Trial Outcomes & Findings for Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS) (NCT NCT00399893)

Results Overview

Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

5 participants

Primary outcome timeframe

6 months

Results posted on

2014-07-24

Participant Flow

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.

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.

Participant milestones

Participant milestones
Measure	Octreotide Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator Placebo to be administered by subcutaneous injection three times daily while on study
Overall Study STARTED	2	3
Overall Study COMPLETED	1	3
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Octreotide Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator Placebo to be administered by subcutaneous injection three times daily while on study
Overall Study Withdrawal by Subject	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study	Total n=5 Participants Total of all reporting groups
Age, Categorical <=18 years	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	11 years STANDARD_DEVIATION 4.2 • n=5 Participants	13 years STANDARD_DEVIATION 4.6 • n=7 Participants	11.8 years STANDARD_DEVIATION 4.0 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Descriptive statistics or percent of change from the baseline for the 5 patients was used. A patient withdrew but included in analysis.

Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Decrease in Fasting Total Ghrelin	1 Participants	1 Participants

PRIMARY outcome

Timeframe: 6 months

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

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Decrease in Weight From Baseline to 6 Months	0 participants	2 participants

PRIMARY outcome

Timeframe: 6 months

Population: A participant withdrew but was included in analysis

Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Decreased BMI Z-score From Baseline to 6 Months	0 participants	2 participants

PRIMARY outcome

Timeframe: 6 months

Population: 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.

Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 6 months

Population: For this outcome measure, data from questionnaires and forms was not completed. Data for all questionnaires is not available to report.

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.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 6 months

Population: Participant withdrew but was used in the data analysis

Number of participants with improved Insulin regulation from baseline to 6 months of Octreotide or Placebo therapy. Insulin regulation was measured by immunochemiluminescent assay.

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Improved Insulin Regulation From Baseline to 6 Months	2 participants	0 participants

PRIMARY outcome

Timeframe: 6 months

Population: A participant withdrew but data was used for analysis

Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months	2 participants	2 participants

PRIMARY outcome

Timeframe: 6 months

Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Improved Leptin Regulation From Baseline to 6 Months	1 participants	2 participants

PRIMARY outcome

Timeframe: 6 months

Population: Participant withdrew but data was used for analysis

Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months	1 participants	1 participants

SECONDARY outcome

Timeframe: 6 months

Population: Participant withdrew but data was used for analysis

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

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD®	2 participants	3 participants

SECONDARY outcome

Timeframe: 6 months

Population: Data for this outcome was not analyzed

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

Outcome measures

Outcome measures
Measure	Octreotide n=2 Participants Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 Participants Placebo to be administered by subcutaneous injection three times daily while on study
Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA	0 participants	0 participants

Adverse Events

Octreotide

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo Comparator

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Octreotide n=2 participants at risk Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily	Placebo Comparator n=3 participants at risk Placebo to be administered by subcutaneous injection three times daily while on study
Gastrointestinal disorders Gallstones	0.00% 0/2 • Adverse event collection began with administration of study drug or placebo and concluded 30 days after discontinuation of study drug.	33.3% 1/3 • Number of events 1 • Adverse event collection began with administration of study drug or placebo and concluded 30 days after discontinuation of study drug.

Additional Information

Michael Freemark, MD

Duke University Medical Center

Phone: 919-684-5091

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60