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| ID | Type | Description | Link |
|---|---|---|---|
| 1U01HD073984 | U.S. NIH Grant/Contract | View source | |
| 13-0593 | Other Identifier | UNC |
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| Name | Class |
|---|---|
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | NIH |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DB Placebo Nasal Spray | Placebo Comparator | Placebo treatment during weeks 0-24 double blind phase |
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| DB Oxytocin Nasal Spray | Active Comparator | DB Oxytocin- quadruply masked treatment with intranasal oxytocin during weeks 0-24 of study during double blind phase of study |
|
| open label intranasal oxytocin | Active Comparator | non masked treatment with intranasal oxytocin from weeks 24-48 in those participants who completed first 24 weeks of double blind treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity | 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. | Least Mean Squares Double-blind phase: change from baseline to week 24 |
| Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity | 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. | Least mean squares for Open Label: Change between weeks 24-48 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Sociability Factor (SF) | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score | 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. | Open Label: weeks 24, 48 |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Linmarie Sikich, MD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lurie Center for Autism, Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32777383 | Background | Spanos M, Chandrasekhar T, Kim SJ, Hamer RM, King BH, McDougle CJ, Sanders KB, Gregory SG, Kolevzon A, Veenstra-VanderWeele J, Sikich L. Rationale, design, and methods of the Autism Centers of Excellence (ACE) network Study of Oxytocin in Autism to improve Reciprocal Social Behaviors (SOARS-B). Contemp Clin Trials. 2020 Nov;98:106103. doi: 10.1016/j.cct.2020.106103. Epub 2020 Aug 8. | |
| 37811711 | Derived | Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2. |
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Most data will be available on NDAR, but will not be identifiable.
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| ID | Title | Description |
|---|---|---|
| FG000 | Oxytocin Nasal Spray | Oxytocin 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 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomized to DB |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 19, 2018 |
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Randomized, Double-blind, placebo-controlled clinical trial for 24 weeks. Followed by 24 week open label treatment period in which ALL participants receive Oxytocin
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Quadruple
|
|
| 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. |
|
|
| 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 |
|
|
| Double-blind phase: change in least means squares between week 0 & 24. |
| Change in Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score | 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. | Double-blind phase: baseline, weeks 12, 24 |
| Change in Stanford Binet-5th Edition (SB-5) IQ Score | 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. | Double-blind phase: baseline to week 24 |
| Change in Vineland II Adaptive Behavior Scales (VABS-II) Daily Living Domain Score | 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. | Double-blind phase: baseline, week 24; Open Label: week 48 |
| Caregiver Strain Questionnaire (CSQ) Subjective Internalizing Subscale Mean Score | 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. | Open Label: weeks 24, 48 |
| Change in Vineland II Adaptive Behavior Scales (VABS-II) Communication Domain Score | 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. | Double-blind phase: baseline, week 24; Open Label: week 48 |
| Change in Caregiver Strain Questionnaire (CSQ) Subjective Internalizing Subscale Score | 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. | Double-blind phase: baseline, week 24 |
| Change in Vineland II Adaptive Behavior Scales (VABS-II) Socialization Domain Score | 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. | Double-blind phase: baseline, week 24; Open Label: week 48 |
| Change in Caregiver Strain Questionnaire (CSQ) Objective Subscale Score | 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. | Double-blind phase: baseline, week 24; Open Label: week 48 |
| Change in Caregiver Strain Questionnaire (CSQ) Subjective Externalizing Subscale Score | 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. | Double-blind phase: baseline, week 24; Open Label: week 48 |
| Change in Vineland II Adaptive Behavior Scales (VABS-II) Composite Score | 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. | Double-blind phase: baseline, week 24 |
| Change in Clinical Global Impressions -Improvement Score (CGI-I) | 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 | Double blind phase: change from Baseline to week 12, and week 24. Open label phase change from week 24 to week 48 |
| 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. | 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. | Double blind phase: change from Baseline and week 24. Note: only those who demonstrated understanding of these concepts were included in sample. |
| Mount Sinai School of Medicine |
| New York |
| New York |
| 10029 |
| United States |
| Center for Autism and the Developing Brain | White Plains | New York | 10605 | United States |
| Duke Center for Autism and Brain Development | Durham | North Carolina | 27705 | United States |
| Duke University , Genetics Center | Durham | North Carolina | 27710 | United States |
| Vanderbilt University | Nashville | Tennessee | 37212 | United States |
| Seattle Children's Hospital Research Institute | Seattle | Washington | 98105 | United States |
| 34644471 | Derived | Sikich L, Kolevzon A, King BH, McDougle CJ, Sanders KB, Kim SJ, Spanos M, Chandrasekhar T, Trelles MDP, Rockhill CM, Palumbo ML, Witters Cundiff A, Montgomery A, Siper P, Minjarez M, Nowinski LA, Marler S, Shuffrey LC, Alderman C, Weissman J, Zappone B, Mullett JE, Crosson H, Hong N, Siecinski SK, Giamberardino SN, Luo S, She L, Bhapkar M, Dean R, Scheer A, Johnson JL, Gregory SG, Veenstra-VanderWeele J. Intranasal Oxytocin in Children and Adolescents with Autism Spectrum Disorder. N Engl J Med. 2021 Oct 14;385(16):1462-1473. doi: 10.1056/NEJMoa2103583. |
| FG001 | Placebo Nasal Spray | Placebo 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 be 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. |
| COMPLETED |
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| NOT COMPLETED |
|
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| Open Label Eligible and Consented |
|
|
Double-Blind Full Analysis Set (FAS). The DB FAS was defined as having both baseline and at least one post-baseline ABC-SW assessment. The DB-FAS excludes 7 participants randomized to oxytocin (2 with no baseline ABC-SW and 5 with no post-baseline ABC-SW) and 6 participants assigned to placebo who had no post-baseline ABC-SW.
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| ID | Title | Description |
|---|---|---|
| BG000 | Oxytocin Nasal Spray | Oxytocin 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 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. |
| BG001 | Placebo Nasal Spray | Placebo 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 be 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. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
| ||||||||||||||||
| Aberrant Behavior Checklist Social Withdrawal Subscale Score (ABC-SW) abc-SW (mean | the Aberrant Behavior Scale Social withdrawal score assesses reciprocal social behaviors. It is composed of 13 questions from the Aberrant Behavior Scale Lethargy subscale that consists of 16 questions. The ABC-SW does not include the 3 questions (i.e. 3,32,53) of the Lethargy subscale that refer to sedentary behaviors. Score for each item ranges from 0 (not at all a problem) to 3 (a serious problem). Items are summed to give a minimal score of 0 and a maximal score of 39. Lower Scores are better | Mean | Standard Deviation | units on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Least Mean Squares Double-blind phase: change from baseline to week 24 |
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| Primary | Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity | 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. | Open Label phase participants in OLE FAS excluding 2 participants without usable ABC-sws (either w24 or at least one pw24 ABC-sw. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Least mean squares for Open Label: Change between weeks 24-48 |
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| Secondary | Change in Sociability Factor (SF) | 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. | Full Analysis Set | Posted | Least Squares Mean | Standard Error | score on a scale | Double-blind phase: change in least means squares between week 0 & 24. |
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| Secondary | Change in Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score | 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. | Full Analysis Set | Posted | Mean | Standard Deviation | T-score | Double-blind phase: baseline, weeks 12, 24 |
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| Secondary | Change in Stanford Binet-5th Edition (SB-5) IQ Score | 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. | Participants with scores collected at baseline and week 24. | Posted | Mean | Standard Deviation | score on a scale | Double-blind phase: baseline to week 24 |
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| Other Pre-specified | Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score | 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. | Open Label phase participants | Posted | Mean | Standard Error | T-score | Open Label: weeks 24, 48 |
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| Other Pre-specified | Change in Vineland II Adaptive Behavior Scales (VABS-II) Daily Living Domain Score | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Double-blind phase: baseline, week 24; Open Label: week 48 |
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| Other Pre-specified | Caregiver Strain Questionnaire (CSQ) Subjective Internalizing Subscale Mean Score | 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. | Full Analysis Set participants who entered double blind and have values at week 24 AND 48 | Posted | Mean | Standard Deviation | mean score on a scale | Open Label: weeks 24, 48 |
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| Other Pre-specified | Change in Vineland II Adaptive Behavior Scales (VABS-II) Communication Domain Score | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Double-blind phase: baseline, week 24; Open Label: week 48 |
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| Other Pre-specified | Change in Caregiver Strain Questionnaire (CSQ) Subjective Internalizing Subscale Score | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Double-blind phase: baseline, week 24 |
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| Other Pre-specified | Change in Vineland II Adaptive Behavior Scales (VABS-II) Socialization Domain Score | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Double-blind phase: baseline, week 24; Open Label: week 48 |
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| Other Pre-specified | Change in Caregiver Strain Questionnaire (CSQ) Objective Subscale Score | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Double-blind phase: baseline, week 24; Open Label: week 48 |
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| Other Pre-specified | Change in Caregiver Strain Questionnaire (CSQ) Subjective Externalizing Subscale Score | 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. | Full Analysis Set | Posted | Mean | Standard Error | score on a scale | Double-blind phase: baseline, week 24; Open Label: week 48 |
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| Other Pre-specified | Change in Vineland II Adaptive Behavior Scales (VABS-II) Composite Score | 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. | Full Analysis Set | Posted | Mean | Standard Deviation | score on a scale | Double-blind phase: baseline, week 24 |
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| Other Pre-specified | Change in Clinical Global Impressions -Improvement Score (CGI-I) | 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 | full analysis set | Posted | Number | percentage of participants in arm | Double blind phase: change from Baseline to week 12, and week 24. Open label phase change from week 24 to week 48 |
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| Other Pre-specified | 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. | 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. | only subjects who could define feelings from fluently verbal subgroup | Posted | Mean | Standard Deviation | percentage of correct responses | Double blind phase: change from Baseline and week 24. Note: only those who demonstrated understanding of these concepts were included in sample. |
|
Through post-treatment follow-up, approximately 55 weeks.
Adverse events collected on Safety Population (subjects who started each phase).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Oxytocin Nasal Spray: Double-blind Phase | Oxytocin 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 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. | 0 | 146 | 2 | 146 | 120 | 146 |
| EG001 | Placebo Nasal Spray: Double-blind Phase | Placebo 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 be 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. | 0 | 144 | 1 | 144 | 120 | 144 |
| EG002 | Oxytocin Nasal Spray: Open Label Phase | Oxytocin 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 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. | 1 | 127 | 4 | 127 | 91 | 127 |
| EG003 | Placebo Nasal Spray: Open Label Phase | Placebo 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 be 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. | 0 | 122 | 0 | 122 | 92 | 122 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Seizures | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Dysphoria | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Appendicitis | General disorders | MedDRA | Non-systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Negativism | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Aggression Or Hostility | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Anger Or Irritability | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Frustration | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Crying | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Inappropriate Affect | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Psychomotor Hyperactivity | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Impulse-Control Disorder | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Disturbance In Attention | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Mood Swings | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Obsessive Rumination | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Apathy Or Boredom | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Depressed Mood | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Perseveration | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Tic | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Nightmares | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Suicidal Ideation | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Delusion | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Mania Or Hypomania | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Pressure Of Speech | Psychiatric disorders | MedDRA | Non-systematic Assessment |
| |
| Agitation | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Initial Insomnia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Stereotypy | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Terminal Insomnia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Middle Insomnia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Restlessness | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Hypersomnia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Confusional State | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Speech Disorder | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Dyskinesia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Dystonia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Paresthesia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Ganglion Cyst | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Photophobia | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Seizure Or Convulsion | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Coordination Abnormal | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Sleep Walking | Nervous system disorders | MedDRA | Non-systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA | Non-systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Abdominal Pain Or Discomfort | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Vomiting Or Nausea | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Tooth Disorder | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Encopresis | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Dry Mouth | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Salivary Hypersecretion | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Anorectal Discomfort | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA | Non-systematic Assessment |
| |
| Increased Appetite | Metabolism and nutrition disorders | MedDRA | Non-systematic Assessment |
| |
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA | Non-systematic Assessment |
| |
| Abnormal Weight Gain | Metabolism and nutrition disorders | MedDRA | Non-systematic Assessment |
| |
| Abnormal Weight Loss | Metabolism and nutrition disorders | MedDRA | Non-systematic Assessment |
| |
| Thirst | Metabolism and nutrition disorders | MedDRA | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Wart | Skin and subcutaneous tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
| |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
| |
| Nasal Discomfort | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
| |
| Rhinitis Nos | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
| |
| Energy Increased | General disorders | MedDRA | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Non-systematic Assessment |
| |
| Swelling | General disorders | MedDRA | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Non-systematic Assessment |
| |
| Chest Pain Or Discomfort | General disorders | MedDRA | Non-systematic Assessment |
| |
| Night Sweats | General disorders | MedDRA | Non-systematic Assessment |
| |
| Allergic Hypersensitivity | Immune system disorders | MedDRA | Non-systematic Assessment |
| |
| Enuresis | Renal and urinary disorders | MedDRA | Non-systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA | Non-systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA | Non-systematic Assessment |
| |
| Hematuria | Renal and urinary disorders | MedDRA | Non-systematic Assessment |
| |
| Micturition Frequency Decreased | Renal and urinary disorders | MedDRA | Non-systematic Assessment |
| |
| Injury | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
| |
| Intentional Self-Injury | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
| |
| Fracture | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA | Non-systematic Assessment |
| |
| Ear Ache | Ear and labyrinth disorders | MedDRA | Non-systematic Assessment |
| |
| Ear Congestion | Ear and labyrinth disorders | MedDRA | Non-systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA | Non-systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA | Non-systematic Assessment |
| |
| Epistaxis | Vascular disorders | MedDRA | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA | Non-systematic Assessment |
| |
| Libido Increased | Reproductive system and breast disorders | MedDRA | Non-systematic Assessment |
| |
| Breast Pain | Reproductive system and breast disorders | MedDRA | Non-systematic Assessment |
| |
| Menstrual Disorder | Reproductive system and breast disorders | MedDRA | Non-systematic Assessment |
| |
| Blepharospasm | Eye disorders | MedDRA | Non-systematic Assessment |
| |
| Dry Eye | Eye disorders | MedDRA | Non-systematic Assessment |
| |
| Eye Discomfort | Eye disorders | MedDRA | Non-systematic Assessment |
| |
| Visual Acuity Reduced | Eye disorders | MedDRA | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Growing Pains | Musculoskeletal and connective tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Specific Muscle Pain | Musculoskeletal and connective tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Increased Tendency To Bruise | Blood and lymphatic system disorders | MedDRA | Non-systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA | Non-systematic Assessment |
| |
| Dizziness | Cardiac disorders | MedDRA | Non-systematic Assessment |
| |
| Cardiac Rhythm Abnormality | Cardiac disorders | MedDRA | Non-systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA | Non-systematic Assessment |
| |
| Enamel Discoloration | General disorders | MedDRA | Non-systematic Assessment |
| |
| Pituitary Analyses Anterior | Investigations | MedDRA | Non-systematic Assessment |
| |
| Abnormal Urinalysis | Investigations | MedDRA | Non-systematic Assessment |
| |
| Thrombocytopenia | Investigations | MedDRA | Non-systematic Assessment |
| |
| Precocious Puberty | Endocrine disorders | MedDRA | Non-systematic Assessment |
| |
| Skeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA | Non-systematic Assessment |
| |
| Sore Throat | Respiratory, thoracic and mediastinal disorders | MedDRA | Non-systematic Assessment |
|
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Linmarie Sikich, M.D. | Duke University | 919-681-0026 | linmarie.sikich@duke.edu |
| May 27, 2021 |
| Prot_SAP_001.pdf |
| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| D001321 | Autistic Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
Not provided
Not provided
| Clinical Worsening |
|
| Withdrawal by Subject |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Double-blind baseline-w12 |
|
| DB w0- w16 |
|
| DB w0- w20 |
|
| DB w0-w24 |
|
Placebo 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 be 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. |
|
|
|
| OG001 | Placebo Nasal Spray | Placebo 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 be 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. |
|
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Placebo 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 be 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. |
|
|
|
|
Placebo 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 be 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. |
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Placebo
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 be 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.
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| OG001 | Placebo Nasal Spray | Placebo 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 be 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. |
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