Trial Outcomes & Findings for Energy Balance & Weight Loss in Craniopharyngioma-related or Other Hypothalamic Tumors in Hypothalamic Obesity (NCT NCT02664441)

Last Updated: 2022-05-05

Results Overview

Percent change of body mass index (BMI), as calculated by the formula: body weight in kg divided by height in meters², between baseline and the end of the 36-week randomized drug treatment phase.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

42 participants

Primary outcome timeframe

From baseline to 36 weeks

Results posted on

2022-05-05

Participant Flow

Patients aged 10-25 years of age with a diagnosis of hypothalamic obesity following treatment for craniopharyngioma were recruited from pediatric endocrinology outpatient clinics at the three research sites (Seattle Children's Hospital, Seattle, WA, USA; Children's Minnesota, St. Paul, MN, USA; and Vanderbilt University Medical Center, Nashville, TN, USA) and hypothalamic obesity internet support groups.

Qualified participants started with a 2-week placebo run-in to test adherence to the protocol prior to randomization.

Participant milestones

Participant milestones
Measure	Exenatide Once Weekly Extended-release Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Randomization STARTED	23	19
Randomization COMPLETED	23	18
Randomization NOT COMPLETED	0	1
36-week Randomized Trial STARTED	23	18
36-week Randomized Trial COMPLETED	20	15
36-week Randomized Trial NOT COMPLETED	3	3
18-week Open Label Extension STARTED	20	15
18-week Open Label Extension COMPLETED	17	14
18-week Open Label Extension NOT COMPLETED	3	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Exenatide Once Weekly Extended-release Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Randomization Withdrawal by Subject	0	1
36-week Randomized Trial Adverse Event	2	0
36-week Randomized Trial Lost to Follow-up	1	0
36-week Randomized Trial Withdrawal by Subject	0	3
18-week Open Label Extension Adverse Event	1	0
18-week Open Label Extension Physician Decision	1	0
18-week Open Label Extension Death	1	0
18-week Open Label Extension Lost to Follow-up	0	1

Baseline Characteristics

One participant withdrawn in placebo group

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=19 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections	Total n=42 Participants Total of all reporting groups
Age, Categorical <=18 years	16 Participants n=5 Participants • One participant withdrawn in placebo group	14 Participants n=7 Participants • One participant withdrawn in placebo group	30 Participants n=5 Participants • One participant withdrawn in placebo group
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants • One participant withdrawn in placebo group	5 Participants n=7 Participants • One participant withdrawn in placebo group	12 Participants n=5 Participants • One participant withdrawn in placebo group
Age, Categorical >=65 years	0 Participants n=5 Participants • One participant withdrawn in placebo group	0 Participants n=7 Participants • One participant withdrawn in placebo group	0 Participants n=5 Participants • One participant withdrawn in placebo group
Age, Continuous	16.9 years STANDARD_DEVIATION 4.3 • n=5 Participants	16.9 years STANDARD_DEVIATION 4.7 • n=7 Participants	16.9 years STANDARD_DEVIATION 4.4 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	11 Participants n=7 Participants	25 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	8 Participants n=7 Participants	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	21 Participants n=5 Participants	19 Participants n=7 Participants	40 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	21 Participants n=5 Participants	16 Participants n=7 Participants	37 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	23 participants n=5 Participants	19 participants n=7 Participants	42 participants n=5 Participants
Body Mass Index	35.8 kg/m² STANDARD_DEVIATION 6.6 • n=5 Participants	38.7 kg/m² STANDARD_DEVIATION 7.5 • n=7 Participants	37.3 kg/m² STANDARD_DEVIATION 7.1 • n=5 Participants

PRIMARY outcome

Timeframe: From baseline to 36 weeks

Population: Analyses include all participants randomized to a study drug and received at least one drug treatment administration. Missing data was imputed using a last observation carry-forward approach.

Percent change of body mass index (BMI), as calculated by the formula: body weight in kg divided by height in meters², between baseline and the end of the 36-week randomized drug treatment phase.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=18 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Percent Change of Body Mass Index (BMI) as Calculated by the Formula: Body Weight in kg Divided by Height in Meters².	1.7 percent change from baseline Standard Error 0.8	3.5 percent change from baseline Standard Error 0.9

SECONDARY outcome

Timeframe: At baseline and 36 weeks

Population: Of the 41 enrolled participants, 20 out of 23 participants randomized to exenatide once weekly extended-release and 15 out of 18 participants randomized to placebo completed both pre- and post-treatment assessments and are included in this analysis.

Body composition change between baseline and the end of the 36-week randomized drug treatment phase assessed by dual energy x-ray absorptiometry (DEXA) and expressed as the change in adipose tissue mass.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=20 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=15 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes in Body Composition as Assessed by Body Fat Mass Using Dual Energy X-ray Absorptiometry (DEXA)	1.5 kilograms Standard Error 0.9	4.6 kilograms Standard Error 1.0

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Population: Of the 41 enrolled participants, 18 out of 23 participants randomized to exenatide once weekly extended-release and 15 out of 18 participants randomized to placebo completed both pre- and post-treatment assessments and are included in this analysis.

Changes in fat and total calorie intake during free buffet meals assessed at baseline and after 36-weeks of study drug treatment. The buffet meal is an objective measure of satiety as it assesses food intake and choice after a caloric preload. A standardized test meal preload provided 20% of estimated daily caloric requirements,based on the Schofield-HW equation. The purpose of the test meal is to ensure that study participants are in an equally fed state. Ninety minutes later, an ad libitum buffet meal was served consisting of a wide variety of food items and more than the child's estimated daily calorie requirements will be offered (5,000 kcal). Children had access to the buffet for 30 min, after which calorie intake and composition of consumed foods was measured by weighing back uneaten food.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=18 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=15 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes in Fat and Total Calorie Intake Assessed by Free Buffet Meal Analysis. Fat Intake	-9.5 grams Standard Error 6.3	14.5 grams Standard Error 7.7
Changes in Fat and Total Calorie Intake Assessed by Free Buffet Meal Analysis. Total Calorie Intake	-287 grams Standard Error 130	224 grams Standard Error 136

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Change in fasting blood glucose between baseline and the end of the 36-week randomized drug treatment phase.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=18 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes in Fasting Glucose	7.4 mg/dL Standard Error 6.0	3.7 mg/dL Standard Error 3.5

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Change in fasting HDL cholesterol and triglycerides between baseline and the end of the 36-week randomized drug treatment phase.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=18 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes in HDL Cholesterol and Triglycerides Assessed by Fasting Lipids HDL Cholesterol	0.7 mg/dL Standard Error 2.1	0.6 mg/dL Standard Error 2.8
Changes in HDL Cholesterol and Triglycerides Assessed by Fasting Lipids Triglycerides	-0.6 mg/dL Standard Error 8.1	4.2 mg/dL Standard Error 8.6

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Change in C-reactive protein (CRP) between baseline and the end of the 36-week randomized drug treatment phase.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=18 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes in Inflammation Assessed by C-reactive Protein (CRP)	0.00 mg/dL Standard Error 0.07	0.18 mg/dL Standard Error 0.27

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Changes of insulin resistance estimated by the homeostasis model assessment of insulin resistance (HOMA-IR) using the formula HOMA-IR = insulin \[mU/l\] x glucose \[mmol/l\]) / 22.5 where both insulin and glucose values are obtained from a fasting blood sample.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=17 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes of Insulin Resistance Assessed by Homeostasis Model Assessment of Insulin Resistance (HOMA-IR)	3.5 HOMA-IR score Standard Error 1.7	2.2 HOMA-IR score Standard Error 2.4

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Change in circulating leptin between baseline and the end of the 36-week randomized drug treatment phase.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=18 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes of Circulating Leptin Levels	1.4 ng/mL Standard Error 6.3	11.8 ng/mL Standard Error 9.4

SECONDARY outcome

Timeframe: Baseline and 36 weeks

Population: Of the 41 enrolled participants, 19 out of 23 participants randomized to exenatide once weekly extended-release and 14 out of 18 participants randomized to placebo completed both pre- and post-treatment assessments and are included in this analysis.

Total energy expenditure in the free-living environment was measured using doubly labeled water which estimates carbon dioxide production by measuring the elimination of the tracers deuterium (²H) and oxygen-18 (¹⁸O) from the body. These measures are used to determine the average daily rate of carbon dioxide production which is then used to calculate total energy expenditure using an equation from Weir and an assumed food quotient (0.85).

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=19 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=14 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes of Energy Expenditure Assessed by Doubly Labeled Water Analysis	-17.8 kcals/day Standard Error 38.0	146.3 kcals/day Standard Error 29.0

SECONDARY outcome

Timeframe: Baseline and 36 weeks

Population: Of the 41 enrolled participants, 14 out of 23 participants randomized to exenatide once weekly extended-release and 9 out of 18 participants randomized to placebo completed both pre- and post-treatment assessments and are included in this analysis.

Self-reported daily energy intake was assessed by Automated Self-Administered 24-Hour Dietary Recall (ASA24-Kids, http://appliedresearch.cancer.gov/tools/instruments/asa24/), a web-based diet assessment tool that allows 24-hour diet recall using branded food items.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=14 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=9 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes of Energy Intake Assessed by Automated Self-Administered 24-Hour Dietary Recall (ASA24-Kids)	-62 kcals Standard Error 155	-210 kcals Standard Error 336

SECONDARY outcome

Timeframe: From baseline to 36 weeks

Change in blood glucose measures 120 minutes post-glucose bolus during an oral glucose tolerance test between baseline and the end of the 36-week randomized drug treatment phase.

Outcome measures

Outcome measures
Measure	Exenatide Once Weekly Extended-release n=23 Participants Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention followed by 18 weeks open label exenatide once weekly extended-release. Exenatide: Weekly injections of active drug.	Matching Placebo n=18 Participants Weekly injections of placebo for 36 weeks followed by 18 weeks open label exenatide once weekly extended-release. placebo: Weekly placebo injections
Changes in Glucose 120 Minutes Following an Oral Glucose Tolerance Test	-12.4 mg/dL Standard Error 10.9	-3.4 mg/dL Standard Error 3.7

Adverse Events

Double-Blind Exenatide Once Weekly Extended-release

Serious events: 2 serious events

Other events: 21 other events

Deaths: 0 deaths

Double-Blind Placebo

Serious events: 1 serious events

Other events: 15 other events

Deaths: 0 deaths

Open Label Exenatide Once Weekly Extended-release

Serious events: 4 serious events

Other events: 17 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Double-Blind Exenatide Once Weekly Extended-release n=23 participants at risk Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention Exenatide: Weekly injections of active drug.	Double-Blind Placebo n=18 participants at risk Weekly injections of placebo for 36 weeks in randomized intervention placebo: Weekly placebo injections	Open Label Exenatide Once Weekly Extended-release n=35 participants at risk 18 weeks open label exenatide once weekly extended-release (Bydureon®) arm. Exenatide: Weekly injections of active drug.
Infections and infestations Cellulitis	4.3% 1/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Hepatobiliary disorders Cholecystitis / Cholecystectomy	0.00% 0/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Metabolism and nutrition disorders Hypernatremic dehydration	0.00% 0/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	5.6% 1/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Nervous system disorders Migraine Headache	0.00% 0/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Nervous system disorders Seizure	4.3% 1/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Nervous system disorders Altered mental status	0.00% 0/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)

Other adverse events

Other adverse events
Measure	Double-Blind Exenatide Once Weekly Extended-release n=23 participants at risk Injections of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (Bydureon®) for 36 weeks in randomized intervention Exenatide: Weekly injections of active drug.	Double-Blind Placebo n=18 participants at risk Weekly injections of placebo for 36 weeks in randomized intervention placebo: Weekly placebo injections	Open Label Exenatide Once Weekly Extended-release n=35 participants at risk 18 weeks open label exenatide once weekly extended-release (Bydureon®) arm. Exenatide: Weekly injections of active drug.
Gastrointestinal disorders Abdominal pain	39.1% 9/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	11.1% 2/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Gastrointestinal disorders Nausea	26.1% 6/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	16.7% 3/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	8.6% 3/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Gastrointestinal disorders Diarrhea	30.4% 7/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	16.7% 3/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	11.4% 4/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Gastrointestinal disorders Vomiting	17.4% 4/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	22.2% 4/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	14.3% 5/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Gastrointestinal disorders Constipation	13.0% 3/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	11.1% 2/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	5.7% 2/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Gastrointestinal disorders Cholelithiasis	0.00% 0/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	0.00% 0/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	2.9% 1/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Nervous system disorders Dizziness	8.7% 2/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	5.6% 1/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	5.7% 2/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
Nervous system disorders Headache	30.4% 7/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	33.3% 6/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	8.6% 3/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)
General disorders Injection site reaction	30.4% 7/23 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	22.2% 4/18 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)	5.7% 2/35 • Double-Blind Randomized Phase - From Study Visit 1 (0 weeks) through end of Study Visit 4 (36 weeks); Open Label Extension - From Study Visit 4 (36 Weeks) through end of Study Visit 5 (54 weeks)

Additional Information

Christian L. Roth, MD

Seattle Children's Hospital

Phone: 206-987-5428

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place