Growth Hormone Study in Adults With Prader-Willi Syndrome

N/ANot Yet RecruitingNCT04484051

Erasmus Medical Center25 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Prader-Willi Syndrome

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Erasmus MC, University Medical Center Rotterdam

Rotterdam, South Holland, Netherlands

Outcomes

Primary Outcomes

Change in lean body mass

Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan

Time frame: 36 months

Secondary Outcomes

Change in fat mass

Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan

Time frame: 36 months

Change in bone density

Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan

Time frame: 36 months

Change in physical strength

Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests

Time frame: 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)

Time frame: 36 months

Change in psychosocial functioning

Change in psychosocial functioning as estimated with the Adult Behaviour Checklist

Time frame: 36 months

Central Contacts

Laura de Graaff, MD, PhD

CONTACT

0031618843010 l.degraaff@erasmusmc.nl

Trui van Essen, MD

CONTACT

0031658869674 g.vanessen@erasmusmc.nl

Data from ClinicalTrials.gov

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