Intranasal Oxytocin in Hypothalamic Obesity | NCT02849743 | Trialant
NCT02849743
Sponsor
Shana McCormack, MD
Status
Completed
Last Update Posted
Oct 5, 2022Actual
Enrollment
18Actual
Phase
Phase 2
Conditions
Craniopharyngioma
Hypothalamic Obesity
Interventions
Syntocinon
Placebo (for Syntocinon)
Countries
United States
Protocol Section
Identification Module
NCT ID
Results Section
Participant Flow Module
Pre-assignment Details
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Derived Section
Miscellaneous Info Module
Version Holder
NCT02849743
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
16-012730
Secondary IDs
Not provided
Brief Title
Intranasal Oxytocin in Hypothalamic Obesity
Official Title
Intranasal Oxytocin to Promote Weight Loss in Children, Adolescents, and Adults With Brain Tumors and Hypothalamic Obesity Syndrome
Acronym
Not provided
Organization
Children's Hospital of PhiladelphiaOTHER
Status Module
Record Verification Date
Sep 2022
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Oct 2016
Primary Completion Date
Apr 2021Actual
Completion Date
May 2022Actual
First Submitted Date
Jul 22, 2016
First Submission Date that Met QC Criteria
Jul 26, 2016
First Posted Date
Jul 29, 2016Estimated
Results Waived
Not provided
Results First Submitted Date
May 4, 2022
Results First Submitted that Met QC Criteria
Sep 28, 2022
Results First Posted Date
Oct 5, 2022Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Sep 28, 2022
Last Update Posted Date
Oct 5, 2022Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Shana McCormack, MD, Attending Physician, Children's Hospital of PhiladelphiaSponsor-Investigator
Lead Sponsor
Shana McCormack, MDOTHER
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This research study will test if oxytocin, delivered by nasal spray, will promote weight loss in children, adolescents, and adults with Hypothalamic Obesity as compared to a placebo. The study is divided into two parts. During the first part, subjects will receive either oxytocin or placebo. In the second part, subjects will "cross-over" to receive the other treatment - either oxytocin or placebo. During study visits participants will do blood tests, physical exams, metabolic testing, a MRI scan, and some surveys and questionnaires.
Detailed Description
Not provided
Conditions Module
Conditions
Craniopharyngioma
Hypothalamic Obesity
Keywords
oxytocin
hypothalamic obesity
craniopharyngioma
metabolism
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
18Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Syntocinon (= Oxytocin), then Placebo
Experimental
Week 0 to Week 7: Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Week 8 to Week 11: Washout
Week 12 to Week 20: Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
*Dose Escalation, as appropriate, at 2 Weeks
Drug: Syntocinon
Drug: Placebo (for Syntocinon)
Placebo, then Syntocinon (= Oxytocin)
Experimental
Week 0 to Week 7: Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Week 8 to Week 11: Washout
Week 12 to Week 20: Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Drug: Syntocinon
Drug: Placebo (for Syntocinon)
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Syntocinon
Drug
The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo, then Syntocinon (= Oxytocin)
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Weight Loss
The primary objective of this study is to determine whether treatment with 8 weeks of intranasal OXT (relative to 8 weeks of placebo) will promote weight loss in children and adolescents with brain tumors and hypothalamic obesity syndrome ages 10 to 35 years. Specifically, the primary outcome will be: the difference of the post-treatment weight between the two periods (treatment vs. placebo); the statistical model will include the difference of the baseline weight between the two periods and the sequence (OXT-PBO versus PBO-OXT).
Assessed at the beginning and end of each Intervention Period (Intervention 1: Visit 1 to Visit 4 = 8 Weeks, Intervention 2: Visit 5 to Visit 8 = 8 Weeks)
Secondary Outcomes
Measure
Description
Time Frame
Peripheral Oxytocin Area Under the Curve (AUC)
We will determine the immediate peripheral pharmacokinetics of intranasal oxytocin (versus placebo/endogenous oxytocin). In order to minimize participant burden, each participant had the assessment at one time point with either the low or high dose of oxytocin. And at one point during the placebo block.
For this exploratory outcome, visits representing lower dose and higher dose oxytocin were Combined versus lower and higher dose placebo.
Other Outcomes
Measure
Description
Time Frame
Cognitive Restraint at Test Meal Attributable to Oxytocin vs Placebo
Cognitive Restraint at Test Meal Attributable to Oxytocin vs Placebo measured using stop-signal task, stop signal reaction time (SSRT).
For this exploratory outcome, in order to minimize participant burden, each participant had the assessment at one time point with either the low or high dose of oxytocin. And at one point during the placebo block for comparison.
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Proficient in English.
Males or females age 10 to 35 years, inclusive.
Weight ≥ 51 kg.
Girls must have a negative urine/serum pregnancy test and post-menarchal girls must use an acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study.
Hypothalamic obesity, defined for the purposes of this protocol as:
previously diagnosed with a brain tumor*
currently overweight or obese (BMI > 85%ile for age/sex for < 18 years, BMI > 25 kg/m2 for 18 - 35 years)
has at least one other endocrinopathy, indicating hypothalamic damage
rate of annualized weight gain during any 6 month period (given variability in clinical course) preceding or after diagnosis and treatment greater than 2 standard deviations above population reference ranges for age and sex.
At least 6 months since completion of therapy with stable disease/lack of recurrence.
Stable for at least 2 months on any pituitary replacement (e.g., glucocorticoid, thyroid hormone, estrogen/progestin or testosterone, growth hormone, except for adjustments of less than or equal to 20%). (Desmopressin is not required to be stable for 2 months. Participants with DI taking desmopressin are required to have intact thirst and be well-controlled on their current dosing regimen.)
Stable for at least 2 months on any appetite-modulating medications (e.g., stimulants).
Be able to ambulate independently.
Parental/guardian permission (informed consent) and child assent.
Exclusion Criteria:
Diabetes insipidus without intact thirst mechanism (i.e., history that participant is not thirsty when hypernatremic and/or continues to be thirsty when hyponatremic, by participant/family and/or practitioner report and medical records) and/or "brittle" diabetes insipidus, defined as requiring >1 admission in the past year and/or any admission within the previous 3 months.
Cardiovascular condition, as defined as any of the following: i) abnormal blood pressure, defined as <3%ile or >97%ile for age, sex and height; ii) history of cardiac arrhythmia or arrhythmia detected on screening ECG; iii) history of heart failure and/or cardiomyopathy; iv) prolonged QTc interval (QTc > 460 msec), and/or long QT syndrome phenotype and/or positive genotype for long QT syndrome pathogenic mutations.
Concurrent use of medications known to prolong QTc interval and pose high risk for Torsades de Pointes (TdP) according to the current information available (www.crediblemeds.org). Concomitant medications will be assessed by IDS pharmacist, in collaboration with study cardiologist, if additional clarification is needed. In addition, we require that potential participants be on a stable dose for at least 2 months of any medication with the potential to alter cardiac rhythm to ensure the screening ECG reflects steady-state physiology.
History of liver disease, with screening laboratory studies:
Laboratory values: ALT/SGPT > 3.0X upper limit of normal or AST/SGOT > 3.0X upper limit of normal
History of chronic kidney disease, with screening laboratory studies:
Laboratory values: eGFR < 60 mL/min/1.73m2, as defined by the Schwartz formula
Clinically significant anemia, with screening laboratory studies:
Laboratory values: Hemoglobin < 10 g/dL
Seizure in the past 12 months.
History of gastrectomy, gastric bypass, small or large bowel resection.
History of active substance abuse.
Current psychotic disorder and/or suicidality.
Supra-physiologic (>15 mg/m2/day) prescribed doses of hydrocortisone equivalent.
Anticipated clinical plan to initiate or modify pituitary hormone replacement and/or appetite-modulating drugs during the course of the study.
Any investigational drug use within 30 days prior to enrollment.
Pregnant or lactating females.
Individuals with a known sensitivity to either oxytocin or the components of its formulation.
Inability to take an intranasal medication (e.g., recent injury).
Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
10 Years
Maximum Age
35 Years
Standard Ages
ChildAdult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Shana E McCormack, MD
Children's Hospital of Philadelphia
Principal Investigator
Locations
Facility
Status
City
State
ZIP
Country
Contacts
Children's Hospital of Philadelphia
Philadelphia
Pennsylvania
19104
United States
References Module
No data available
No data is available for this block.
IPD Sharing Statement Module
No data available
No data is available for this block.
Once consent was obtained, participants were reviewed for eligibility and inclusion in the trial. Individuals meeting criteria were randomized. In total, 18 participants signed informed consent and completed in-person screening. 5 of 18 were considered ineligible after screening due to: concomitant medications (2), ECG findings (2), and BMI (1).
Intervention Period 1: Week 0 to Week 7, Washout: Week 8 to Week 11, Intervention Period 2: Week 12 to Week 20.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Syntocinon (= Oxytocin), Then Placebo
Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks) *Dose Escalation, as appropriate, at 2 Weeks
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
FG001
Placebo, Then Syntocinon (= Oxytocin)
Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
Periods
Title
Milestones
Reasons Not Completed
First Intervention (8 Weeks)
Type
Comment
Milestone Data
STARTED
FG0006 subjects
FG0017 subjects
COMPLETED
FG0006 subjects
FG0016 subjects
NOT COMPLETED
FG0000 subjects
FG0011 subjects
Type
Comment
Reasons
Stopping Criteria Met
FG0000 subjects
FG0011 subjects
Washout (4 Weeks)
Type
Comment
Milestone Data
STARTED
FG0006 subjects
FG0016 subjects
COMPLETED
FG0006 subjects
FG001
Second Intervention (8 Weeks)
Type
Comment
Milestone Data
STARTED
FG0006 subjects
FG0016 subjects
COMPLETED
FG0005 subjects
FG001
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Syntocinon (= Oxytocin), Then Placebo
Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks) *Dose Escalation, as appropriate, at 2 Weeks
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
Denominators
Units
Counts
Participants
BG000
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Median
Outcome Measures Module
Outcome Measures
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
Primary
Weight Loss
The primary objective of this study is to determine whether treatment with 8 weeks of intranasal OXT (relative to 8 weeks of placebo) will promote weight loss in children and adolescents with brain tumors and hypothalamic obesity syndrome ages 10 to 35 years. Specifically, the primary outcome will be: the difference of the post-treatment weight between the two periods (treatment vs. placebo); the statistical model will include the difference of the baseline weight between the two periods and the sequence (OXT-PBO versus PBO-OXT).
In order to be included, participants needed data in Intervention 1 (Block 1) and Intervention 2 (Block 2). 10 of 13 Participants with complete data available for analysis.
Posted
Median
Inter-Quartile Range
kg
Assessed at the beginning and end of each Intervention Period (Intervention 1: Visit 1 to Visit 4 = 8 Weeks, Intervention 2: Visit 5 to Visit 8 = 8 Weeks)
ID
Title
Description
OG000
Oxytocin
Adverse Events Module
Frequency Threshold
0
Time Frame
Adverse Event data were collected from the time of informed consent through the duration of participation (Up to 20 Weeks).
Description
The Safety Monitoring Uniform Report Form (SMURF) was administered at each in-person visit and each telephone check-in visit to assess OXT specific side effects. The full SMURF contains a general inquiry, several questions about daily activities, and modified questions specific to potential OXT side effects.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Syntocinon (Intranasal Oxytocin)
1) Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Serious Adverse Events
Not provided
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Fatigue
General disorders
CTCAE (4.0)
Systematic Assessment
More Info Module
Limitations and Caveats
A new package insert for intranasal oxytocin was released indicating a possible risk for QTc prolongation when administered with other medications known to prolong the QTc. In response with advise from our study cardiologist, we revised exclusion criteria, and added post-dose ECG studies to monitor acute effects. These activities impacted recruitment, as did the COVID-19 pandemic, thus target sample size is less than originally planned (see statistical plan for details).
The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
Placebo, then Syntocinon (= Oxytocin)
Syntocinon (= Oxytocin), then Placebo
Assessed during each treatment block: at either initial lower dose at baseline (week 0 & week 12) or increased dose at 2 weeks (week 2 & week 15); 50% of participants at each set.
Intervention 1: Week 2 (Low Dose) or Week 4 (High Dose) and Intervention 2: Week 14 (Low Dose) or Week 16 (High Dose)
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 1 (Week 0 & Week 12), as a % of kcal Offered.
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 1 (Week 0 & Week 12). Total number of calories consumed during the standardized test meal.
Assessed during Intervention 1: Week 0 (Dose 1) and Intervention 2: Week 12 (Dose 1)
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 2 (Week 2 & Week 14), as a % of kcal Offered.
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 2 (Week 2 & Week 14). Total number of calories consumed during the standardized test meal.
Assessed during Intervention 1: Week 2 (Dose 2) and Intervention 2: Week 14 (Dose 2).
Within Participant Difference After Oxytocin vs After Placebo in Resting Energy Expenditure (kcal/kg Lean Body Mass/Day)
Within Participant Difference After Oxytocin vs After Placebo in Resting Energy Expenditure (kcal/kg lean body mass/day). Resting Energy Expenditure (REE) with output of kcal/kg lean body mass/day measured using indirect calorimetry.
Assessed at the end of each treatment block: Week 8 & Week 20.
Respiratory Quotient (VCO2/VO2)
Within Participant Difference After Oxytocin vs Placebo in Respiratory Quotient (RQ) measured using indirect calorimetry.
Assessed at the end of each treatment block: week 8 & week 20.
Within Participant Difference After Oxytocin vs Placebo in % Body Fat
Total % Body Fat measured using dual energy x-ray absorptiometry (DXA).
Assessed at the end of each treatment block: week 8 & week 20.
Measured using MRI-based post-exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) decline exponential time constant.
Assessed at the end of each treatment block.
Within Participant Change in Hyperphagia (Total Score) Attributable to Oxytocin vs Placebo
Measured using the Dykens Hyperphagia Questionnaire.
The Dykens Hyperphagia Questionnaire is a 11-item questionnaire with responses on a scale of 1 to 5. 1 = Not a Problem, 5 = Severe or Frequent Problem. The scale includes three domains: hyperphagic behavior, hyperphagic drive, and hyperphagic severity. There is also a total or overall score which is calculated by combining the scores from each domain. Maximum Possible Score: 55, Minimum Possible Score: 11. Higher scores indicated more severe and/or frequent Hyperphagia.
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
Within Participant Change in Disinhibition of Eating Attributable to Oxytocin vs Placebo
Within Participant Change in Disinhibition of Eating Attributable to Oxytocin vs Placebo. Measured using the Eating Inventory questionnaire.
The Eating Inventory consists of 36 statements where participants respond true or false, 14 questions where participants select a response from 1 (least severe: rarely or not at all) to 4 (most severe: always or very much), one final question askes participants to rate their level of restraint in eating from 1 (no restraint) to 6 (constantly limiting food). The scores are assessed in 3 dimensions: Dietary Restraint (Max Score: 21), Disinhibition (Max Score: 16), and Hunger (Max Score: 14).
Within Participant Change in Scores from the Disinhibition of Eating dimension are reported.
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
Within Participant Change in Mental and Emotional Health Related Quality of Life Attributable to Oxytocin vs Placebo
The National Institute of Neurological Disorders and Stroke (NINDS) quality of life in neurological disorders (Neuro-QoL) scale is a set of self-reported measures to assess health-related quality of life of adults and children.
Individuals rate domains on a scale from 1 (Never/Not at all) to 5 (Always/Very Often). Each response is assigned a value. Values are combined for a total raw score, then converted to a T-Scores (cohort-specific mean 50, SD 10). Higher scores on the sub-scale domains indicate more of the entity. Adult and child scores were combined in analyses.
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GFS) Attributable to Oxytocin vs Placebo
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GFS) Attributable to Oxytocin vs Placebo.
The Family Assessment Device (FAD-GFS) includes 12 statements where individuals select from a scale of 4 responses ranging from Strongly Agree (SA) to Strongly Disagree (SD). Each response has a score ranging from 1 to 4, some of the items are reverse scored (i.e. 1 = 4, 2 = 3, 3 = 2, 4 = 1). Minimum Total Score: 12, Maximum Total Score: 48. The scores for each question are added and then divided by the total number of questions. The Minimum Overall Score: 1, Maximum Overall Score: 4. A score of 2 or above is considered dysfunction.
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
Within Participant Change in Voluntary Physical Activity Attributable to Oxytocin vs Placebo
Measured using the Bone Mineral Density in Childhood Study (BMDCS) physical activity questionnaire.
The Bone Mineral Density in Childhood Study (BMDCS) questionnaire captures the amount of time spent in physical activity. The assessment asks participants to record the number of hours spent in different categories of physical activity (Min: 0 hours per week, Max: 11+ hours per week).
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)
Eye-Tracking Task
Amount of time spent viewing socially relevant versus socially irrelevant stimuli during eye-tracking.
Assessed at the end of each treatment block.
EEG Task
N170 EEG signal during eye-tracking
Assessed at the end of each treatment block.
6 subjects
NOT COMPLETED
FG0000 subjects
FG0010 subjects
6 subjects
NOT COMPLETED
FG0001 subjects
FG0010 subjects
Type
Comment
Reasons
Withdrawal by Subject
FG0001 subjects
FG0010 subjects
BG001
Placebo, Then Syntocinon (= Oxytocin)
Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
BG002
Total
Total of all reporting groups
6
BG0017
BG00213
Inter-Quartile Range
Years
Title
Denominators
Categories
Title
Measurements
BG00017.0(11.9 to 21.4)
BG00115.3(14.3 to 16.0)
BG00215.3(13.3 to 20.6)
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0002
BG0014
BG0026
Male
BG0004
BG0013
BG0027
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0000
BG0012
BG0022
Not Hispanic or Latino
BG0006
BG0015
BG00211
Unknown or Not Reported
BG0000
BG0010
BG0020
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG0020
Asian
BG0000
BG0010
BG0020
Native Hawaiian or Other Pacific Islander
BG0000
BG0010
BG0020
Black or African American
BG0000
BG0010
BG0020
White
BG0005
BG0016
BG00211
More than one race
BG0001
BG0011
BG0022
Unknown or Not Reported
BG0000
BG0010
BG0020
Region of Enrollment
Number
participants
Title
Denominators
Categories
United States
Title
Measurements
BG0006
BG0017
BG00213
Within Participant Change in Body Weight with Oxytocin for 8 Weeks
OG001
Placebo
Within Participant Change in Body Weight with Placebo for 8 Weeks
Units
Counts
Participants
OG00010
OG00110
Title
Denominators
Categories
Baseline
Title
Measurements
OG00088.3(74.7 to 108.6)
OG00191.9(77.9 to 114.6)
Change at 8 Weeks
Title
Measurements
OG0001.2(0.9 to 1.8)
OG0010.9(-0.2 to 1.6)
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Linear
0.92
Least Means Square
-0.5
2-Sided
Other
Secondary
Peripheral Oxytocin Area Under the Curve (AUC)
We will determine the immediate peripheral pharmacokinetics of intranasal oxytocin (versus placebo/endogenous oxytocin). In order to minimize participant burden, each participant had the assessment at one time point with either the low or high dose of oxytocin. And at one point during the placebo block.
For this exploratory outcome, visits representing lower dose and higher dose oxytocin were Combined versus lower and higher dose placebo.
In order to be included participants had to have samples collected during both Intervention 1 and Intervention 2. IV Access was not able to be obtained in some individuals. 7 of 13 Participants had data available for analysis. Specimens were collected during the lower dose (Visit 1 or Visit 5) or the higher dose (Visit 2 or Visit 6). As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
Posted
Median
Inter-Quartile Range
Concentration (min*pg/mL)
Assessed during each treatment block: at either initial lower dose at baseline (week 0 & week 12) or increased dose at 2 weeks (week 2 & week 15); 50% of participants at each set.
ID
Title
Description
OG000
Oxytocin
Within Participant Difference After Oxytocin in Peripheral Oxytocin (Area Under the Curve)
OG001
Placebo
Within Participant Difference After Placebo in Peripheral Oxytocin (Area Under the Curve)
Units
Counts
Participants
OG0007
OG0017
Title
Denominators
Categories
Baseline
Title
Measurements
OG000101792(92722 to 123638)
OG00199110(90692 to 132989)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Linear
0.57
Regression Coefficient
-15784
2-Sided
Other
Other Pre-specified
Cognitive Restraint at Test Meal Attributable to Oxytocin vs Placebo
Cognitive Restraint at Test Meal Attributable to Oxytocin vs Placebo measured using stop-signal task, stop signal reaction time (SSRT).
For this exploratory outcome, in order to minimize participant burden, each participant had the assessment at one time point with either the low or high dose of oxytocin. And at one point during the placebo block for comparison.
In order to be included, participants needed data in Intervention 1 (Block 1) and Intervention 2 (Block 2) in order to be included. 11 of 13 Participants with Data for Analysis. As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
Posted
Median
Inter-Quartile Range
msec
Intervention 1: Week 2 (Low Dose) or Week 4 (High Dose) and Intervention 2: Week 14 (Low Dose) or Week 16 (High Dose)
ID
Title
Description
OG000
Oxytocin
Within participant difference in response time (msec) on SSRT with Oxytocin
Mixed effects model of other outcomes were also constructed. The "treatment duration" variable reflects the time (in days) in within the treatment Block when the outcome was measured.
OG001
Placebo
Within participant difference in response time (msec) on SSRT with Placebo
Mixed effects model of other outcomes were also constructed. The "treatment duration" variable reflects the time (in days) in within the treatment Block when the outcome was measured.
Units
Counts
Participants
OG00011
OG00111
Title
Denominators
Categories
Baseline
Title
Measurements
OG000282(255 to 326)
OG001247(170 to 276)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mixed Models Analysis
0.21
Regression Coefficient
48
2-Sided
Other
Other Pre-specified
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 1 (Week 0 & Week 12), as a % of kcal Offered.
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 1 (Week 0 & Week 12). Total number of calories consumed during the standardized test meal.
In order to be included, participants had to have data in Intervention 1 (Visit 1, Week 0) and Intervention 2 (Visit 5, Week 12). 9 of 13 Participants had data available for analysis. As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
Posted
Median
Inter-Quartile Range
% of total kcal offered
Assessed during Intervention 1: Week 0 (Dose 1) and Intervention 2: Week 12 (Dose 1)
ID
Title
Description
OG000
Oxytocin
Within Participant Difference in Calories Consumed with Oxytocin Dose 1. Total number of kcal eaten, as a % of total kcal offered.
OG001
Placebo
Within Participant Difference in Calories Consumed with Placebo Dose 1. Total number of kcal eaten, as a % of total kcal offered.
Units
Counts
Participants
OG0009
OG0019
Title
Denominators
Categories
Baseline (Week 0 of Intervention)
Title
Measurements
OG00072.4(59.7 to 75.5)
OG00172.6(70.9 to 77.7)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Linear
0.55
Regression Coefficient
-2.8
2-Sided
Other
Other Pre-specified
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 2 (Week 2 & Week 14), as a % of kcal Offered.
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 2 (Week 2 & Week 14). Total number of calories consumed during the standardized test meal.
In order to be included, participants had to have data in Intervention 1 (Visit 2, Week 2) and Intervention 2 (Visit 6, Week 14). 9 of 13 Participants had data available for analysis. As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
Posted
Median
Inter-Quartile Range
% of total kcal offered
Assessed during Intervention 1: Week 2 (Dose 2) and Intervention 2: Week 14 (Dose 2).
ID
Title
Description
OG000
Oxytocin
Within Participant Difference in Calories Consumed with Oxytocin Dose 2.
Total number of kcal eaten, as a % of total kcal offered.
OG001
Placebo
Within Participant Difference in Calories Consumed with Placebo Dose 2.
Total number of kcal eaten, as a % of total kcal offered.
Units
Counts
Participants
OG0009
OG0019
Title
Denominators
Categories
Week 2 of Intervention
Title
Measurements
OG00072.6(66.3 to 78.3)
OG00167.7(55.7 to 78.5)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Linear
0.86
Regression Coefficient
1.2
2-Sided
Other
Other Pre-specified
Within Participant Difference After Oxytocin vs After Placebo in Resting Energy Expenditure (kcal/kg Lean Body Mass/Day)
Within Participant Difference After Oxytocin vs After Placebo in Resting Energy Expenditure (kcal/kg lean body mass/day). Resting Energy Expenditure (REE) with output of kcal/kg lean body mass/day measured using indirect calorimetry.
In order to be included, participants had to have data in Intervention 1 (Visit 4, Week 8) and Intervention 2 (Visit 8, Week 20). 9 of 13 Participants had data available for analysis from the end of both treatment blocks.
Posted
Median
Inter-Quartile Range
kcal/kg lean mass/day
Assessed at the end of each treatment block: Week 8 & Week 20.
ID
Title
Description
OG000
Oxytocin
Within Participant Difference After Oxytocin in Resting Energy Expenditure (kcal/kg lean body mass/day) at the end of the Intervention Period (8 Weeks).
Mixed effects model of other outcomes were also constructed. The "treatment duration" variable reflects the time (in days) in within the treatment Block when the outcome was measured.
OG001
Placebo
Within Participant Difference After Placebo in Resting Energy Expenditure (kcal/kg lean body mass/day) at the end of the Intervention Period (8 Weeks).
Mixed effects model of other outcomes were also constructed. The "treatment duration" variable reflects the time (in days) in within the treatment Block when the outcome was measured.
Units
Counts
Participants
OG0009
OG0019
Title
Denominators
Categories
End of Intervention
Title
Measurements
OG000129.5(118.2 to 148.1)
OG001127.2(108.3 to 137.9)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Linear
0.88
Regression Coefficient
2.4
2-Sided
Other
Other Pre-specified
Respiratory Quotient (VCO2/VO2)
Within Participant Difference After Oxytocin vs Placebo in Respiratory Quotient (RQ) measured using indirect calorimetry.
In order to be included, participants had to have data in Intervention 1 (Visit 4, Week 8) and Intervention 2 (Visit 8, Week 20). 9 of 13 Participants had data available for analysis from the end of both treatment blocks. SSRT was assessed at either the lower dose (Visit 1 or Visit 5) or the higher dose (Visit 2 or Visit 6). As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
Posted
Median
Inter-Quartile Range
Ratio (L C02/L 02)
Assessed at the end of each treatment block: week 8 & week 20.
ID
Title
Description
OG000
Oxytocin
Within Participant Difference After Oxytocin in Respiratory Quotient (RQ) (VCO2/VO2) at the end of the Intervention (8 Weeks).
OG001
Placebo
Within Participant Difference After Placebo in Respiratory Quotient (RQ) (VCO2/VO2) at the end of the Intervention (8 Weeks).
Units
Counts
Participants
OG0009
OG0019
Title
Denominators
Categories
End of Intervention
Title
Measurements
OG0000.8(0.8 to 0.8)
OG0010.8(0.8 to 0.9)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Linear
0.50
Regression Coefficient
-0.02
2-Sided
Other
Other Pre-specified
Within Participant Difference After Oxytocin vs Placebo in % Body Fat
Total % Body Fat measured using dual energy x-ray absorptiometry (DXA).
In order to be included, participants had to have data in Intervention 1 and Intervention 2. 10 of 13 Participants had data available for analysis. As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
Posted
Median
Inter-Quartile Range
percent body fat
Assessed at the end of each treatment block: week 8 & week 20.
ID
Title
Description
OG000
Oxytocin
Within Participant Difference After Oxytocin in % Body Fat
Linear regression model.
OG001
Placebo
Within Participant Difference After Placebo in % Body Fat
Measured using MRI-based post-exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) decline exponential time constant.
Limited data was collected for the skeletal muscle oxidative phosphorylation capacity. The data requires extensive review for quality prior to analysis. The post-processing requirements are extensive. Analyzed data is not available.
Posted
Assessed at the end of each treatment block.
ID
Title
Description
OG000
Within Participant Change in Skeletal Muscle OXPHOS Capacity Attributable to Oxytocin vs Placebo
Within Participant Change in Skeletal Muscle Oxidative Phosphorylation Capacity Attributable to Oxytocin vs Placebo
Units
Counts
Participants
OG0000
Other Pre-specified
Within Participant Change in Hyperphagia (Total Score) Attributable to Oxytocin vs Placebo
Measured using the Dykens Hyperphagia Questionnaire.
The Dykens Hyperphagia Questionnaire is a 11-item questionnaire with responses on a scale of 1 to 5. 1 = Not a Problem, 5 = Severe or Frequent Problem. The scale includes three domains: hyperphagic behavior, hyperphagic drive, and hyperphagic severity. There is also a total or overall score which is calculated by combining the scores from each domain. Maximum Possible Score: 55, Minimum Possible Score: 11. Higher scores indicated more severe and/or frequent Hyperphagia.
In order to be included, participants needed data in Intervention 1 (Block 1) and Intervention 2 (Block 2). 10 of 13 Participants with complete data available for analysis.
Posted
Median
Inter-Quartile Range
score on a scale
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
ID
Title
Description
OG000
Oxytocin
Within Participant Change in Hyperphagia (Total Score) with Oxytocin for 8 Weeks.
Change in overall score on Hyperphagia Questionnaire.
OG001
Placebo
Within Participant Change in Hyperphagia (Total Score) with Placebo for 8 Weeks.
Change in overall score on Hyperphagia Questionnaire.
Units
Counts
Participants
OG00010
OG00110
Title
Denominators
Categories
Baseline
Title
Measurements
OG00020(15.3 to 27)
OG00120.5(16.5 to 23.8)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
Regression, Linear
0.68
Regression Coefficient
-0.7
2-Sided
Other
Other Pre-specified
Within Participant Change in Disinhibition of Eating Attributable to Oxytocin vs Placebo
Within Participant Change in Disinhibition of Eating Attributable to Oxytocin vs Placebo. Measured using the Eating Inventory questionnaire.
The Eating Inventory consists of 36 statements where participants respond true or false, 14 questions where participants select a response from 1 (least severe: rarely or not at all) to 4 (most severe: always or very much), one final question askes participants to rate their level of restraint in eating from 1 (no restraint) to 6 (constantly limiting food). The scores are assessed in 3 dimensions: Dietary Restraint (Max Score: 21), Disinhibition (Max Score: 16), and Hunger (Max Score: 14).
Within Participant Change in Scores from the Disinhibition of Eating dimension are reported.
In order to be included, participants needed data in Intervention 1 (Block 1) and Intervention 2 (Block 2). 10 of 13 Participants had complete data available for analysis.
Posted
Median
Inter-Quartile Range
score on a scale
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
ID
Title
Description
OG000
Oxytocin
Within Participant Change in Disinhibition of Eating with Oxytocin for 8 Weeks. Measured using the Eating Inventory.
OG001
Placebo
Within Participant Change in Disinhibition of Eating with Placebo for 8 Weeks. Measured using the Eating Inventory.
Units
Counts
Participants
OG00010
OG00110
Title
Denominators
Categories
Baseline
Title
Measurements
OG0008.5(4.5 to 10.8)
OG0014(3.3 to 10.5)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
Regression, Linear
0.52
Regression Coefficient
-0.6
2-Sided
Other
Other Pre-specified
Within Participant Change in Mental and Emotional Health Related Quality of Life Attributable to Oxytocin vs Placebo
The National Institute of Neurological Disorders and Stroke (NINDS) quality of life in neurological disorders (Neuro-QoL) scale is a set of self-reported measures to assess health-related quality of life of adults and children.
Individuals rate domains on a scale from 1 (Never/Not at all) to 5 (Always/Very Often). Each response is assigned a value. Values are combined for a total raw score, then converted to a T-Scores (cohort-specific mean 50, SD 10). Higher scores on the sub-scale domains indicate more of the entity. Adult and child scores were combined in analyses.
In order to be included, participants had to have data in Intervention 1 and Intervention 2. 11 of 13 Participants had data available for analysis.
Posted
Median
Inter-Quartile Range
T-Score
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)
ID
Title
Description
OG000
Oxytocin
Within Participant Change in Quality of Life Measures with Oxytocin for 8 Weeks. Measured using the Neuro-QoL scale.
OG001
Placebo
Within Participant Change in Quality of Life Measures with Placebo for 8 Weeks. Measured using the Neuro-QoL scale.
Units
Counts
Participants
OG00011
OG00111
Title
Denominators
Categories
Baseline: Anxiety
Title
Measurements
OG00049.9(47.7 to 52.8)
OG00145.7(37.5 to 55.6)
Change at 8 Weeks: Anxiety
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
Neuro-QoL - Anxiety
Regression, Linear
0.91
Regression Coefficient
-0.2
2-Sided
Other
OG000
Neuro-QoL - Depression
Regression, Linear
Other Pre-specified
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GFS) Attributable to Oxytocin vs Placebo
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GFS) Attributable to Oxytocin vs Placebo.
The Family Assessment Device (FAD-GFS) includes 12 statements where individuals select from a scale of 4 responses ranging from Strongly Agree (SA) to Strongly Disagree (SD). Each response has a score ranging from 1 to 4, some of the items are reverse scored (i.e. 1 = 4, 2 = 3, 3 = 2, 4 = 1). Minimum Total Score: 12, Maximum Total Score: 48. The scores for each question are added and then divided by the total number of questions. The Minimum Overall Score: 1, Maximum Overall Score: 4. A score of 2 or above is considered dysfunction.
In order to be included, participants had to have data in Intervention 1 and Intervention 2. 9 of 13 Participants had data available for analysis. This assessment was administered for participants who lived in the same house as the caregiver who would complete the assessment.
Posted
Median
Inter-Quartile Range
score on a scale
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
ID
Title
Description
OG000
Oxytocin
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GRS) with Oxytocin for 8 Weeks
OG001
Placebo
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GRS) with Placebo for 8 Weeks
Units
Counts
Participants
OG0009
OG0019
Title
Denominators
Categories
Baseline
Title
Measurements
OG0001.6(1 to 2.3)
OG0011.4(1.1 to 1.8)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
Regression, Linear
0.64
Regression Coefficient
0.05
2-Sided
Other
Other Pre-specified
Within Participant Change in Voluntary Physical Activity Attributable to Oxytocin vs Placebo
Measured using the Bone Mineral Density in Childhood Study (BMDCS) physical activity questionnaire.
The Bone Mineral Density in Childhood Study (BMDCS) questionnaire captures the amount of time spent in physical activity. The assessment asks participants to record the number of hours spent in different categories of physical activity (Min: 0 hours per week, Max: 11+ hours per week).
In order to be included, participants had to have data in Intervention 1 and Intervention 2. 10 of 13 Participants had data available for analysis.
Posted
Median
Inter-Quartile Range
hours per week of physical activity
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)
ID
Title
Description
OG000
Oxytocin
Median change reported in physical activity, time spent in any activity (hours per week), with Oxytocin.
OG001
Placebo
Median change reported in physical activity, time spent in any activity (hours per week), with Placebo.
Units
Counts
Participants
OG00010
OG00110
Title
Denominators
Categories
Baseline
Title
Measurements
OG0001.3(1.2 to 2)
OG0011.1(0.3 to 1.5)
Change at 8 Weeks
Title
Measurements
OG000
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
Regression, Linear
0.83
Regression Coefficient
0.05
2-Sided
Other
Other Pre-specified
Eye-Tracking Task
Amount of time spent viewing socially relevant versus socially irrelevant stimuli during eye-tracking.
Limited data was collected for the Eye Tracking Task. The data requires extensive review for quality prior to analysis. The post-processing requirements are extensive. Analyzed data is not available.
Posted
Assessed at the end of each treatment block.
ID
Title
Description
OG000
Within Participant Change in Eye Tracking Attributable to Oxytocin vs Placebo
Within Participant Change in Eye Tracking Attributable to Oxytocin vs Placebo
Units
Counts
Participants
OG0000
Other Pre-specified
EEG Task
N170 EEG signal during eye-tracking
EEG task was not completed due to difficulties with equipment. No data collected for analysis.
Posted
Assessed at the end of each treatment block.
ID
Title
Description
OG000
Syntocinon (= Oxytocin), Then Placebo
Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks) *Dose Escalation, as appropriate, at 2 Weeks
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
OG001
Placebo, Then Syntocinon (= Oxytocin)
Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Intranasal oxytocin, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Syntocinon: The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
Units
Counts
Participants
OG0000
OG0010
0
12
0
12
12
12
EG001
Placebo
1) Intranasal placebo, administered 3 times per day (at mealtimes) for 8 weeks (dosage based on weight: 16 IU to 24 IU; dose escalation, if appropriate, occurs at 2 weeks)
Placebo (for Syntocinon): The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.
0
12
0
12
12
12
EG002
Overall
All Participants from Both Treatment Groups.
0
12
0
12
12
12
EG0002 events2 affected12 at risk
EG0010 events0 affected12 at risk
EG0022 events2 affected12 at risk
Irritability
Psychiatric disorders
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0011 events1 affected12 at risk
EG0022 events2 affected12 at risk
Constipation
Gastrointestinal disorders
CTCAE (4.0)
Systematic Assessment
EG0002 events2 affected12 at risk
EG0010 events0 affected12 at risk
EG0022 events2 affected12 at risk
Diarrhea
Gastrointestinal disorders
CTCAE (4.0)
Systematic Assessment
EG0000 events0 affected12 at risk
EG0013 events3 affected12 at risk
EG0023 events3 affected12 at risk
Rectal Irritation
Gastrointestinal disorders
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0011 events1 affected12 at risk
EG0021 events1 affected12 at risk
Worsening Irritable Bowel Syndrome (IBS)
Gastrointestinal disorders
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0010 events0 affected12 at risk
EG0021 events1 affected12 at risk
Electrocardiogram QT Corrected Interval Prolonged
Cardiac disorders
CTCAE (4.0)
Systematic Assessment
EG0002 events2 affected12 at risk
EG0013 events3 affected12 at risk
EG0025 events5 affected12 at risk
Allergic Rhinitis
Respiratory, thoracic and mediastinal disorders
CTCAE (4.0)
Systematic Assessment
EG0002 events2 affected12 at risk
EG0010 events0 affected12 at risk
EG0022 events2 affected12 at risk
Sore Throat
Respiratory, thoracic and mediastinal disorders
CTCAE (4.0)
Systematic Assessment
EG0002 events2 affected12 at risk
EG0012 events2 affected12 at risk
EG0024 events4 affected12 at risk
Insomnia
Psychiatric disorders
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0011 events1 affected12 at risk
EG0022 events2 affected12 at risk
Dermatitis
Skin and subcutaneous tissue disorders
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0010 events0 affected12 at risk
EG0021 events1 affected12 at risk
Dry Lips
Skin and subcutaneous tissue disorders
CTCAE (4.0)
Systematic Assessment
EG0000 events0 affected12 at risk
EG0010 events0 affected12 at risk
EG0021 events1 affected12 at risk
Eye Disorders - Other (Possible Disc Blurring, Eyelid Twitching)
Eye disorders
CTCAE (4.0)
Systematic Assessment
EG0002 events2 affected12 at risk
EG0010 events0 affected12 at risk
EG0022 events2 affected12 at risk
Myalgia
Musculoskeletal and connective tissue disorders
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0011 events1 affected12 at risk
EG0022 events2 affected12 at risk
Infections - Other (Pharyngitis, Sinusitis)
Infections and infestations
CTCAE (4.0)
Systematic Assessment
EG0000 events0 affected12 at risk
EG0011 events1 affected12 at risk
EG0021 events1 affected12 at risk
Investigations - Other (Low Free T4)
Investigations
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0012 events2 affected12 at risk
EG0023 events3 affected12 at risk
Investigations - Other (Elevated AST)
Investigations
CTCAE (4.0)
Systematic Assessment
EG0001 events1 affected12 at risk
EG0010 events0 affected12 at risk
EG0021 events1 affected12 at risk
Investigations - Other (Elevated Bilirubin)
Investigations
CTCAE (4.0)
Systematic Assessment
EG0000 events0 affected12 at risk
EG0011 events1 affected12 at risk
EG0021 events1 affected12 at risk
Restriction Type
Not provided
Results Disclosure Restriction on PI(s)?
No
Other Details
Not provided
D009380
Neoplasms, Nerve Tissue
D006059
Gonadal Dysgenesis
D012734
Disorders of Sex Development
D014564
Urogenital Abnormalities
D052776
Female Urogenital Diseases
D005261
Female Urogenital Diseases and Pregnancy Complications
D000091642
Urogenital Diseases
D052801
Male Urogenital Diseases
D000013
Congenital Abnormalities
D009358
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D006058
Gonadal Disorders
D004700
Endocrine System Diseases
D007006
Hypogonadism
D006730
Hormones, Hormone Substitutes, and Hormone Antagonists
D010455
Peptides
D000602
Amino Acids, Peptides, and Proteins
NA
(NA to NA)
As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
NA
(NA to NA)
SSRT was assessed at either the lower dose (Visit 1 or Visit 5) or the higher dose (Visit 2 or Visit 6). As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)SSRT was assessed at either the lower dose (Visit 1 or Visit 5) or the higher dose (Visit 2 or Visit 6). As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
NA
(NA to NA)
As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
NA
(NA to NA)
As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
NA
(NA to NA)
As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
NA
(NA to NA)
As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)As this assessment was only administered once per Intervention, we are not able to calculate/report the change at 8 weeks.
NA
(NA to NA)
As this assessment was only administered once per Intervention Period, we are not able to calculate/report the change at 8 weeks.
OG001NA(NA to NA)As this assessment was only administered once per Intervention Period, we are not able to calculate/report the change at 8 weeks.
2
(-0.8 to 2.8)
OG0010(-3.3 to 0.8)
0
(-1.8 to 0.8)
OG0010(-0.8 to 1)
-2
(-4.6 to 0.7)
OG0010(0 to 0)
Baseline: Depression
Title
Measurements
OG00042(36.4 to 49.7)
OG00136.4(36.4 to 49.9)
Change at 8 Weeks: Depression
Title
Measurements
OG0000(0 to 3.1)
OG0010(0 to 1.2)
Baseline: Cognition
Title
Measurements
OG00050.4(48.6 to 56.6)
OG00144(39.8 to 51.7)
Change at 8 Weeks: Cognition
Title
Measurements
OG0000(-4.1 to 0)
OG0011.2(0 to 2)
Baseline: Social Function (Adults) or Peer Interaction (Children)