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Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Mitchell E. Geffner, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier:
NCT01444898
First received: September 27, 2011
Last updated: September 27, 2016
Last verified: September 2016
Results First Received: June 14, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Prader-Willi Syndrome
Intervention: Drug: Exenatide

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Exenatide All subjects enrolled in this study will be given Exenatide for 6 months. The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Participant Flow:   Overall Study
    Exenatide
STARTED   10 
COMPLETED   9 
NOT COMPLETED   1 
Withdrawal by Subject                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Exenatide

All subjects enrolled in this study will be given Exenatide for 6 months.

Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).


Baseline Measures
   Exenatide 
Overall Participants Analyzed 
[Units: Participants]
 10 
Age 
[Units: Participants]
 
<=18 years   6 
Between 18 and 65 years   4 
>=65 years   0 
Age 
[Units: Years]
Median (Full Range)
 16 
 (14.7 to 24.6) 
Gender 
[Units: Participants]
 
Female   6 
Male   4 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   1 
Not Hispanic or Latino   6 
Unknown or Not Reported   3 
Race (NIH/OMB) 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   0 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   1 
White   6 
More than one race   2 
Unknown or Not Reported   1 
Region of Enrollment [1] 
[Units: Participants]
 
United States   10 
[1] Region of Enrollment = California
Weight [1] 
[Units: Kg]
Mean (Standard Deviation)
 102.5  (18.6) 
[1] Weight (kg)
BMI [1] 
[Units: Kg m^-2]
Median (Inter-Quartile Range)
 41.7 
 (34.1 to 55) 
[1] BMI (kg m^-2)
BMI Z-Score 
[Units: Units on a scale]
Mean (Standard Deviation)
 3.5  (1.1) 
Glycosylated hemoglobin (HbA1c) [1] 
[Units: Percentage of total hemoglobin]
Median (Inter-Quartile Range)
 5.9 
 (5.6 to 7.9) 
[1] Glycosylated hemoglobin (HbA1c), percentage of total hemoglobin
Insulin [1] 
[Units: uIU ml^-1]
Median (Inter-Quartile Range)
 10.5 
 (6 to 21) 
[1] Insulin (u/U ml^-1)
Leptin [1] 
[Units: Ng ml ^-1]
Mean (Standard Deviation)
 36.4  (18.3) 
[1] Leptin (ng ml ^-1)
Acy Ghr [1] 
[Units: Pg ml ^-1]
Median (Inter-Quartile Range)
 362.3 
 (259 to 740.7) 
[1] Acylated ghrelin, Acy Ghr (pg ml ^-1)
PP [1] 
[Units: Pg ml ^-1]
Median (Inter-Quartile Range)
 89 
 (31 to 662) 
[1] Pancreatic polypeptide, PP (pg ml ^-1)
Appetite [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 
Total Appetite Score   32.2  (8.7) 
Appetite Behavior   14.5  (4.9) 
Appetite Drive   12.6  (3.1) 
Appetite Severity   5.1  (1.5) 
[1]

Appetite scores - For 1 and 2: 11 item questionnaire divided into subcategories of behavior (5 questions), drive (4 questions), severity (2 questions). Tallied and analyzed as total and subcategory scores. Each question scored 1-5 with higher scores correlating with worse hyperphagia.

Possible ranges: Total 11-55, behavior 5-25, drive 4-20, severity 2-10

Adiposity [1] 
[Units: Percentage of fat]
 44.7 
[1] Truncal fat as measured by dual x-ray absorptiometry
Adiposity [1] 
[Units: Kg]
Mean (Standard Deviation)
 20  (4.6) 
[1] Mean truncal fat


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Change in Weight   [ Time Frame: 6 months ]

2.  Primary:   % Change in Body Mass Index (BMI)   [ Time Frame: 6 months ]

3.  Primary:   Change in BMI Z-Score   [ Time Frame: 6 months ]

4.  Primary:   Change in HbA1c (%)   [ Time Frame: 6 months ]

5.  Primary:   Change in Insulin Levels   [ Time Frame: 6 months ]

6.  Primary:   Change in Leptin   [ Time Frame: 6 months ]

7.  Primary:   Change in Acy Ghr   [ Time Frame: 6 months ]

8.  Primary:   Change in Pancreatic Peptide (PP)   [ Time Frame: 6 months ]

9.  Primary:   Appetite Scores   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
  • small sample size
  • referral bias due to small recruitment area
  • confounding factors during the study, including variations in eating schedule, psychotropic medications, caretaker supervision and activity


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Mitchell Geffner
Organization: Children's Hospital Los Angeles
phone: 323-361-7032
e-mail: mgeffner@chla.usc.edu


Publications of Results:
Other Publications:


Responsible Party: Mitchell E. Geffner, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT01444898     History of Changes
Other Study ID Numbers: CCI 11-00227
Study First Received: September 27, 2011
Results First Received: June 14, 2016
Last Updated: September 27, 2016
Health Authority: United States: Institutional Review Board