater than or equal to the 5th percentile
• Expert clinical diagnosis of ASD
• Availability of parent/guardian to provide informed consent

You may not qualify if:

• Fragile X, tuberous sclerosis, William's syndrome, Angelman's syndrome, Noonan syndrome, and other single gene defects that are syndromic and affect heart or bone density
• Other conditions that may contribute to low bone density (e.g., hypogonadism)
• Medications that may impact bone other than calcium or vitamin D supplementation, other than calcium or vitamin D supplementation, such as specific anti-seizure medications (Phenytoin, Phenobarbital), oral glucocorticoids, hormonal contraceptive injection (Medroxyprogesterone acetate (Depo-Provera)
• Hyponatremia
• Liver enzymes (AST, ALT, and Bilirubin) more than three times the upper limit of the normal range
• Estimated glomerular filtration rate (eGFR) less than 60
• Substance use disorder within the last 6 months
• History of known coronary artery disease, heart failure, reduced ejection fraction, hypertrophic cardiomyopathy, ventricular arrhythmias, or prolonged QT (QTc greater than or equal to 480 msec)
• Active seizures within 6 months preceding the Screening visit or the Baseline visit
• Subjects who are pregnant, lactating, or who refuse contraception if sexually active
• Subjects who have had previous treatment with OXT (within 2 months of Randomization)
• Subjects who are not able to cooperate with medication administration, blood drawing, or imaging procedures despite behavior training
• 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
• Any significant illness, condition, medication, or medical device that the Investigator determines could interfere with study participation and impact data collection or subject safety

Sponsors & Collaborators

• Elizabeth Austen Lawson: lead
• United States Department of Defense: collaborator
• University of Virginia: collaborator

Study Sites (2)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

University of Virginia Medical Center

Charlottesville, Virginia, 22903, United States

RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Elizabeth A Lawson, MD

  Neuroendocrine Unit Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Madhusmita Misra, MD, MPH

CONTACT

434-924-9141; abp6bd@uvahealth.org

Sarah Smith, DNP

CONTACT

617-726-3870; mghboxstudy@mgb.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: All subjects and all study staff except the pharmacist will be blinded to treatment assignment.
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director of the Interdisciplinary Oxytocin Research Program

Study Record Dates

• First Submitted: February 22, 2023
• First Posted: March 3, 2023
• Study Start: August 1, 2023
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2028
• Last Updated: March 2, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)