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| ID | Type | Description | Link |
|---|---|---|---|
| Protocol #00005426 | Other Grant/Funding Number | NIH:1K23-RR021979, GCRC M01-RR-30 (NCRR) |
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Inadequate recruitment
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| Name | Class |
|---|---|
| National Institutes of Health (NIH) | NIH |
| National Center for Research Resources (NCRR) | NIH |
| Novartis | INDUSTRY |
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The purpose of this study is to investigate over a 6 month period the effect of octreotide therapy on food intake, sense of hunger, body weight, body composition, efficiency of burning calories, biomarkers of weight regulation and growth hormone markers in children and young Adults with Prader-Willi Syndrome(PWS).
Obesity continues to be a prevalent health concern affecting every race of the American population. According to data from the World Health Organization, 54% of U.S. adults are overweight (body mass index (BMI) >25 kg/m2 ) and 22% are obese (BMI >30 kg/m2) (1). In addition, 25% of U.S. children are overweight or obese (1). Studies show that obese children are likely to become obese adults (2-5). Also, recent studies report significant years of life lost due to the impact of being an obese adult (6, 7). Thus, insights into the pathogenesis of childhood obesity and preventative measures are needed to combat the inevitable increase in worldwide incidence of obesity and its associated co-morbidities. Recent studies have identified a new gastroenteric hormone, ghrelin, as a long-term regulator of energy balance in humans (12). Ghrelin is a 28 amino acid acylated peptide which is an endogenous ligand of the growth hormone secretagogue receptor (GHS-R), a hypothalamic G-protein-coupled receptor (13). Enteroendocrine cells (X/A-like cells) of the stomach are the major site of ghrelin synthesis, although a minor proportion of ghrelin synthesis occurs in other sites such as the hypothalamus, pituitary, duodenum, jejunum and lung (14) (15, 16).
The hypothesis that hyperghrelinemia causes some of the features of PWS predicts that this disorder will be ameliorated (partially or completely) by lowering ghrelin levels. We have recently shown that the somatostatin agonist, octreotide, suppresses ghrelin levels in humans. If octreotide remains effective in longer term studies, the drug may become an adjuvant therapy, in addition to growth hormone, to control the insatiable appetite and morbid obesity seen in this condition.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Octreotide | Experimental | Octreotide to be administered by subcutaneous injection three times daily while on study |
|
| Placebo | Placebo Comparator | Placebo to be administered by subcutaneous injection three times daily while on study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Octreotide | Drug | Octreotide to be administered by subcutaneous injection three times daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Decrease in Fasting Total Ghrelin | Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo | 6 months |
| Number of Participants With Decrease in Weight From Baseline to 6 Months | Number of participants who had a decrease in weight from baseline to 6 months of Octreotide or placebo therapy | 6 months |
| Number of Participants With Decreased BMI Z-score From Baseline to 6 Months | Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy | 6 months |
| Number of Participants With Decreased Skin-fold Measurements From Baseline to 6 Months | Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy | 6 months |
| Number of Participants With Decrease in Hunger and Food Intake | 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. | 6 months |
| Number of Participants With Improved Insulin Regulation From Baseline to 6 Months | Number of participants with improved Insulin regulation from baseline to 6 months of Octreotide or Placebo therapy. Insulin regulation was measured by immunochemiluminescent assay. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD® | 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 | 6 months |
| Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Andrea M Haqq, MD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 12915638 | Background | 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. |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Octreotide | Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily |
| FG001 | Placebo Comparator | Placebo to be administered by subcutaneous injection three times daily while on study |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Octreotide | Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily |
| BG001 | Placebo Comparator | Placebo to be administered by subcutaneous injection three times daily while on study |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Decrease in Fasting Total Ghrelin | Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo | Descriptive statistics or percent of change from the baseline for the 5 patients was used. A patient withdrew but included in analysis. | Posted | Number | Participants | 6 months |
|
Adverse event collection began with administration of study drug or placebo and concluded 30 days after discontinuation of study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Octreotide | Octreotide : Octreotide : Octreotide or placebo to be administered by subcutaneous injection three times daily |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gallstones | Gastrointestinal disorders | Systematic Assessment | 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. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Michael Freemark, MD | Duke University Medical Center | 919-684-5091 | andrea.haqq@albertahealthservices.ca |
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| ID | Term |
|---|---|
| D011218 | Prader-Willi Syndrome |
| D063766 | Pediatric Obesity |
| D015431 | Weight Loss |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D015282 | Octreotide |
| ID | Term |
|---|---|
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
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| Placebo | Drug | Placebo to be administered by subcutaneous injection three times daily while on study |
|
| 6 months |
| Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months | Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy | 6 months |
| Number of Participants With Improved Leptin Regulation From Baseline to 6 Months | Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy | 6 months |
| Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months | Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy | 6 months |
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 |
| 6 months |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Secondary | Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD® | 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 | Participant withdrew but data was used for analysis | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Decrease in Weight From Baseline to 6 Months | Number of participants who had a decrease in weight from baseline to 6 months of Octreotide or placebo therapy | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Decreased BMI Z-score From Baseline to 6 Months | Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy | A participant withdrew but was included in analysis | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Decreased Skin-fold Measurements From Baseline to 6 Months | Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy | 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. | Posted | 6 months |
|
|
| Primary | Number of Participants With Decrease in Hunger and Food Intake | 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. | For this outcome measure, data from questionnaires and forms was not completed. Data for all questionnaires is not available to report. | Posted | 6 months |
|
|
| Secondary | Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA | 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 | Data for this outcome was not analyzed | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Improved Insulin Regulation From Baseline to 6 Months | Number of participants with improved Insulin regulation from baseline to 6 months of Octreotide or Placebo therapy. Insulin regulation was measured by immunochemiluminescent assay. | Participant withdrew but was used in the data analysis | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months | Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy | A participant withdrew but data was used for analysis | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Improved Leptin Regulation From Baseline to 6 Months | Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy | Posted | Number | participants | 6 months |
|
|
|
| Primary | Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months | Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy | Participant withdrew but data was used for analysis | Posted | Number | participants | 6 months |
|
|
|
| 0 |
| 2 |
| 0 |
| 2 |
| EG001 | Placebo Comparator | Placebo to be administered by subcutaneous injection three times daily while on study | 0 | 3 | 1 | 3 |
|
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| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D009765 | Obesity |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
| D001835 | Body Weight |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001836 | Body Weight Changes |
| D000602 |
| Amino Acids, Peptides, and Proteins |