CraniopharyngiomaPhase 2NCT02849743

Intranasal Oxytocin in Hypothalamic Obesity

A Phase 2 study testing Syntocinon in people with Craniopharyngioma and Hypothalamic Obesity, run by Shana McCormack, MD at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 10 to 35 years
Conditions the study is for: Craniopharyngioma, Hypothalamic Obesity
What is being tested?: Syntocinon
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 18 participants
Where: 1 siteUnited States
Study dates: Oct 2016to May 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 10 to 35 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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*

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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.

Who cannot join

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.
Diabetes mellitus requiring insulin or insulin secretagogue. Laboratory values: HgbA1c ≥8%
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

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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.

Trial locations and contacts

1 site in 1 country.

United States1 site

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: Intranasal Oxytocin to Promote Weight Loss in Children, Adolescents, and Adults With Brain Tumors and Hypothalamic Obesity Syndrome

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.

Conditions: Craniopharyngioma, Hypothalamic Obesity
Being tested: Syntocinon, Placebo (for Syntocinon)
Keywords: oxytocin, hypothalamic obesity, craniopharyngioma, metabolism
Medical subject headings: Craniopharyngioma, Sexual Infantilism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SyntocinonDrug
The active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.
Placebo (for Syntocinon)Drug
The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.

Study groups in NCT02849743
Study group	Role	What they receive
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 Receives: 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) Receives: Drug: Syntocinon; Drug: Placebo (for Syntocinon)

What is being measured

The primary outcome is the main question the study is designed to answer.

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).
Measured: 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.
Measured: 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.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Oct 2016May 2022

First posted29 Jul 2016 (estimated)
Study startOct 2016
Primary completionApr 2021
Study completionMay 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for CraniopharyngiomaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02849743
Sponsor's protocol ID: 16-012730
Status last verified: Sep 2022
First submitted: 22 Jul 2016
Last updated: 5 Oct 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Craniopharyngioma trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Intranasal Oxytocin to Promote Weight Loss in Children, Adolescents, and Adults With Brain Tumors and Hypothalamic Obesity Syndrome
ClinicalTrials.gov ID: NCT02849743
Acronym / sponsor ID: 16-012730
Conditions: Craniopharyngioma, Hypothalamic Obesity
Keywords: oxytocin, hypothalamic obesity, craniopharyngioma, metabolism
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: Shana McCormack, MDUniversity, hospital or other
Study start: Oct 2016
Primary completion: Apr 2021 (actual)
Study completion: May 2022 (actual)
First posted: 29 Jul 2016 (estimated)
Results: Posted 5 Oct 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
Syntocinon (= Oxytocin), then Placebo	Experimental	Drug: Syntocinon, Drug: Placebo (for Syntocinon)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
Placebo, then Syntocinon (= Oxytocin)	Experimental	Drug: Syntocinon, Drug: Placebo (for Syntocinon)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)

Who may qualify

Ages: 10 to 35 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (14 listed)

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:

Key exclusion criteria (21 listed)

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.
Diabetes mellitus requiring insulin or insulin secretagogue. Laboratory values: HgbA1c ≥8%
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:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
SyntocinonThe active substance of Syntocinon is a synthetic nonapeptide identical to the posterior pituitary hormone oxytocin.	Drug	Placebo, then Syntocinon (= Oxytocin), Syntocinon (= Oxytocin), then Placebo
Placebo (for Syntocinon)The placebo is identical to the Syntocinon formulation with the exception of the active compound, i.e., without oxytocin.	Drug	Placebo, then Syntocinon (= Oxytocin), Syntocinon (= Oxytocin), then Placebo

Primary outcome

Weight Loss
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)
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).

1 secondary outcome

Peripheral Oxytocin Area Under the Curve (AUC)
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.

Results and safety

Results posted to the registry on 5 Oct 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Syntocinon (= Oxytocin), Then Placebo	6	6
Placebo, Then Syntocinon (= Oxytocin)	7	6

Primary outcome: Weight Loss

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). Median (Inter-Quartile Range). Unit: kg

Measure	Oxytocin n = 10	Placebo n = 10
Baseline	88.3 (74.7 to 108.6)	91.9 (77.9 to 114.6)
Change at 8 Weeks	1.2 (0.9 to 1.8)	0.9 (-0.2 to 1.6)

Oxytocin vs Placebo: Least Means Square: -0.5, p = 0.92, Regression, Linear

Population analysed: 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.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Adverse Event data were collected from the time of informed consent through the duration of participation (Up to 20 Weeks).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Syntocinon (Intranasal Oxytocin)	0 of 12 (0%)	12 of 12 (100%)	0 of 12
Placebo	0 of 12 (0%)	12 of 12 (100%)	0 of 12
Overall	0 of 12 (0%)	12 of 12 (100%)	0 of 12

Limitations and caveats (from the sponsor): 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).

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Shana E McCormack, MD principal investigator

Children's Hospital of Philadelphia

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Syntocinon in people with Craniopharyngioma and Hypothalamic Obesity, run by Shana McCormack, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Oct 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Weight Loss (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)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02849743.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2022.