A Study of Oxytocin for the Treatment of Social Impairment in Individuals With High Functioning Autism Spectrum Disorder

A Pilot Study of Oxytocin for the Treatment of Social Impairment in Individuals With High Functioning Autism Spectrum Disorder

This study is an 8-week pilot trial with oxytocin nasal spray (Syntocinon) as a treatment for social impairment in children and adults with high functioning autism spectrum disorders (ASD). The investigators hypothesize that oxytocin will be safe, tolerable, and effective in improving social deficits in individuals with ASD.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder; ASD; Pervasive Developmental Disorder; PDD
• Intervention / Treatment: Drug: Intranasal Oxytocin

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Cambridge, Massachusetts, United States, 02114
      • Massachusetts General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 55 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Males and females between 12 and 55 years of age.
• Meets the DSM-5 diagnostic criteria for Autism Spectrum Disorder (ASD) as established by clinical diagnostic interview
• At least moderate severity of ASD impairment as measured by a raw score of ≥85 on the SRS
• Participants and their parent/guardian must be able to speak and understand English sufficiently to comprehend the nature of the study and to allow for the completion of all study procedures required per protocol.
• Subjects and their parent/guardian must be considered reliable reporters. They must understand the nature of the study and must sign an IRB-approved informed consent form before initiation of any study procedures. Subjects and their parent/guardian must have a level of understanding sufficient to communicate with the investigator and study coordinator, and to cooperate with all tests and examinations required by the protocol.
• Each subject and their parent/guardian must understand the nature of the study and provide written informed assent/consent.
• Subjects with mood, anxiety, or disruptive behavior disorders will be allowed to participate in the study provided they do not meet any exclusionary criteria.
• If the subject is sexually active, he/she must agree to use an acceptable form of birth control during the study. These include:
• Abstinence (no sexual contact)
• A barrier method (diaphragm plus spermicide or a condom plus spermicide) in addition to one of the following methods:
• Consistent use of an approved birth control pill
• Birth control patch
• Injected contraceptives
• Intrauterine device (IUD)

Exclusion Criteria:

• Impaired intellectual functioning and/or impaired spoken language.
• Clinically unstable psychiatric conditions or any serious medical illness, which will be assessed by study clinicians during the psychiatric interview and medical history review. If the clinical assessment suggests a psychiatric or medical condition demanding acute clinical attention, then the subject will be excluded from participating in the trial.
• Clinically unstable psychiatric conditions.
• Any serious medical illness
• Pregnant or nursing females.
• Known hypersensitivity to oxytocin.
• Severe allergies or multiple adverse drug reactions.
• A non-responder or history of intolerance to oxytocin, after treatment at adequate doses as determined by the clinician.
• Subjects with significant nasal pathology (including atrophic rhinitis, recurrent nose bleeds, and history of hypophysectomy).
• Clinically abnormal baseline laboratory values falling significantly outside of the standard reference ranges for a basic metabolic screen.
• Investigator and his/her immediate family defined as the investigator's spouse, parent, child, grandparent, or grandchild.
• Currently enrolled or recently participated (within the past 6 months) in a clinical trial of intranasal oxytocin.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Oxytocin- Participants ages 12-17; Intranasal Oxytocin (brand name Syntocinon) will be administered daily (for a total daily dose of 48 IU) for 8 weeks.	Drug: Intranasal Oxytocin; This study is examining the short-term efficacy and tolerability of intranasal oxytocin for the treatment of social impairment in youths and adults with high-functioning ASD (HF-ASD).; Other Names: Syntocinon
Experimental: Oxytocin- Participants ages 18-55; Intranasal Oxytocin (brand name Syntocinon) will be administered daily (for a total daily dose of 48 IU) for 8 weeks.	Drug: Intranasal Oxytocin; This study is examining the short-term efficacy and tolerability of intranasal oxytocin for the treatment of social impairment in youths and adults with high-functioning ASD (HF-ASD).; Other Names: Syntocinon

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in ASD Symptoms Social Responsiveness Scale 2 (SRS-2) Scale From Baseline to Week 8	Change in ASD symptoms as measured by change from baseline on the Social Responsiveness Scale 2 (SRS-2) scale. The SRS-2 is a 65-item rating scale completed that is used to measure the severity of autism spectrum symptoms as they occur in natural settings. The SRS-2 School Age form is completed by a parent or guardian for patients ages 9-17 and the SRS-2 Adult Self-report is completed by patients ages 18-59. Total raw scores range from 0 to 195, with higher scores indicating increased symptom severity.	8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Quality of Life Enjoyment and Satisfaction (PQ-LES-Q and Q-LES-Q)	A 15-item rating scale will be completed by the participant (ages 18-55) or the participant's parent/guardian (ages 6-17) to assess the quality of life from the subject's perspective.	8 weeks

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Investigators: Principal Investigator: Gagan Joshi, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): October 9, 2017
• Primary Completion (Actual): January 9, 2019
• Study Completion (Actual): January 9, 2019

Study Registration Dates

• First Submitted: November 17, 2016
• First Submitted That Met QC Criteria: December 5, 2016
• First Posted (Estimated): December 7, 2016

Study Record Updates

• Last Update Posted (Actual): July 1, 2024
• Last Update Submitted That Met QC Criteria: June 27, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Keywords: Treatment; Oxytocin; ASD; Pervasive Developmental Disorder; Social Impairment; Autism Spectrum Disorders; Syntocinon; PDD
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Developmental Disabilities; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Oxytocin
• Other Study ID Numbers: 2016P002148

Drug and device information, study documents

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