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ID
Source
Brief title
Health condition
Prader-Willi Syndrome
Sponsors and support
Prader-Willi Fonds
Pfizer (sponsoring of growth hormone and placebo)
Intervention
Outcome measures
Primary outcome
The primary endpoint is change in lean body mass (LBM (kg)) as assessed by Dual Energy X-ray Absorptiometry (DEXA) scan
Secondary outcome
Secondary endpoints are total fat mass, bone density, physical health, endurance, psychosocial functioning and quality of life.
Background summary
Rationale:
Prader-Willi Syndrome (PWS) is a rare, complex disorder. The mortality in PWS is 3% per year, even in young patients. Besides insatiable appetite and intellectual disability, patients with PWS show symptoms of growth hormone (GH) deficiency, like small hands and feet, a low muscle mass and increased body fat. Treatment with GH in patients with PWS improves the body composition and can therefore prevent obesity and the complications of obesity, like diabetes mellitus and cardiovascular diseases. Currently, GH treatment (GHt) in adults with PWS is only reimbursed if they have proven GH deficiency. However, due to the unsuitability of ‘regular’ GH tests for patients with PWS, (functional) GH deficiency cannot be proven in these patients. GHt in children with PWS has resulted in a tremendous improvement of the physical and mental health. We hypothesize that GHt in adults with PWS will improve their metabolic and cardiovascular health, thereby resulting in a reduced mortality.
Objective:
To measure the effect of GHt on metabolic and cardiovascular health in adults with PWS.
Study design:
Randomized, double-blinded, placebo controlled crossover trial for two years with a washout period of 3 months.
Study population:
72 adults with PWS of 30 years or older who have not been treated with GH during the past three years from the participating countries Australia and the Netherlands.
Intervention:
Participants will be randomized to placebo or GHt for one year. After a washout period of 3 months, the patients who received placebo during the first year will be switched to GHt during the second year and vice versa.
Main study parameters/endpoints:
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, endurance, psychosocial functioning and quality of life (QoL).
Study objective
Our hypothesis is that growth hormone threatment in adults with Prader-Willi Syndrome will improve their metabolic and cardiovascuar health, thereby resulting in a reduced mortality.
Study design
t=0, t=3, t=6, t=9, t=12, t=15, t=18, t=21, t=24, t=27
Intervention
Participants will be randomized to placebo or growth hormone treatment for one year. After a washout period of 3 months, the patients who received placebo during the first year will be switched to growth hormone treatment during the second year and vice versa.
Inclusion criteria
- The patient is diagnosed with PWS
- The patient is 30 years or older
- GH therapy was suspended at least three years before starting the study
Exclusion criteria
- Non cooperative behaviour
- Known malignancies
- Poorly controlled diabetes (HbA1c > 64 mmol/mol (8%))
- Untreated obstructive sleep apnea (apnea-hypopnea index > 5)
- BMI above 40 kg/m2
- Osteosynthesis material
- Testosterone suppletion is not stable for three months
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
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Other (possibly less up-to-date) registrations in this register
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In other registers
| Register | ID |
|---|---|
| NTR-new | NL8274 |
| Other | METC EMC : ABR71549 |