Growth Hormone Study in Adults With Prader-Willi Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Prader-Willi Syndrome. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Netherlands
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Growth Hormone Study in Adults With Prader-Willi Syndroom
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.
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.
Primary outcome measures
- Change in lean body mass [Time frame: 36 months]
Secondary outcome measures (6)
- Change in fat mass [Time frame: 36 months]
- Change in bone density [Time frame: 36 months]
- Change in physical strength [Time frame: 36 months]
- Change in laboratory measurements [Time frame: 36 months]
- Change in psychosocial functioning [Time frame: 36 months]
- Change in caregiver burden [Time frame: 36 months]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Netherlands · 1 center
- Erasmus MC, University Medical Center Rotterdam — Rotterdam
Identifiers
NCT: NCT04484051 · GAP