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Completed

NCT Number: NCT01944046

Study of Oxytocin in Autism to Improve Reciprocal Social Behaviors

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.

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Key information

Conditions

Autism Spectrum Disorders Autism Spectrum Disorder Autistic Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

3 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Lurie Center for Autism, Massachusetts General Hospital, Boston, Massachusetts, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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 -added part way through study in response to subject death.

Treatment and study plan

Double blind phase Placebo Nasal Spray

Drug

This nasal spray will contain all of the ingredients that are in the active oxytocin spray in the same quantities, except oxytocin will NOT be 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.

Other names: DB Placebo (PL)

double Blind Oxytocin Nasal Spray

Drug

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 weeks 4 and 8 until achieving the target dose of 24 IU BID at week 8. 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. During the open label phase after approximately March 2019 the study used only the 24 IU /0.10 ml formulation and the maximum dose was 72 IU per day.

Other names: DB Intranasal Oxytocin (OT)

Open Label intranasal oxytocin

Drug

All participants who completed the 24 week double blind phase were eligible to join a 24 week open label phase in which all participants received intranasal oxytocin

Other names: open label treatment

Primary outcomes

  1. Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity

    Time frame: Least Mean Squares Double-blind phase: change from baseline to week 24

    The primary outcome is Change in Aberrant Behavior Checklist-Modified Social Withdrawal subscale- a measure of reciprocal social behaviors. ABC-mSW is a modification of the ABC-Lethargy subscale. The ABC-mSW consists of the sum of questions 5,12,16, 20, 23, 26, 30, 37, 40, 42, 43, 55, and 58. In contrast to the ABC-Lethargy subscale it eliminates question 3 (listless, sluggish, inactive), question 32 (sits or stands in one position for a long time), and question 53 (inactive, never moves spontaneously). Thirteen individual items are scored 0-3, therefore the range is 0-39. Higher score indicates lower social reciprocity. Repeated measures were obtained at baseline, weeks 4, 8, 12, 16, 20, 24.

  2. Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity

    Time frame: Least mean squares for Open Label: Change between weeks 24-48

    The ABC-mSW is described above and involves 13 items reflecting lack of reciprocal interaction. Each item is scored from 0 (never shows behavior) to 3 (behavior is a major problem). The range is 0-39. Higher scores indicate worse reciprocal social functioning.

Secondary outcomes

  1. Change in Sociability Factor (SF)

    Time frame: Double-blind phase: change in least means squares between week 0 & 24.

    The Sociability Factor (SF) is a summed measure of the13 items of the ABC-SW and the 18 items of the Pervasive Development Disorders Behavior Inventory-Screening Version (PDDBI-SV).The PDDBI-SV assesses both adaptive social behaviors and social problems typical of ASD. The adaptive behaviors are reverse scored so that all the analyzed scores range from 0-performing in a neurotypical fashion to 3 typically performs in a way associated with ASD. the total # of items on this summed measure is 31 with a range from 0 to 93. More impaired social functioning indicated by higher scores. This measure was changed to a secondary outcome in the final statistical analysis plan.

  2. Change in Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score

    Time frame: Double-blind phase: baseline, weeks 12, 24

    The SRS-Social Motivation subscale was developed to provide a quantitative measure of social impairments typically observed in ASD in children 3-18 years. Reported as T-score with a range of 38-90 for both boys and girls. Higher score indicates more severe clinical condition. Lower value in change indicates more improvement.

  3. Change in Stanford Binet-5th Edition (SB-5) IQ Score

    Time frame: Double-blind phase: baseline to week 24

    Cognitive skills will be assessed using the Stanford Binet-5th Edition (SB-5) (Roid). Acceptable IQ range is 47-153, with higher score being better. Higher change scores indicate more improvement.

Other outcomes

  1. Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score

    Time frame: Open Label: weeks 24, 48

    The SRS-Social Motivation subscale was developed to provide a quantitative measure of social impairments typically observed in ASD in children 3-18 years. Reported as T-score with a range of 38-90 for both boys and girls. Higher score indicates more severe clinical condition. Lower value in change indicates more improvement.

  2. Change in Vineland II Adaptive Behavior Scales (VABS-II) Daily Living Domain Score

    Time frame: Double-blind phase: baseline, week 24; Open Label: week 48

    Functional skills will be assessed using the VABS-II Daily Living Domain Score. Uses standard score with a mean of 100 and SD of 15 with a range of 20-160. Higher score is better. Higher value in change indicates more improvement.

  3. Caregiver Strain Questionnaire (CSQ) Subjective Internalizing Subscale Mean Score

    Time frame: Open Label: weeks 24, 48

    Caregiver questionnaire that assesses the impact of caring for the proband on caregiver and family. Each item on the subjective internalizing CSQ subscale is rated from 1 to 5. Then all items within the subscale are summed and the mean is determined based on the number of items in the subscale. Higher score indicates more caregiver strain. Lower value in change indicates more improvement.

  4. Change in Vineland II Adaptive Behavior Scales (VABS-II) Communication Domain Score

    Time frame: Double-blind phase: baseline, week 24; Open Label: week 48

    Functional skills will be assessed using the VABS-II Communication Domain Score. Uses standard score with a mean of 100 and SD of 15 with a range of 20-160. Higher score is better. Higher value in change indicates more improvement.

  5. Change in Caregiver Strain Questionnaire (CSQ) Subjective Internalizing Subscale Score

    Time frame: Double-blind phase: baseline, week 24

    Caregiver questionnaire that assesses the impact of caring for the proband on caregiver and family. CSQ subscale scores are ranged from 1 to 5. with each item of the subscale having the same range, the sum of the items within the subscale are summed, and the mean score is determined (I.e. a single # between 1 and 5) and reported. Higher scores indicate more caregiver strain. Lower value in change indicates more improvement.

  6. Change in Vineland II Adaptive Behavior Scales (VABS-II) Socialization Domain Score

    Time frame: Double-blind phase: baseline, week 24; Open Label: week 48

    Functional skills will be assessed using the VABS-II Socialization Domain Score. Uses standard score with a mean of 100 and SD of 15 with a range of 20-160. Higher score is better. Higher value in change indicates more improvement.

  7. Change in Caregiver Strain Questionnaire (CSQ) Objective Subscale Score

    Time frame: Double-blind phase: baseline, week 24; Open Label: week 48

    Caregiver questionnaire that assesses the impact of caring for the proband on caregiver and family. CSQ subscale scores are ranged from 1 to 5. Higher score indicates more caregiver strain. Lower value in change indicates more improvement. The analysis directions for the instrument that are used in these analyses are the mean of all the responses in the scale or subscale.

  8. Change in Caregiver Strain Questionnaire (CSQ) Subjective Externalizing Subscale Score

    Time frame: Double-blind phase: baseline, week 24; Open Label: week 48

    Caregiver questionnaire that assesses the impact of caring for the proband on caregiver and family. CSQ subscale scores are ranged from 1 to 5. Higher score indicates more caregiver strain. Lower value in change indicates more improvement.

  9. Change in Vineland II Adaptive Behavior Scales (VABS-II) Composite Score

    Time frame: Double-blind phase: baseline, week 24

    Functional skills including communication will be assessed using the VABS-II Adaptive Behavior Composite Score. Uses standard score with a mean of 100 and SD of 15 with a range of 20-160. Higher score is better. Higher value in change indicates more improvement.

  10. Change in Clinical Global Impressions -Improvement Score (CGI-I)

    Time frame: Double blind phase: change from Baseline to week 12, and week 24. Open label phase change from week 24 to week 48

    The Clinical Global Impressions - Improvement score and Severity score, which is routinely used in pharmacologic clinical trials, will capture the study physician's global impression of response. scores of 1 and 2 are considered as a percentage of total subjects in arm

  11. Reading Mind in the Eyes Test is an Objective Measures of the Extent to Which Verbal Participants With Rudimentary Knowledge of Emotion Names Are Able to Correctly Identify the Emotion Shown in a Black and White Picture of the Eyes and Nose of an Actor.

    Time frame: Double blind phase: change from Baseline and week 24. Note: only those who demonstrated understanding of these concepts were included in sample.

    This computerized task consists of a series of pictures of eyes in which the participant needs to determine which emotion the eyes are expressing from 4 emotions listed along with the picture. The outcome is the % of pictures with correct emotion identified. The range is 0 to 100%. The larger percent identified correctly indicates better ability to perceive emotions. An increase or positive change indicates better ability to identify emotions since baseline.

Sponsors and collaborators

Lead sponsor

Linmarie Sikich

Other

Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Registry information

Official study title

Phase II Study of Oxytocin in Autism to Improve Reciprocal Social Behaviors

Acronym: SOARS-B

Important dates

Study start
2014
Primary completion
2017
Study completion
2017
First posted
Sep 17, 2013
Registry last updated
Jun 18, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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