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CompletedNCT02849743Updated Oct 5, 2022Results posted

Intranasal Oxytocin in Hypothalamic Obesity

A Phase 2 interventional study of Syntocinon and Placebo (for Syntocinon) in Craniopharyngioma and Hypothalamic Obesity, sponsored by Shana McCormack, MD. Completed at 1 site in United States. Open to participants aged 10 Years to 35 Years. Per ClinicalTrials.gov, last updated 2022-10-05.

Sponsored by Shana McCormack, MD · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
18
Allocation
Randomized
Ages
10 Years to 35 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Craniopharyngioma
  • Hypothalamic Obesity

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Keywords

  • oxytocin
  • hypothalamic obesity
  • craniopharyngioma
  • metabolism
03

In context

Craniopharyngioma

66 studies on the registry are indexed under Craniopharyngioma; 19 are open to participants now.

This study's enrollment of 18 is below the median of 41 across 46 interventional studies indexed under Craniopharyngioma.

Browse Craniopharyngioma studies →

Lead sponsor

Shana McCormack, MD is the lead sponsor of 2 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
10 Years to 35 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Proficient in English.
  2. Males or females age 10 to 35 years, inclusive.
  3. Weight ≥ 51 kg.
  4. 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.

  5. 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.
  6. At least 6 months since completion of therapy with stable disease/lack of recurrence.
  7. 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.)
  8. Stable for at least 2 months on any appetite-modulating medications (e.g., stimulants).
  9. Be able to ambulate independently.
  10. Parental/guardian permission (informed consent) and child assent.

Exclusion criteria

Exclusion Criteria:

  1. 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.
  2. Diabetes mellitus requiring insulin or insulin secretagogue. Laboratory values: HgbA1c ≥8%
  3. 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.
  4. 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.

  5. 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

  6. History of chronic kidney disease, with screening laboratory studies:

    Laboratory values: eGFR \< 60 mL/min/1.73m2, as defined by the Schwartz formula

  7. Clinically significant anemia, with screening laboratory studies:

    Laboratory values: Hemoglobin \< 10 g/dL

  8. Seizure in the past 12 months.
  9. History of gastrectomy, gastric bypass, small or large bowel resection.
  10. History of active substance abuse.
  11. Current psychotic disorder and/or suicidality.
  12. Supra-physiologic (>15 mg/m2/day) prescribed doses of hydrocortisone equivalent.
  13. Anticipated clinical plan to initiate or modify pituitary hormone replacement and/or appetite-modulating drugs during the course of the study.
  14. Any investigational drug use within 30 days prior to enrollment.
  15. Pregnant or lactating females.
  16. Individuals with a known sensitivity to either oxytocin or the components of its formulation.
  17. Inability to take an intranasal medication (e.g., recent injury).
  18. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Crossover assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
18 participants (actual)

Study arms

  • Experimental
    Syntocinon (= Oxytocin), then Placebo

    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)

  • Experimental
    Placebo, then Syntocinon (= Oxytocin)

    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

  • DrugSyntocinon

    The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.

  • DrugPlacebo (for Syntocinon)

    The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.

06

What researchers measure

Primary outcomes

  1. 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).

    Time frame: 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

  1. 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.

    Time frame: 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.

Other outcomes

  1. 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.

    Time frame: Intervention 1: Week 2 (Low Dose) or Week 4 (High Dose) and Intervention 2: Week 14 (Low Dose) or Week 16 (High Dose)

  2. 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.

    Time frame: Assessed during Intervention 1: Week 0 (Dose 1) and Intervention 2: Week 12 (Dose 1)

  3. 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.

    Time frame: Assessed during Intervention 1: Week 2 (Dose 2) and Intervention 2: Week 14 (Dose 2).

  4. 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.

    Time frame: Assessed at the end of each treatment block: Week 8 & Week 20.

  5. Respiratory Quotient (VCO2/VO2)

    Within Participant Difference After Oxytocin vs Placebo in Respiratory Quotient (RQ) measured using indirect calorimetry.

    Time frame: Assessed at the end of each treatment block: week 8 & week 20.

  6. Within Participant Difference After Oxytocin vs Placebo in % Body Fat

    Total % Body Fat measured using dual energy x-ray absorptiometry (DXA).

    Time frame: Assessed at the end of each treatment block: week 8 & week 20.

  7. Skeletal Muscle Oxidative Phosphorylation (OXPHOS) Capacity

    Measured using MRI-based post-exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) decline exponential time constant.

    Time frame: Assessed at the end of each treatment block.

  8. 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.

    Time frame: Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).

  9. 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.

    Time frame: Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).

  10. 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.

    Time frame: Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)

  11. 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.

    Time frame: Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).

  12. 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).

    Time frame: Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)

  13. Eye-Tracking Task

    Amount of time spent viewing socially relevant versus socially irrelevant stimuli during eye-tracking.

    Time frame: Assessed at the end of each treatment block.

  14. EEG Task

    N170 EEG signal during eye-tracking

    Time frame: Assessed at the end of each treatment block.

07

Results

Posted Oct 5, 2022
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).

Participant flow

First Intervention (8 Weeks)
Participant flow — First Intervention (8 Weeks)
MilestoneSyntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)
Started67
Completed66
Not completed01
Withdrew: Stopping criteria met01
Washout (4 Weeks)
Participant flow — Washout (4 Weeks)
MilestoneSyntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)
Started66
Completed66
Not completed00
Second Intervention (8 Weeks)
Participant flow — Second Intervention (8 Weeks)
MilestoneSyntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)
Started66
Completed56
Not completed10
Withdrew: Withdrawal by subject10

Outcome measures

PrimaryWeight 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).

Time frame:
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)
Reported as:
Median · kg
Weight Loss
kgOxytocinPlacebo
Baseline88.3 (74.7 to 108.6)91.9 (77.9 to 114.6)
Change at 8 Weeks1.2 (0.9 to 1.8)0.9 (-0.2 to 1.6)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.92 · Least means square: -0.5
SecondaryPeripheral 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.

Time frame:
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.
Reported as:
Median · Concentration (min*pg/mL)
Peripheral Oxytocin Area Under the Curve (AUC)
Concentration (min*pg/mL)OxytocinPlacebo
Baseline101792 (92722 to 123638)99110 (90692 to 132989)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.57 · Regression coefficient: -15784
Other pre-specifiedCognitive 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.

Time frame:
Intervention 1: Week 2 (Low Dose) or Week 4 (High Dose) and Intervention 2: Week 14 (Low Dose) or Week 16 (High Dose)
Reported as:
Median · msec
Cognitive Restraint at Test Meal Attributable to Oxytocin vs Placebo
msecOxytocinPlacebo
Baseline282 (255 to 326)247 (170 to 276)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Mixed Models Analysis · p = 0.21 · Regression coefficient: 48
Other pre-specifiedWithin 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.

Time frame:
Assessed during Intervention 1: Week 0 (Dose 1) and Intervention 2: Week 12 (Dose 1)
Reported as:
Median · % of total kcal offered
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 1 (Week 0 & Week 12), as a % of kcal Offered.
% of total kcal offeredOxytocinPlacebo
Baseline (Week 0 of Intervention)72.4 (59.7 to 75.5)72.6 (70.9 to 77.7)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.55 · Regression coefficient: -2.8
Other pre-specifiedWithin 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.

Time frame:
Assessed during Intervention 1: Week 2 (Dose 2) and Intervention 2: Week 14 (Dose 2).
Reported as:
Median · % of total kcal offered
Within Participant Difference in Calories Consumed Attributable to Oxytocin vs Placebo Dose 2 (Week 2 & Week 14), as a % of kcal Offered.
% of total kcal offeredOxytocinPlacebo
Week 2 of Intervention72.6 (66.3 to 78.3)67.7 (55.7 to 78.5)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.86 · Regression coefficient: 1.2
Other pre-specifiedWithin 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.

Time frame:
Assessed at the end of each treatment block: Week 8 & Week 20.
Reported as:
Median · kcal/kg lean mass/day
Within Participant Difference After Oxytocin vs After Placebo in Resting Energy Expenditure (kcal/kg Lean Body Mass/Day)
kcal/kg lean mass/dayOxytocinPlacebo
End of Intervention129.5 (118.2 to 148.1)127.2 (108.3 to 137.9)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.88 · Regression coefficient: 2.4
Other pre-specifiedRespiratory Quotient (VCO2/VO2)

Within Participant Difference After Oxytocin vs Placebo in Respiratory Quotient (RQ) measured using indirect calorimetry.

Time frame:
Assessed at the end of each treatment block: week 8 & week 20.
Reported as:
Median · Ratio (L C02/L 02)
Respiratory Quotient (VCO2/VO2)
Ratio (L C02/L 02)OxytocinPlacebo
End of Intervention0.8 (0.8 to 0.8)0.8 (0.8 to 0.9)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.50 · Regression coefficient: -0.02
Other pre-specifiedWithin Participant Difference After Oxytocin vs Placebo in % Body Fat

Total % Body Fat measured using dual energy x-ray absorptiometry (DXA).

Time frame:
Assessed at the end of each treatment block: week 8 & week 20.
Reported as:
Median · percent body fat
Within Participant Difference After Oxytocin vs Placebo in % Body Fat
percent body fatOxytocinPlacebo
End of Intervention50.1 (44.1 to 54.3)50.6 (45.9 to 55.0)
Change at 8 WeeksNA (NA to NA)NA (NA to NA)
Statistical analysis
  • Oxytocin vs Placebo · Regression, Linear · p = 0.49 · Regression coefficient: -0.02
Other pre-specifiedSkeletal Muscle Oxidative Phosphorylation (OXPHOS) Capacity

Measured using MRI-based post-exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) decline exponential time constant.

Time frame:
Assessed at the end of each treatment block.

No measurements were reported for this outcome.

Other pre-specifiedWithin 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.

Time frame:
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
Reported as:
Median · score on a scale
Within Participant Change in Hyperphagia (Total Score) Attributable to Oxytocin vs Placebo
score on a scaleOxytocinPlacebo
Baseline20 (15.3 to 27)20.5 (16.5 to 23.8)
Change at 8 Weeks2 (-0.8 to 2.8)0 (-3.3 to 0.8)
Statistical analysis
  • Oxytocin · Regression, Linear · p = 0.68 · Regression coefficient: -0.7
Other pre-specifiedWithin 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.

Time frame:
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
Reported as:
Median · score on a scale
Within Participant Change in Disinhibition of Eating Attributable to Oxytocin vs Placebo
score on a scaleOxytocinPlacebo
Baseline8.5 (4.5 to 10.8)4 (3.3 to 10.5)
Change at 8 Weeks0 (-1.8 to 0.8)0 (-0.8 to 1)
Statistical analysis
  • Oxytocin · Regression, Linear · p = 0.52 · Regression coefficient: -0.6
Other pre-specifiedWithin 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.

Time frame:
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)
Reported as:
Median · T-Score
Within Participant Change in Mental and Emotional Health Related Quality of Life Attributable to Oxytocin vs Placebo
T-ScoreOxytocinPlacebo
Baseline: Anxiety49.9 (47.7 to 52.8)45.7 (37.5 to 55.6)
Change at 8 Weeks: Anxiety-2 (-4.6 to 0.7)0 (0 to 0)
Baseline: Depression42 (36.4 to 49.7)36.4 (36.4 to 49.9)
Change at 8 Weeks: Depression0 (0 to 3.1)0 (0 to 1.2)
Baseline: Cognition50.4 (48.6 to 56.6)44 (39.8 to 51.7)
Change at 8 Weeks: Cognition0 (-4.1 to 0)1.2 (0 to 2)
Baseline: Social Function (Adults) or Peer Interaction (Children)47.9 (45.1 to 51.4)47.9 (38.6 to 60.2)
Change at 8 Weeks: Social Function-1.6 (-4.9 to 0)-1.8 (-9.6 to 0)
Statistical analysis
  • Oxytocin · Regression, Linear · p = 0.91 · Regression coefficient: -0.2
  • Oxytocin · Regression, Linear · p = 0.91 · Regression coefficient: -0.2
  • Oxytocin · Regression, Linear · p = 0.04 · Regression coefficient: 4.7
  • Oxytocin · Regression, Linear · p = 0.36 · Regression coefficient: 2.3
Other pre-specifiedWithin 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.

Time frame:
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20).
Reported as:
Median · score on a scale
Within Participant Change in Family Assessment Device-General Function Scale (FAD-GFS) Attributable to Oxytocin vs Placebo
score on a scaleOxytocinPlacebo
Baseline1.6 (1 to 2.3)1.4 (1.1 to 1.8)
Change at 8 Weeks-0.1 (-0.5 to 0.1)0 (0 to 0.2)
Statistical analysis
  • Oxytocin · Regression, Linear · p = 0.64 · Regression coefficient: 0.05
Other pre-specifiedWithin 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).

Time frame:
Assessed using data from Intervention 1: Screening and Visit 4 (Week 8) and Intervention 2: Visit 5 (Week 12) and Visit 8 (Week 20)
Reported as:
Median · hours per week of physical activity
Within Participant Change in Voluntary Physical Activity Attributable to Oxytocin vs Placebo
hours per week of physical activityOxytocinPlacebo
Baseline1.3 (1.2 to 2)1.1 (0.3 to 1.5)
Change at 8 Weeks-0.4 (-1.6 to -0.08)-0.1 (-0.6 to 0.3)
Statistical analysis
  • Oxytocin · Regression, Linear · p = 0.83 · Regression coefficient: 0.05
Other pre-specifiedEye-Tracking Task

Amount of time spent viewing socially relevant versus socially irrelevant stimuli during eye-tracking.

Time frame:
Assessed at the end of each treatment block.

No measurements were reported for this outcome.

Other pre-specifiedEEG Task

N170 EEG signal during eye-tracking

Time frame:
Assessed at the end of each treatment block.

No measurements were reported for this outcome.

Adverse events

Collected over Adverse Event data were collected from the time of informed consent through the duration of participation (Up to 20 Weeks).. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Syntocinon (Intranasal Oxytocin)0/12 (0%)0/12 (0%)12/12 (100%)
Placebo0/12 (0%)0/12 (0%)12/12 (100%)
Overall0/12 (0%)0/12 (0%)12/12 (100%)
Most frequent other events
Showing 10 of 18
Most frequent other events
EventSyntocinon (Intranasal Oxytocin)PlaceboOverall
Electrocardiogram QT Corrected Interval ProlongedCardiac disorders2/123/125/12
Sore ThroatRespiratory, thoracic and mediastinal disorders2/122/124/12
DiarrheaGastrointestinal disorders0/123/123/12
Investigations - Other (Low Free T4)Investigations1/122/123/12
FatigueGeneral disorders2/120/122/12
IrritabilityPsychiatric disorders1/121/122/12
ConstipationGastrointestinal disorders2/120/122/12
Allergic RhinitisRespiratory, thoracic and mediastinal disorders2/120/122/12
InsomniaPsychiatric disorders1/121/122/12
Eye Disorders - Other (Possible Disc Blurring, Eyelid Twitching)Eye disorders2/120/122/12

Baseline characteristics

Age, Continuous
Age, Continuous(Years)Syntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)Total
Median17.0 (11.9 to 21.4)15.3 (14.3 to 16.0)15.3 (13.3 to 20.6)
Sex: Female, Male
Sex: Female, Male(Participants)Syntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)Total
Female246
Male437
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Syntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)Total
Hispanic or Latino022
Not Hispanic or Latino6511
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Syntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)Total
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American000
White5611
More than one race112
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)Syntocinon (= Oxytocin), Then PlaceboPlacebo, Then Syntocinon (= Oxytocin)Total
United States6713
08

Study locations

1 site
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
09

References and documents

Study documents

  • Study protocol · Aug 20, 2019
  • Statistical analysis plan · Jun 30, 2021
  • Informed consent form · Oct 21, 2020

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 5, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02849743
Lead sponsor
Shana McCormack, MD
Responsible party
Shana McCormack, MD (Attending Physician, Children's Hospital of Philadelphia) — Sponsor-investigator
First posted
Jul 29, 2016
Start date
Oct 2016
Primary completion
Apr 2021
Completion
May 2022
Results posted
Oct 5, 2022
Last update
Oct 5, 2022

Study contacts

Shana E McCormack, MD
principal investigator · Children's Hospital of Philadelphia

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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