The purpose of this research study is to learn about the effects of supplemental intranasal oxytocin as a treatment for improving social difficulties in children and adolescents with autism. This study will also provide additional information about the safety and tolerability of intranasal oxytocin. Investigators expect oxytocin will increase social motivation, improving daily living skills and quality of life.
Full Title of Study: “Phase II Study of Oxytocin in Autism to Improve Reciprocal Social Behaviors”
- Study Type: Interventional
- Study Design
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Primary Purpose: Treatment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Study Primary Completion Date: November 30, 2017
There is a tremendous unmet need for accessible treatments that address core symptoms of ASD and are safe for sustained use. The Study of Oxytocin in ASD to improve Reciprocal Social Behaviors or (SOARS-B) will test a very promising potential treatment−intranasal oxytocin−for ASD's fundamental social communication deficits in a large, group of verbal and nonverbal children. SOARS-B will also provide information about the regulation of DNA methylation and transcription of the oxytocin receptor gene (OXTR), as well as other genes relevant to oxytocin's CNS activity, as a function of time and in response to oxytocin treatment. These data will fill a key gap in our understanding of oxytocin's role in ASD and its ability to alter epigenetic modifications of the OXTR.
- Drug: Placebo Nasal Spray
- This nasal spray will contain all of the ingredients that are in the active oxytocin spray in the same quantities, except there will we no oxytocin added to the solution. It will be packaged using the same container system as the active oxytocin nasal spray. Each bottle’s label will have its own unique nonsequential randomly assigned number and not a lot number to facilitate masking. Dose titration will occur using exactly the same criteria and procedures as for active study drug.
- Drug: Oxytocin Nasal Spray
- Each insufflation will deliver 8 IU or 24 IU of oxytocin. A maximum of 3 insufflations at a time will be required. Dosing will be flexible between 8 IU/day and 80 IU/day, typically in two divided doses delivered in the morning and in the afternoon. Doses will typically increase by 8 IU twice daily (BID) at week 2 and months 1 and 2 until achieving the target dose of 24 IU BID at Month 2. Subsequently doses may be increased in 8 IU BID increments ONLY at each visit until a maximum dose of 40 IU BID is achieved.Each bottle’s label will have its own unique nonsequential randomly assigned number and not a lot number to facilitate masking.
Arms, Groups and Cohorts
- Placebo Comparator: Placebo Nasal Spray
- Active Comparator: Oxytocin Nasal Spray
Clinical Trial Outcome Measures
- Change in ABC-SW subscale–Reciprocal Social Behavior
- Time Frame: Baseline- 2-4-6-8-9/10-12 months
- The primary outcome is reciprocal social behaviors, which will be assessed using two co-primary measures. The first measure is the ABC-SW subscale, which is being used in other clinical trials focusing on the core social and communication symptoms of autism.
- Change in SRS-Social Motivation Subscale
- Time Frame: Baseline-3-6-9-12 months
- Social Responsiveness Scale (SRS)-Social Motivation subscale, was developed to provide a quantitative measure of social impairments typically observed in ASD in children 3-18 years.
- Change in Stanford Binet-5th Edition (SB-5)/Mullen
- Time Frame: Baseline-6 months
- Cognitive skills will be assessed using the Stanford Binet-5th Edition (SB-5) (Roid). If a participant cannot complete the routing tests on the SB-5, they will be assessed using the Mullen.
- Change in Vineland II Adaptive Behavior Composite and all its subscales
- Time Frame: Baseline-3-6-9-12 months
- Functional skills including communication will be assessed using the standard score of the Vineland 2 adaptive behavior composite and all its subscales.
- Change in Caregiver Strain Questionnaire total and subscale scores
- Time Frame: Baseline-6-12 months
- caregiver questionnaire that assesses the impact of caring for the proband on caregiver and family.
- Change in Social Opportunity (Questionaire)
- Time Frame: Baseline-12 months inclusive
- This UNC created form asks parents to rate how frequently their child has the opportunity to interact with different individuals in the community, home, school and daycare/after-school setting. It also asks, of those opportunities that their child has, does their child actually utilize those opportunities to interact with individuals in a social manner.
Participating in This Clinical Trial
- Be between the ages of 3 years 0 months and 17 years 11 months at the time of randomization
- Be diagnosed by clinician experienced in assessment of ASD with autistic disorder, Asperger's syndrome, or PDD-NOS using DSM-V-TR criteria
- Must have clinical diagnosis of ASD confirmed using the Autism Diagnostic Observation Scale (ADOS, Lord et al., 2001)
- Must have clinical diagnosis of ASD confirmed using the Autism Diagnostic Interview-Revised (ADI-R, Rutter, 2003). ASD criteria proposed by Risi (2006). Specifically, subject must be within 1 point of autism criteria on both social and communication domains of the ADI or meet autism criteria in one of these ADI domains and come within 2 points of autism criteria in the other
- Have a guardian who is able to provide informed consent
- If cognitively able, subject must be able to provide informed assent/consent
- Have a known diagnosis of Rett Syndrome or Childhood Disintegrative Disorder, or have marked sensory impairment such as deafness or blindness
- Have active cardiovascular disease or renal disease that is not controlled by medication
- Subjects who are pregnant, lactating, or who refuse to practice contraception if sexually active
- Subjects who have had changes in allied health therapies, behavioral or educational interventions within the two months prior to randomization other than those associated with school holidays
- Subjects who have had changes in psychiatric medications within 4 weeks of randomization
- Subjects who have had previous chronic treatment with oxytocin
- Subjects who have caretakers who are unable to speak English, be consistently present at visits to report on symptoms, or are otherwise judged as unable to comply with the protocol by the data collection site team
- Subjects with active seizures within the 6 months preceding screening or baseline.
Gender Eligibility: All
Minimum Age: 3 Years
Maximum Age: 17 Years
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- Linmarie Sikich
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Provider of Information About this Clinical Study
- Sponsor-Investigator: Linmarie Sikich, Associate professor – Duke University
- Overall Official(s)
- Linmarie Sikich, MD, Principal Investigator, Duke University
Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.