Trial Outcomes & Findings for Effects of Exenatide on Hypothalamic Obesity (NCT NCT01061775)

NCT ID: NCT01061775

Last Updated: 2019-10-08

Results Overview

BMI was collected at baseline and 24 weeks

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 19 participants
• Primary outcome timeframe: 24 weeks
• Results posted on: 2019-10-08

Participant Flow

Participant milestones

Measure	Exenatide Participants received 5mcg of exenatide twice a day for 4 weeks and increased to 10 mcg twice a day for 20 weeks.
Overall Study STARTED	19
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Exenatide Participants received 5mcg of exenatide twice a day for 4 weeks and increased to 10 mcg twice a day for 20 weeks.
Overall Study Physician Decision	2
Overall Study Lost to Follow-up	1

Baseline Characteristics

Effects of Exenatide on Hypothalamic Obesity

Baseline characteristics by cohort

Measure	Exenatide n=19 Participants Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	15 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Region of Enrollment United States	19 participants n=5 Participants
Tumor Type Craniopharyngioma	14 participants n=5 Participants
Tumor Type Optic chiasm-hypothalamic glioma	2 participants n=5 Participants
Tumor Type Suprarasellar germinoma	2 participants n=5 Participants
Tumor Type Thalamic astrocytoma	1 participants n=5 Participants
BMI	35 kg/m^2 n=5 Participants
Height	160.8 cm STANDARD_DEVIATION 14.3 • n=5 Participants
Weight	89 kg STANDARD_DEVIATION 28.2 • n=5 Participants
Waist Circumference	111.6 cm STANDARD_DEVIATION 19.5 • n=5 Participants
Hip Circumference	113.5 cm STANDARD_DEVIATION 16 • n=5 Participants
Waist to Height Ratio	0.98 cm STANDARD_DEVIATION 0.08 • n=5 Participants
Child Eating Behaviour Questionnaire	11.47 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Population: The effects of exenatide on BMI were analyzed using a paired t-test comparing BMI at baseline with BMI after six months of treatment with each patient serving as his or her own control.

BMI was collected at baseline and 24 weeks

Outcome measures

Measure	Exenatide n=16 Participants Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
BMI Change	-0.77 kg/m^2 Interval -1.97 to 0.43

PRIMARY outcome

Timeframe: 24 weeks

Population: All analyses were performed using SPSS (version 20.0.0, SPSS Inc, Chicago, IL).

Waist circumference was measured at the natural waist level (midway between the lowest rib margin and the iliac crest) at baseline and 24 weeks

Outcome measures

Measure	Exenatide n=16 Participants Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Waist to Height Ratio (WHtR)	-2.06 percentage Standard Deviation 0.08

SECONDARY outcome

Timeframe: 24 weeks

Population: Paired t-tests were performed to compare the satiety survey (continuous). Three subjects discontinued prior to completing the Week 24 CEBQ.

The Child Eating Behaviour Questionnaire (CEBQ) was designed as parent-report measure comprised of 35 items, each rated on a five-point likert scale that ranges from never to always. We utilized the CEBQ as a self-report measure during this study; it has not been validated for such use. For the purposes of this study, we looked at the Satiety Responsiveness Subscale Scores (5 questions; total scores could range from 5-25 with lower scores denoting a lower level of satiety). The results reported show the change between baseline and week 24 scores.

Outcome measures

Measure	Exenatide n=16 Participants Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Childhood Eating Behavior Questionnaire (CEBQ)	14 units on a scale Interval 12.2167 to 15.7833

SECONDARY outcome

Timeframe: 24 weeks

Population: The sample size was small due to missing data

Dietary data were collected via 3-day diet records (Crawford et al. 1994) twice during the study, at baseline and week 24. Three-day diet records were collected on consecutive days including one weekend day. A registered dietitian (RD) instructed subjects on dietary data collection at baseline appointment. Depending on the age and capacity of the subject, the patient, parent or a collaboration of both recorded dietary intake. A RD entered dietary data into Nutritionist Pro software (First DataBank, SanBruno, CA) and the mean difference was analyzed.

Outcome measures

Measure	Exenatide n=10 Participants Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Calorie Intake Based on 3-day Diet Records	406.700 kcals Interval -117.088 to 930.488

Adverse Events

Exenatide

Serious events: 2 serious events

Other events: 17 other events

Deaths: 0 deaths

Serious adverse events

Measure	Exenatide n=19 participants at risk Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Endocrine disorders cholecystitis	10.5% 2/19 • Number of events 2

Other adverse events

Measure	Exenatide n=19 participants at risk Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Gastrointestinal disorders Nausea and stomachache	73.7% 14/19 • Number of events 20
Gastrointestinal disorders vomiting	52.6% 10/19 • Number of events 20
Gastrointestinal disorders Diarrhoea	15.8% 3/19 • Number of events 6
Gastrointestinal disorders Abdominal Pain	21.1% 4/19 • Number of events 8
Gastrointestinal disorders Blood in Stool	5.3% 1/19 • Number of events 2
Gastrointestinal disorders Constipation	5.3% 1/19 • Number of events 1
Gastrointestinal disorders Reflux Gastritis	5.3% 1/19 • Number of events 1
Gastrointestinal disorders Absomen Sore to Touch	5.3% 1/19 • Number of events 1
General disorders Pyrexia	5.3% 1/19 • Number of events 2
General disorders Fatigue	10.5% 2/19 • Number of events 2
General disorders Injection Site Pain	5.3% 1/19 • Number of events 1
General disorders Body Aches	10.5% 2/19 • Number of events 2
Immune system disorders Seasonal Allergies	5.3% 1/19 • Number of events 1
Infections and infestations Nasopharyngitis	15.8% 3/19 • Number of events 3
Immune system disorders Ear Infection	10.5% 2/19 • Number of events 4
Immune system disorders Pharyngitis	10.5% 2/19 • Number of events 2
Immune system disorders Sinusitis	5.3% 1/19 • Number of events 1
Infections and infestations Upper Respiratory Infection	5.3% 1/19 • Number of events 1
Investigations Blood in Urine Present	5.3% 1/19 • Number of events 1
Metabolism and nutrition disorders Decreased Appetite	10.5% 2/19 • Number of events 2
Musculoskeletal and connective tissue disorders Back Pain	15.8% 3/19 • Number of events 3
Nervous system disorders Headache	15.8% 3/19 • Number of events 3
Nervous system disorders Dizziness	5.3% 1/19 • Number of events 1
Respiratory, thoracic and mediastinal disorders Cough	5.3% 1/19 • Number of events 1
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.3% 1/19 • Number of events 1
Respiratory, thoracic and mediastinal disorders Tonsillar Hypertrophy	5.3% 1/19 • Number of events 1
Skin and subcutaneous tissue disorders Alopecia	5.3% 1/19 • Number of events 1
Skin and subcutaneous tissue disorders Rash	5.3% 1/19 • Number of events 1

Additional Information

Dr. Jennifer Abuzzahab

Children's Hospitals and Clinics of Minnesota

Phone: 651-220-6867

Email: jennifer.abuzzahab@childrensmn.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place