Growth Hormone Study in Adults With Prader-Willi Syndrome
- Conditions
- Prader-Willi Syndrome
- Registration Number
- NCT04484051
- Lead Sponsor
- Erasmus Medical Center
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 25
- The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in lean body mass 36 months Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
- Secondary Outcome Measures
Name Time Method Change in fat mass 36 months Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
Change in bone density 36 months Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan
Change in physical strength 36 months Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests
Change in laboratory measurements 36 months 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)Change in psychosocial functioning 36 months Change in psychosocial functioning as estimated with the Adult Behaviour Checklist
Change in caregiver burden 36 months Change in caregiver burden as estimated with the Zarit Burden Interview
Trial Locations
- Locations (1)
Erasmus MC, University Medical Center Rotterdam
🇳🇱Rotterdam, Zuid-Holland, Netherlands