Growth Hormone Study in Adults With Prader-Willi Syndrome

Study Overview
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Brief Summary
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The overall objective of this study is to measure the effect of growth hormone (GH) treatment on physical and psychosocial health in adults with Prader-Willi syndrome. Adults with PWS who have not been treated with GH during the past three years and who will start with GH treatment as part of regular patient care will be asked for informed consent to participate in this open-label prospective cohort study. We hypothesize that growth hormone treatment will improve the physical and psychosocial health.

Detailed Description
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OBJECTIVES: To measure the effect of GHt on physical and psychosocial health in adults with PWS. The primary endpoint is change in lean body mass (LBM (kg)) as assessed by Dual Energy X-ray Absorptiometry (DEXA) scan. Secondary endpoints are total fat mass, bone density, physical health and psychosocial health. Also the occurrence of side-effects will be assessed. Only data that are collected as part of regular patient care will be used. STUDY DESIGN: Open-label prospective cohort study. STUDY POPULATION: Adults with PWS who have not been treated with GH during the past three years and who will start with GHt as part of regular patient care.

Official Title
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Growth Hormone Study in Adults With Prader-Willi Syndroom

Conditions
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Prader-Willi Syndrome

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The patient is diagnosed with Prader-Willi syndrome (genetically confirmed) Exclusion Criteria: Non cooperative behaviour Pregnancy Known malignancies Poorly controlled diabetes (HbA1c > 64 mmol/mol (8%)) Untreated obstructive sleep apnea (apnea-hypopnea index > 5) Body mass index above 40 kg/m2 Upper-airway obstruction of any cause

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Routine clinical care with Genotropin treatment<br>Data collection throughout routine clinical care with subcutaneous injections Genotropin, 0.6-0.8 mg/day. Participants start with 0.2 mg/day and the dose increases with 0.2 mg/day per month to a maximum dose of 0.6-0.8 mg/day. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in lean body mass | Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan | 36 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in fat mass | Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan | 36 months | 
| Change in bone density | Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan | 36 months | 
| Change in physical strength | Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests | 36 months | 
| Change in laboratory measurements | Changes in the following laboratory measurements: Glycosylated hemoglobin (mmol/mol) Total cholesterol (mmol/L) Low-density lipoprotein cholesterol (mmol/L) High-density lipoprotein cholesterol (mmol/L) Insulin-like growth factor 1 (nmol/L) Free thyroxine 4 (pmol/L) Luteinizing hormone (U/I) Follicle stimulating hormone (U/I) Estradiol or testosterone (nmol/L) Sex hormone binding globulin (nmol/L) Aspartate transaminase (U/L) Alanine transaminase (U/L) Alkaline phosphatase (U/L) Gamma glutamyl transpeptidase (U/L) Total bilirubin (micromol/L) Lactate dehydrogenase (U/L) Urea (mmol/L) Creatinine (micromol/L) Hemoglobin (mmol/L) Hematocrit (L/L) Mean corpuscular volume (fL) Leukocytes (10^9/L) Thrombocytes (10^9/L) 25-OH vitamin D (nmol/L) | 36 months | 
| Change in psychosocial functioning | Change in psychosocial functioning as estimated with the Adult Behaviour Checklist | 36 months | 
| Change in caregiver burden | Change in caregiver burden as estimated with the Zarit Burden Interview | 36 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Prader-Willi syndrome, Growth hormone, Body composition, Cardiovascular disease