Clinical Trial: Intranasal Oxytocin in Hypothalamic Obesity

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief Summary: This research study will test if oxytocin, delivered by nasal spray, will promote weight loss in children and adolescents 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 Summary:
Sponsor: Shana McCormack, MD

Current Primary Outcome: Weight loss [ Time Frame: 8 weeks ]

The primary objective of this study is to determine whether treatment with 8 weeks of inhaled 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 21 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).


Original Primary Outcome: Weight loss [ Time Frame: 8 weeks ]

The primary objective of this study is to determine whether treatment with 8 weeks of inhaled 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 21 years. "Weight loss" is defined as the difference between the change in weight during the oxytocin block and the change in weight during the placebo block.


Current Secondary Outcome:

  • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Assessed during each treatment block: at weeks 0 & 12, 0.3 & 12.3, 2 & 14, 2.3 & 14.3, 6 & 18, 8 & 20 and any unscheduled visits ]
    All subjects receiving at least one dose of OXT and/or placebo will be included in the safety analysis. The frequencies of AEs by type, body system, severity and relationship to study drug will be summarized. SAEs (if any) will be described in detail. In particular, the proportion of study subjects who were able to achieve the full dosing amount and the proportion of those receiving synthetic vasopressin requiring modification to their usual synthetic vasopressin regimen will also be noted. The incidence of hyponatremia will be reported. Participants will be screened for adverse events using a safety monitoring uniform report form (SMURF).
  • Peripheral oxytocin area under the curve (AUC) [ Time Frame: Assessed during each treatment block: at either initial dose at baseline (week 0 & week 12) or increased dose at 2 weeks (week 2 & week 15); 50% of participants at each set ]
    We will determine the immediate peripheral pharmacokinetics of intranasal oxytocin (versus placebo/endogenous oxytocin).


Original Secondary Outcome: Same as current

Information By: Children's Hospital of Philadelphia

Dates:
Date Received: July 22, 2016
Date Started: October 2016
Date Completion: July 2020
Last Updated: November 30, 2016
Last Verified: November 2016