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

• ClinicalTrials.gov Identifier: NCT00399893
• Recruitment Status: Terminated
• First Posted: November 15, 2006
• Results First Posted: July 24, 2014
• Last Update Posted: July 24, 2014
• Sponsor: Duke University
• 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

Participant Flow

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
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

Baseline Characteristics

Arm/Group Title		Octreotide	Placebo Comparator	Total
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
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

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Decrease in Fasting Total Ghrelin
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

Outcome Measure Data

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
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
Description	Number of participants who had a decrease in weight from baseline to 6 months of Octreotide or placebo therapy
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Octreotide	Placebo Comparator
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
Description	Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

A participant withdrew but was included in analysis

Arm/Group Title	Octreotide	Placebo Comparator
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
Description	Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy
Time Frame	6 months

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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

Outcome Measure Data

Analysis Population Description

Participant withdrew but was used in the data analysis

Arm/Group Title	Octreotide	Placebo Comparator
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
Description	Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

A participant withdrew but data was used for analysis

Arm/Group Title	Octreotide	Placebo Comparator
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
Description	Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Octreotide	Placebo Comparator
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
Description	Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

Participant withdrew but data was used for analysis

Arm/Group Title	Octreotide	Placebo Comparator
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®
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

Outcome Measure Data

Analysis Population Description

Participant withdrew but data was used for analysis

Arm/Group Title	Octreotide	Placebo Comparator
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
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

Outcome Measure Data

Analysis Population Description

Data for this outcome was not analyzed

Arm/Group Title	Octreotide	Placebo Comparator
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

Adverse Events

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
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	--/--		--/--
Serious Adverse Events
	Octreotide		Placebo Comparator
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/2 (0.00%)		0/3 (0.00%)
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.

Limitations and Caveats

[Not Specified]

More Information

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

• Name/Title: Michael Freemark, MD
• Organization: Duke University Medical Center
• Phone: 919-684-5091
• EMail: andrea.haqq@albertahealthservices.ca

Publications:

Haqq AM, Stadler DD, Rosenfeld RG, Pratt KL, Weigle DS, Frayo RS, LaFranchi SH, Cummings DE, Purnell JQ. Circulating ghrelin levels are suppressed by meals and octreotide therapy in children with Prader-Willi syndrome. J Clin Endocrinol Metab. 2003 Aug;88(8):3573-6. doi: 10.1210/jc.2003-030205.

• 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