Ghrelin Suppression by Octreotide in Prader-Willi

Study Overview
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Brief Summary
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The purpose of this study is to learn more about how octreotide (Sandostatin LAR® Depot) affects levels of ghrelin, hunger, and body weight in people with Prader-Willi Syndrome.

Detailed Description
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After baseline tests, a nurse will administer monthly injections of Sandostatin LAR® or placebo for 6 months. At the end of this initial 6-month treatment period and a 4-month washout period, study subjects will then crossover to receive the alternative therapy (placebo or octreotide) for an additional 6 months. Subjects will be followed for 16 months total at scheduled visits: 0, 2, 6, 10, 12, and 16 months (Table 2).

Official Title
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Ghrelin Suppression by Sandostatin LAR® Depot (Octreotide Acetate for Injectable Suspension) in Patients With Prader-Willi Syndrome

Conditions
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Prader Willis Syndrome

Intervention / Treatment
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* Drug: Placebo
* Drug: Octreotide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: o Diagnosis of PWS confirmed by chromosome analysis (i.e. interstitial deletion of paternally-derived chromosome 15Q, uniparental maternal disomy or other chromosome 15 abnormalities) Age 18 years and older Written informed consent obtained (by subject or guardian) and willingness to comply with the study schedule and procedures Free T4, TSH values in the normal range (with or without thyroxine replacement) Subjects with confirmed hypogonadism who are corrected with adequate doses of sex steroid replacement, will have been treated for at least 6 months prior to entry and have no change in dosages over the study period. Patients with confirmed growth hormone deficiency who are corrected with adequate doses of replacement, will have been treated for at least 6 months prior to entry and have no change in dosages over the study period. Exclusion Criteria: Symptomatic gallstones. Smokers. Alcohol consumption > 2 drinks per day. Exercise > 30 minutes 3 times a week. Weight > 500 lbs (exceeds the limit for the BODPOD). Active pulmonary infection. Sleep disorder. Subjects will be excluded if they have clinically significant disease that would have an impact on metabolic outcomes or body composition including diabetes mellitus, chronic inflammatory bowel disease, chronic severe liver or kidney disease, heart disease, cancer, symptomatic gallstones, or neurological disorders history of hepatic disease (patients with minimal, i.e., <3Xs the upper limit of normal for LFTs indicative of hepatic steatosis, MAY participate) pregnant or lactating known hypersensitivity to Sandostatin acetate or other related drug or compound subjects who exhibit symptoms indicative of intolerance following the 100 mcg test dose of Sandostatin Injection, SC subjects who have previously received Sandostatin® (octreotide) subjects who have received other investigational drugs administered or received within 30 days of study entry

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Normal Saline | Drug: Placebo<br>* After the first study visit, subjects will begin monthly treatment with either the study drug or a placebo (an inactive substance), which will be continued for the first six months of the study. For the last six months of the study, subjects will be switched to the opposite treatment. During these study periods participants will return monthly for administration of study medication, physical examination, and blood draw to check liver enzymes.<br>* Other names: Normal Saline;|
| Active Comparator: Octreotide<br>They will be admitted to the inpatient unit of the OCTRI for testing six times. During each of these visits, testing will include measuring how well glucose (sugar) is processed, how much energy is burned off as heat, their amount of body fat, levels of the hormone ghrelin, and how much food is eaten at a meal. After the first study visit, subjects will begin monthly treatment with either the study drug or a placebo (an inactive substance), which will be continued for the first six months of the study. For the last six months of the study, subjects will be switched to the opposite treatment. During these study periods participants will return monthly for administration of study medication, physical examination, and blood draw to check liver enzymes. | Drug: Octreotide<br>* They will be admitted to the inpatient unit of the OCTRI for testing six times. During each of these visits, testing will include measuring how well glucose (sugar) is processed, how much energy is burned off as heat, their amount of body fat, levels of the hormone ghrelin, and how much food is eaten at a meal. After the first study visit, subjects will begin monthly treatment with either the study drug or a placebo (an inactive substance), which will be continued for the first six months of the study. For the last six months of the study, subjects will be switched to the opposite treatment. During these study periods participants will return monthly for administration of study medication, physical examination, and blood draw to check liver enzymes.<br>* Other names: Sandostatin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ghrelin levels |  | Change from baseline to 6 months | 
| Appetite |  | Change from baseline to 6 months | 
| Body weight |  | Change from baseline to 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hormone levels |  | Change from baseline to 6 months | 
| Body composition |  | Change from baseline to 6 months | 
| Energy expenditure |  | Change from baseline to 6 months | 
| Glucose metabolism |  | Change from baseline to 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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obesity