Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome (NCT03197662) | Clinical Trial Compass
CompletedPhase 2
Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome
United States32 participantsStarted 2018-04-11
Plain-language summary
This study is a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, Glucagon-Like Peptide-1 (GLP-1), insulin, glucagon, testosterone, and estrogen). If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS.
Funding Source- FDA OOPD
Who can participate
Age range
5 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female pediatric outpatients aged 5 to 17 years
. Must be in PWS nutritional phase 2b or 3 as determined by PI
. Must be on growth hormone treatment and have been receiving stable doses of growth hormone treatment for at least 3 months prior to screening date. Treatment cannot have been interrupted for more than one week within 3 months of screening.
. Diagnosis of PWS confirmed by patient medical records.
. A score of at least moderate severity on the Hyperphagia Questionnaire for Clinical Trials at both screening and baseline visits.
. Stable dosages of hormone treatments (including testosterone and estrogen supplements) for 4 weeks prior to randomization and for the duration of the study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT)
Timeframe: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)
. Stable dosages of metabolic treatments that could affect appetite (including metformin) for 4 weeks prior to randomization and for the duration of the study.
. Physical exam and laboratory results that are within the normal range for individuals with PWS.
Exclusion criteria
. Exposure to any investigational agent in the 30 days prior to randomization.
. Child not receiving growth hormone treatment
. Children weighing less than 40 lbs
. Children with unstable Type 2 Diabetes confirmed by Hemoglobin A1C levels at screening
. Children with unstable medical co-morbidities at baseline.
. Children with active upper respiratory infections at screening.
. A primary psychiatric diagnosis other than Autism Spectrum Disorder (ASD), including bipolar disorder, psychosis, schizophrenia, Post-traumatic stress disorder (PTSD) or major depressive disorder (MDD). These patients will be excluded due to potential confounding results.
. Pregnant or lactating patients or patients who will not agree to use a double barrier method of contraception. IN-OXT has not been studied in pregnant or lactating women.