Phase 3 N=42 Randomized Quadruple-blind Treatment

Energy Balance & Weight Loss in Craniopharyngioma-related or Other Hypothalamic Tumors in Hypothalamic Obesity

Hypothalamic Obesity

Bottom Line

View on ClinicalTrials.gov: NCT02664441 ↗

Enrolled (actual)

Serious AEs

9.2%

Results posted

May 2022

Primary outcome: Primary: Percent Change of Body Mass Index (BMI) as Calculated by the Formula: Body Weight in kg Divided by Height in Meters². — 1.7; 3.5 percent change from baseline — p=0.40

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Exenatide (Drug); placebo (Drug)
Age: Pediatric, Adult · 10+ yrs
Sex: All
Sponsor: Seattle Children's Hospital
Primary completion: Mar 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Percent Change of Body Mass Index (BMI) as Calculated by the Formula: Body Weight in kg Divided by Height in Meters².	1.7; 3.5	0.40
SECONDARY Changes in Body Composition as Assessed by Body Fat Mass Using Dual Energy X-ray Absorptiometry (DEXA)	1.5; 4.6	0.02 sig
SECONDARY Changes in Fat and Total Calorie Intake Assessed by Free Buffet Meal Analysis.	-9.5; 14.5; -287; 224	.032 sig
SECONDARY Changes in Fasting Glucose	7.4; 3.7	0.19
SECONDARY Changes in HDL Cholesterol and Triglycerides Assessed by Fasting Lipids	0.7; 0.6; -0.6; 4.2	0.82
SECONDARY Changes in Inflammation Assessed by C-reactive Protein (CRP)	0.00; 0.18	0.03 sig
SECONDARY Changes of Insulin Resistance Assessed by Homeostasis Model Assessment of Insulin Resistance (HOMA-IR)	3.5; 2.2	0.32
SECONDARY Changes of Circulating Leptin Levels	1.4; 11.8	0.69
SECONDARY Changes of Energy Expenditure Assessed by Doubly Labeled Water Analysis	-17.8; 146.3	0.004 sig
SECONDARY Changes of Energy Intake Assessed by Automated Self-Administered 24-Hour Dietary Recall (ASA24-Kids)	-62; -210	0.58
SECONDARY Changes in Glucose 120 Minutes Following an Oral Glucose Tolerance Test	-12.4; -3.4	0.088

Summary

The proposed multicenter study will test the effect of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (ExQW, Bydureon®) on clinical outcomes and metabolic parameters in a double-blind, placebo-controlled 36 week randomized trial with an 18 week open label extension. Following baseline testing, 48 patients will be randomly assigned with equal allocation to ExQW or matching placebo injection for 36 weeks, followed by an 18 week open label extension during which all patients receive ExQW. Changes of weight status, body composition, free-living total daily energy expenditure (EE) by doubly labeled water (DLW), activity by acetimetry, energy intake (questionnaires and food diary), as well as glucose tolerance and hormonal parameters of energy homeostasis and insulin resistance will be assessed before treatment and at the end of the placebo-controlled phase (week 36). Activity, metabolic outcomes, energy intake will be also assessed at study week 18 (mid treatment of randomized study), as well as week 54 (end of open label treatment).

Eligibility Criteria

Inclusion Criteria

Age 10-25 years at time of enrollment
Diagnosis of hypothalamic obesity with age- and sex adjusted BMI ≥ 95% or BMI ≥30 kg/m² if over 18 y
History of craniopharyngioma or another tumor located in the hypothalamic area
Hypothalamic lesion documented by neuroradiology
≥ 6 months post-surgical or radiation treatment
Weight stable or increasing over 3 months prior to screening visit
Stable hormone replacement for at least 3 months prior to screening visit

Exclusion Criteria

Renal impairment (GFR 50 mg/L at screening
Initiation of weight loss medications within 3 months of screening visit
Previous donation of blood >10% of estimated blood volume within 3 months prior study
Current warfarin use
Current use of any other GLP1 receptor agonist
Untreated thyroid disorder or adrenal insufficiency
History of bariatric surgery or planned bariatric surgery until end of study
Pregnancy, lactation or expectation to conceive during study period
Subject unlikely to adhere to study procedures in opinion of investigator
Subject with contraindication to neuroimaging by MRI

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02664441). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.