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1. Intranasal oxytocin

Phase 2

Autism Spectrum Disorder | Small molecule | Psychiatry |United Therapeutics Corporation|Last Updated: Mar 2, 2026

Target and mechanism

ModalitySmall molecule

Also known as 1. Intranasal oxytocin spray

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment96

FDA Designations

No designations recorded

Clinical trial landscape

1. Intranasal oxytocin · 1 trial · 2 indications

Phase 2 1
NCT05754073Oxytocin Effects on Bone in Children With Autism Spectrum DisorderAutism Spectrum Disorder
RECRUITING96 Analytics
PHASE2RECRUITING
Oxytocin Effects on Bone in Children With Autism Spectrum Disorder
Autism Spectrum DisorderUnlock trial analytics

Study Endpoints

Primary Endpoints

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

Secondary Endpoints

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
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposePREVENTION

Treatment Arms

ArmTypeDescription
1. Intranasal OxytocinEXPERIMENTALIntranasal 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. PlaceboPLACEBO_COMPARATORIntranasal 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

Interventions

NameTypeDescription
1. Intranasal oxytocin sprayDRUG30 IU, twice daily for 12 months in the experimental arm in double-blinded phase
2. Intranasal placebo sprayDRUG30 IU, twice daily for 12 months in the placebo comparator arm in double-blinded phase
3. Intranasal Oxytocin sprayDRUG30 IU, twice daily for 6 months in both experimental and placebo comparator arm in open-label phase
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Eligibility Criteria

Age Range6 Years to 18 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: 1. Ages 6 to 18 years old at Randomization 2. BMI greater than or equal to the 5th percentile 3. Expert clinical diagnosis of ASD 4. Availability of parent/guardian to provide informed consent Exclusion Criteria: 1. Fragile X, tuberous sclerosis, William's syndrome, Angelman's...

Countries:United States
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Frequently asked questions about 1. Intranasal oxytocin

What is intranasal oxytocin used for in Autism Spectrum Disorder?

Intranasal oxytocin is an investigational small molecule being studied for Autism Spectrum Disorder, specifically for its effects on bone health in children with the condition. It is administered as a nasal spray and is currently in Phase 2 clinical development.

What does intranasal oxytocin target?

Intranasal oxytocin targets the oxytocin receptor, a G protein-coupled receptor involved in social behavior and bone metabolism. By activating this receptor, the drug is hypothesized to influence bone health in children with Autism Spectrum Disorder, though its exact mechanism in this context is still under investigation.

Who makes intranasal oxytocin?

Intranasal oxytocin is being developed by United Therapeutics Corporation, a publicly traded biopharmaceutical company listed on NASDAQ under the ticker symbol UTHR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in Autism Spectrum Disorder.

What phase is intranasal oxytocin in?

Intranasal oxytocin is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 2 trial is recruiting participants to study its effects on bone health in children with Autism Spectrum Disorder.

What clinical trials is intranasal oxytocin in?

Intranasal oxytocin is being studied in a single Phase 2 clinical trial with the identifier NCT05754073, titled 'Oxytocin Effects on Bone in Children With Autism Spectrum Disorder.' This randomized, double-blind, placebo-controlled trial is recruiting 96 participants aged 6 years and older in the United States.

Is intranasal oxytocin the same as intranasal oxytocin spray?

Yes, intranasal oxytocin is also known as intranasal oxytocin spray. Both names refer to the same investigational drug being developed by United Therapeutics Corporation for Autism Spectrum Disorder. The drug is administered via nasal spray and is currently in Phase 2 clinical trials.