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Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00399893
Recruitment Status : Terminated (Inadequate recruitment)
First Posted : November 15, 2006
Results First Posted : July 24, 2014
Last Update Posted : July 24, 2014
Sponsor:
Collaborators:
National Institutes of Health (NIH)
National Center for Research Resources (NCRR)
Novartis
Information provided by (Responsible Party):
Duke University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Prader-Willi Syndrome
Interventions Drug: Octreotide
Drug: Placebo
Enrollment 5
Recruitment Details Participants for the study were recruited over a period of 2.5 years. Participants were identified from the medical clinics or from IRB approved advertisements.
Pre-assignment Details Participants were eligible to enroll into the study with a diagnosis of PWS proven by chromosome analysis and thyroid function tests within normal ranges. Participants could not have used the study drug within the past year and could not have been on steroids within the past 30 days. These items would exclude participation.
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Period Title: Overall Study
Started 2 3
Completed 1 [1] 3
Not Completed 1 0
Reason Not Completed
Withdrawal by Subject             1             0
[1]
Participant withdrew after 3 months but was included in results
Arm/Group Title Octreotide Placebo Comparator Total
Hide Arm/Group Description

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study Total of all reporting groups
Overall Number of Baseline Participants 2 3 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 3 participants 5 participants
<=18 years
2
 100.0%
3
 100.0%
5
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 2 participants 3 participants 5 participants
11  (4.2) 13  (4.6) 11.8  (4.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 3 participants 5 participants
Female
2
 100.0%
1
  33.3%
3
  60.0%
Male
0
   0.0%
2
  66.7%
2
  40.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 2 participants 3 participants 5 participants
2 3 5
1.Primary Outcome
Title Number of Participants With Decrease in Fasting Total Ghrelin
Hide Description Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description

Descriptive statistics or percent of change from the baseline for the 5 patients was used.

A patient withdrew but included in analysis.

Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: Participants
1 1
2.Primary Outcome
Title Number of Participants With Decrease in Weight From Baseline to 6 Months
Hide Description Number of participants who had a decrease in weight from baseline to 6 months of Octreotide or placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
0 2
3.Primary Outcome
Title Number of Participants With Decreased BMI Z-score From Baseline to 6 Months
Hide Description Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
A participant withdrew but was included in analysis
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
0 2
4.Primary Outcome
Title Number of Participants With Decreased Skin-fold Measurements From Baseline to 6 Months
Hide Description Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
The skin fold measurements were performed; however, the data was not completed in a manner that could be analyzed; therefore we could not analyze the data. Completed data is not available to complete this outcome.
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Primary Outcome
Title Number of Participants With Decrease in Hunger and Food Intake
Hide Description Measured by hunger and hyperphagia by questionnaires and parent-reported 72-hour food recall from baseline to 6 months. Multiple questionnaires consisting of a battery of free text answer questions and food diaries are combined in order to make a behavioral assessment of the participants food state of hunger and food intake. There is no defined scale for this assessment. Each participants responses and parent responses are combined.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
For this outcome measure, data from questionnaires and forms was not completed. Data for all questionnaires is not available to report.
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
6.Primary Outcome
Title Number of Participants With Improved Insulin Regulation From Baseline to 6 Months
Hide Description Number of participants with improved Insulin regulation from baseline to 6 months of Octreotide or Placebo therapy. Insulin regulation was measured by immunochemiluminescent assay.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participant withdrew but was used in the data analysis
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
2 0
7.Primary Outcome
Title Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months
Hide Description Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
A participant withdrew but data was used for analysis
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
2 2
8.Primary Outcome
Title Number of Participants With Improved Leptin Regulation From Baseline to 6 Months
Hide Description Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
1 2
9.Primary Outcome
Title Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months
Hide Description Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participant withdrew but data was used for analysis
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
1 1
10.Secondary Outcome
Title Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD®
Hide Description Number of participants with decreased body-composition as Measured by BOD POD® body composition tracking system from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participant withdrew but data was used for analysis
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
2 3
11.Secondary Outcome
Title Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA
Hide Description Number of participants with decreased body-composition as Measured by Dual Energy X-ray Absorptiometry (DEXA) scan from baseline to 6 months of Octreotide or Placebo therapy
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Data for this outcome was not analyzed
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description:

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
Overall Number of Participants Analyzed 2 3
Measure Type: Number
Unit of Measure: participants
0 0
Time Frame Adverse event collection began with administration of study drug or placebo and concluded 30 days after discontinuation of study drug.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Octreotide Placebo Comparator
Hide Arm/Group Description

Octreotide :

Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily

Placebo to be administered by subcutaneous injection three times daily while on study
All-Cause Mortality
Octreotide Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Octreotide Placebo Comparator
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/2 (0.00%)      0/3 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Octreotide Placebo Comparator
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/2 (0.00%)      1/3 (33.33%)    
Gastrointestinal disorders     
Gallstones  [1]  0/2 (0.00%)  0 1/3 (33.33%)  1
Indicates events were collected by systematic assessment
[1]
One patient in the placebo group developed a small gallstone at 6 months; all patients in the active treatment group maintained normal gallbladder ultrasounds at 6 months.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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
Layout table for Results Point of Contact information
Name/Title: Michael Freemark, MD
Organization: Duke University Medical Center
Phone: 919-684-5091
EMail: andrea.haqq@albertahealthservices.ca
Layout table for additonal information
Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT00399893    
Other Study ID Numbers: Pro00005426
Protocol #00005426 ( Other Grant/Funding Number: NIH:1K23-RR021979, GCRC M01-RR-30 (NCRR) )
First Submitted: November 14, 2006
First Posted: November 15, 2006
Results First Submitted: January 28, 2013
Results First Posted: July 24, 2014
Last Update Posted: July 24, 2014