Autism Spectrum DisorderPhase 2BOXNCT05754073
Oxytocin Effects on Bone in Children With Autism Spectrum Disorder
A Phase 2 study testing 1. Intranasal oxytocin spray and 3. Intranasal Oxytocin spray in people with Autism Spectrum Disorder and Bone Health, run by Elizabeth Austen Lawson at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Autism Spectrum Disorder, Bone Health
- What is being tested?
- 1. Intranasal oxytocin spray and 3. Intranasal Oxytocin spray
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Aug 2023to 31 Aug 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Sarah Smith, DNP, 617-726-3870, mghboxstudy@mgb.org
Elizabeth A Lawson, MD
- Recruiting
University of Virginia Medical Center
Charlottesville, Virginia, 22903
Andrea Marrs, MS, 434-982-0871, misralab@uvahealth.org
Madhusmita Misra, MD,MPH
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Study of Intranasal Oxytocin for Bone Health in Children With Autism Spectrum Disorder
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.
Read the detailed description
The prevalence of autism spectrum disorder (ASD), a group of behaviorally-defined disorders characterized by impaired social interactions and verbal and non-verbal communication, is increasing among children. Studies have shown that children with ASD are at a higher risk for low bone mineral density and fractures. ASD is also characterized by low levels of oxytocin (OXT), a peptide hormone with prosocial effects. In addition, OXT promotes bone formation over resorption and low levels of OXT are associated with poor bone health. Hence, OXT administration represents a potential strategy for improving bone health in children with ASD, particularly during the childhood and adolescent years when bone accrual peaks.
The investigators aim to examine (i) whether intranasal OXT administration vs. placebo increases areal bone mineral density (BMD) and improves overall bone health in children with ASD, and (ii) other pathways whereby OXT may impact bone health favorably.
The investigators will enroll 96 participants 6-18 years old with ASD and randomize them into the intranasal oxytocin vs. placebo groups. The study subjects will undergo 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
- Autism Spectrum Disorder, Bone Health
- Being tested
- 1. Intranasal oxytocin spray, 2. Intranasal placebo spray, 3. Intranasal Oxytocin spray
- Keywords
- Autism spectrum disorder, Bone density, Oxytocin, Peri-pubertal children
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
1. Intranasal oxytocin sprayDrug
30 IU, twice daily for 12 months in the experimental arm in double-blinded phase
2. Intranasal placebo sprayDrug
30 IU, twice daily for 12 months in the placebo comparator arm in double-blinded phase
3. Intranasal Oxytocin sprayDrug
30 IU, twice daily for 6 months in both experimental and placebo comparator arm in open-label phase
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Drug: 2. Intranasal placebo spray; Drug: 3. Intranasal Oxytocin spray |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference between IN OXT vs placebo in 12-month change in whole body less head BMD Z-scores.
Measured: 12 months
Secondary outcomes (5)
Difference between IN OXT vs placebo in 12-month change in areal BMD Z-score at the femoral neck.
Measured: 12 months
Difference between IN OXT vs placebo in 12-month change in radial and tibial cortical area and radial trabecular thickness.
Measured: 12 months
Difference between IN OXT vs placebo in 12-month change in radial and tibial failure load.
Measured: 12 months
Difference between IN OXT vs placebo in 12-month change in bone turnover markers, cortisol.
Measured: 12 months
Difference between IN OXT vs placebo in 12-month change in lean mass and muscle area
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2023
- Study start1 Aug 2023
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 96 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Elizabeth Austen Lawson
- Sponsor type
- University, hospital or other
- Collaborators
- United States Department of Defense, University of Virginia
- Responsible party
- Sponsor-investigator, Elizabeth Austen Lawson, Massachusetts General Hospital
- Investigators
- Elizabeth A Lawson, MD, Principal investigator, Neuroendocrine Unit Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05754073
- Sponsor's protocol ID
- 2023P000307
- Acronym
- BOX
- Status last verified
- Nov 2025
- First submitted
- 22 Feb 2023
- Last updated
- 2 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Study of Intranasal Oxytocin for Bone Health in Children With Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT05754073
- Acronym / sponsor ID
- BOX / 2023P000307
- Conditions
- Autism Spectrum Disorder, Bone Health
- Keywords
- Autism spectrum disorder, Bone density, Oxytocin, Peri-pubertal children
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 96 participants (estimated)
- Lead sponsor
- Elizabeth Austen LawsonUniversity, hospital or other
- Collaborators
- United States Department of Defense, University of Virginia
- Study start
- 1 Aug 2023 (actual)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Aug 2028 (estimated)
- First posted
- 3 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The prevalence of autism spectrum disorder (ASD), a group of behaviorally-defined disorders characterized by impaired social interactions and verbal and non-verbal communication, is increasing among children. Studies have shown that children with ASD are at a higher risk for low bone mineral density and fractures. ASD is also characterized by low levels of oxytocin (OXT), a peptide hormone with prosocial effects. In addition, OXT promotes bone formation over resorption and low levels of OXT are associated with poor bone health. Hence, OXT administration represents a potential strategy for improving bone health in children with ASD, particularly during the childhood and adolescent years when bone accrual peaks.
The investigators aim to examine (i) whether intranasal OXT administration vs. placebo increases areal bone mineral density (BMD) and improves overall bone health in children with ASD, and (ii) other pathways whereby OXT may impact bone health favorably.
The investigators will enroll 96 participants 6-18 years old with ASD and randomize them into the intranasal oxytocin vs. placebo groups. The study subjects will undergo 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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1. Intranasal Oxytocin | Experimental | Drug: 1. Intranasal oxytocin spray, Drug: 3. Intranasal Oxytocin sprayIntranasal 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 |
| 2. Placebo | Placebo comparator | Drug: 2. Intranasal placebo spray, Drug: 3. Intranasal Oxytocin sprayIntranasal 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 |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 1. Intranasal oxytocin spray30 IU, twice daily for 12 months in the experimental arm in double-blinded phase | Drug | 1. Intranasal Oxytocin |
| 2. Intranasal placebo spray30 IU, twice daily for 12 months in the placebo comparator arm in double-blinded phase | Drug | 2. Placebo |
| 3. Intranasal Oxytocin spray30 IU, twice daily for 6 months in both experimental and placebo comparator arm in open-label phase | Drug | 1. Intranasal Oxytocin, 2. Placebo |
Primary outcome
Difference between IN OXT vs placebo in 12-month change in whole body less head BMD Z-scores.
Time frame: 12 months
5 secondary outcomes
Difference between IN OXT vs placebo in 12-month change in areal BMD Z-score at the femoral neck.
Time frame: 12 months
Difference between IN OXT vs placebo in 12-month change in radial and tibial cortical area and radial trabecular thickness.
Time frame: 12 months
Difference between IN OXT vs placebo in 12-month change in radial and tibial failure load.
Time frame: 12 months
Difference between IN OXT vs placebo in 12-month change in bone turnover markers, cortisol.
Time frame: 12 months
Difference between IN OXT vs placebo in 12-month change in lean mass and muscle area
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Elizabeth A Lawson, MD principal investigator
Neuroendocrine Unit Massachusetts General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 1. Intranasal oxytocin spray and 3. Intranasal Oxytocin spray in people with Autism Spectrum Disorder and Bone Health, run by Elizabeth Austen Lawson at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Madhusmita Misra, MD, MPH (434-924-9141, abp6bd@uvahealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Difference between IN OXT vs placebo in 12-month change in whole body less head BMD Z-scores. (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2026.