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Effects of Exenatide on Hypothalamic Obesity

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ClinicalTrials.gov Identifier: NCT01061775
Recruitment Status : Completed
First Posted : February 3, 2010
Results First Posted : March 21, 2016
Last Update Posted : October 8, 2019
Sponsor:
Collaborator:
Amylin Pharmaceuticals, LLC.
Information provided by (Responsible Party):
Children's Hospitals and Clinics of Minnesota

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hypothalamic Obesity
Intervention Drug: Exenatide
Enrollment 19
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Exenatide
Hide Arm/Group Description Participants received 5mcg of exenatide twice a day for 4 weeks and increased to 10 mcg twice a day for 20 weeks.
Period Title: Overall Study
Started 19
Completed 16
Not Completed 3
Reason Not Completed
Physician Decision             2
Lost to Follow-up             1
Arm/Group Title Exenatide
Hide Arm/Group Description Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Overall Number of Baseline Participants 19
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
<=18 years
16
  84.2%
Between 18 and 65 years
3
  15.8%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 19 participants
15
(10 to 21)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
Female
8
  42.1%
Male
11
  57.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 19 participants
19
Tumor Type  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants
Craniopharyngioma 14
Optic chiasm-hypothalamic glioma 2
Suprarasellar germinoma 2
Thalamic astrocytoma 1
BMI  
Mean (Full Range)
Unit of measure:  Kg/m^2
Number Analyzed 19 participants
35
(26.9 to 45.8)
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 19 participants
160.8  (14.3)
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 19 participants
89  (28.2)
Waist Circumference  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 19 participants
111.6  (19.5)
Hip Circumference  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 19 participants
113.5  (16)
Waist to Height Ratio  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 19 participants
0.98  (0.08)
Child Eating Behaviour Questionnaire   [1] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 19 participants
11.47
(5.0 to 16.0)
[1]
Measure Description: 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).
1.Primary Outcome
Title BMI Change
Hide Description BMI was collected at baseline and 24 weeks
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Exenatide
Hide Arm/Group Description:
Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Overall Number of Participants Analyzed 16
Mean (95% Confidence Interval)
Unit of Measure: kg/m^2
-0.77
(-1.97 to 0.43)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Exenatide
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
2.Primary Outcome
Title Waist to Height Ratio (WHtR)
Hide Description Waist circumference was measured at the natural waist level (midway between the lowest rib margin and the iliac crest) at baseline and 24 weeks
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All analyses were performed using SPSS (version 20.0.0, SPSS Inc, Chicago, IL).
Arm/Group Title Exenatide
Hide Arm/Group Description:
Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Overall Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: percentage
-2.06  (0.08)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Exenatide
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
3.Secondary Outcome
Title Childhood Eating Behavior Questionnaire (CEBQ)
Hide Description 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.
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Paired t-tests were performed to compare the satiety survey (continuous). Three subjects discontinued prior to completing the Week 24 CEBQ.
Arm/Group Title Exenatide
Hide Arm/Group Description:
Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
Overall Number of Participants Analyzed 16
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
14
(12.2167 to 15.7833)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Exenatide
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
4.Secondary Outcome
Title Calorie Intake Based on 3-day Diet Records
Hide Description 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.
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The sample size was small due to missing data
Arm/Group Title Exenatide
Hide Arm/Group Description:

Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.

Of 55 potential participants screened, 19 agreed to participate. Reasons for decline included reluctance to add another medication to their current regimens, travel distance, and aversion to an injectable medication. Sixteen patients completed the study. Of the three who failed to complete the study, one was lost to follow up, and two dropped out due to difficulty adhering to the twice daily injections, but none experienced significant side effects from therapy. Three of the 19 patients were on chronic metformin therapy.

Overall Number of Participants Analyzed 10
Mean (95% Confidence Interval)
Unit of Measure: kcals
406.700
(-117.088 to 930.488)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Exenatide
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Exenatide
Hide Arm/Group Description Exenatide: 5mcg twice a day for 4 weeks increased to 10 mcg twice a day for 20 weeks.
All-Cause Mortality
Exenatide
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Exenatide
Affected / at Risk (%) # Events
Total   2/19 (10.53%)    
Endocrine disorders   
cholecystitis  [1]  2/19 (10.53%)  2
Indicates events were collected by systematic assessment
[1]
2 developed cholecystitis requiring cholecystectomy. Both super-responders, with 1 losing 18 lbs in six months and the other remaining on medication for a full year losing nearly 40 lbs. Both developed symptoms well after last dose of study drug.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Exenatide
Affected / at Risk (%) # Events
Total   17/19 (89.47%)    
Gastrointestinal disorders   
Nausea and stomachache *  14/19 (73.68%)  20
vomiting *  10/19 (52.63%)  20
Diarrhoea *  3/19 (15.79%)  6
Abdominal Pain *  4/19 (21.05%)  8
Blood in Stool *  1/19 (5.26%)  2
Constipation *  1/19 (5.26%)  1
Reflux Gastritis *  1/19 (5.26%)  1
Absomen Sore to Touch *  1/19 (5.26%)  1
General disorders   
Pyrexia *  1/19 (5.26%)  2
Fatigue *  2/19 (10.53%)  2
Injection Site Pain *  1/19 (5.26%)  1
Body Aches *  2/19 (10.53%)  2
Immune system disorders   
Seasonal Allergies *  1/19 (5.26%)  1
Ear Infection *  2/19 (10.53%)  4
Pharyngitis *  2/19 (10.53%)  2
Sinusitis *  1/19 (5.26%)  1
Infections and infestations   
Nasopharyngitis *  3/19 (15.79%)  3
Upper Respiratory Infection *  1/19 (5.26%)  1
Investigations   
Blood in Urine Present *  1/19 (5.26%)  1
Metabolism and nutrition disorders   
Decreased Appetite *  2/19 (10.53%)  2
Musculoskeletal and connective tissue disorders   
Back Pain *  3/19 (15.79%)  3
Nervous system disorders   
Headache *  3/19 (15.79%)  3
Dizziness *  1/19 (5.26%)  1
Respiratory, thoracic and mediastinal disorders   
Cough *  1/19 (5.26%)  1
Rhinorrhoea *  1/19 (5.26%)  1
Tonsillar Hypertrophy *  1/19 (5.26%)  1
Skin and subcutaneous tissue disorders   
Alopecia *  1/19 (5.26%)  1
Rash *  1/19 (5.26%)  1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Jennifer Abuzzahab
Organization: Children's Hospitals and Clinics of Minnesota
Phone: 651-220-6867
EMail: jennifer.abuzzahab@childrensmn.org
Layout table for additonal information
Responsible Party: Children's Hospitals and Clinics of Minnesota
ClinicalTrials.gov Identifier: NCT01061775     History of Changes
Other Study ID Numbers: 0903-028
First Submitted: February 2, 2010
First Posted: February 3, 2010
Results First Submitted: February 25, 2015
Results First Posted: March 21, 2016
Last Update Posted: October 8, 2019