|- candidate number||2704|
|- NTR Number||NTR1038|
|- Date ISRCTN created||5-sep-2007|
|- date ISRCTN requested||31-aug-2007|
|- Date Registered NTR||16-aug-2007|
|- Secondary IDs||n/a |
|- Public Title||Effects of growth hormone treatment after final height in Prader-Willi Syndrome|
|- Scientific Title||Effects of growth hormone treatment after final height in Prader-Willi Syndrome: A double-blind multicenter, cross-over study on the effects of growth hormone versus placebo on body composition and psychosocial behaviour in transition|
|- hypothesis||GH treatment after reaching final height is beneficial for body composition and social wellbeing in young adults with PWS|
|- Healt Condition(s) or Problem(s) studied||Prader-Willi syndrome|
|- Inclusion criteria||1. Young adults, originally participating in the Dutch GH study in PWS children (ISRCTN49726762) or otherwise GH-treated patients and
2. Final height is reached or epiphysial fusion is complete and
3. Treated with GH during childhood for at least 2 years
|- Exclusion criteria||1. non cooperative behaviour
2. extremely low dietary intake of less than minimal required intake according to WHO
3. medication to reduce weight (fat)
|- mec approval received||no|
|- multicenter trial||yes|
|- planned startdate ||1-okt-2007|
|- planned closingdate||1-okt-2011|
|- Target number of participants||20|
|- Interventions||Treatment with GH: Genotropin 0.67 mg/m2/day s.c. or placebo|
|- Primary outcome||To assess effects of GH-treatment versus placebo on
a. body composition
b. carbohydrate metabolism
c. psychosocial functioning
d. sleep-related breathing disorders
e. circulating lipids
f. blood pressure
|- Secondary outcome||1. To study the effects of GH-treatment versus placebo on thyroid hormone levels, IGF-I and IGF binding proteins, adiponectin, ghrelin.
2. To study compliance to the diet.
|- Trial web site||n/a|
|- CONTACT FOR PUBLIC QUERIES||MD. Dederieke Festen|
|- CONTACT for SCIENTIFIC QUERIES||Prof. Dr. A.C.S. Hokken-Koelega|
|- Sponsor/Initiator ||Dutch Growth Foundation|
(Source(s) of Monetary or Material Support)
|- Brief summary||Background: GH improves height velocity, and body composition in PWS children. Preliminary data also suggest improvement of psychosocial functioning during GH. When epiphysial fusion is complete and final height is reached, GH-treatment has to be discontinued. However, discontinuation of GH results in a decrease of lean body mass, an increase of body fat percentage and a deterioration of psychosocial behaviour. A preliminary study showed that also young adults with PWS might benefit from GH-treatment, with regard to body composition, and psychosocial wellbeing.
To assess effects of GH-treatment versus placebo on
- body composition
- carbohydrate metabolism
- psychosocial functioning
- sleep-related breathing disorders
- circulating lipids
- blood pressure
- To study the effects of GH-treatment versus placebo on thyroid hormone levels, IGF-I and IGF binding proteins, adiponectin, ghrelin.
- To study compliance to the diet.
Patients: subjects with PWS, aged 18-24 years, who reached final height after they were treated with GH according to the Dutch National GH study in children with PWS (ISRCTN49726762), or after they were otherwise treated with GH (at least 2 years) during childhood.
Intervention: Treatment with GH: Genotropin 0.67 mg/m2/day s.c. or placebo
Design/Assessments: After stratification for BMI, gender, originally followed in the GH study vs. otherwise GH-treated patients, subjects will be randomized to either placebo or GH-treatment group, according to a double blind, placebo-controlled cross-over design during the first 2 years. After 2 years, all patients receive GH treatment in a dose of 0.67 mg/m2/day, after ATT-GHRH test has been performed. Anthropometric assessments and blood pressure will be performed every 3 months. Six-monthly, assessment of body composition (DXA), carbohydrate metabolism and circulating lipids and other laboratory parameters will be performed. Yearly, evaluation of sleep-related breathing (polysomnography), and cognition and behaviour (GIT, TVZ) will be performed.
|- Main changes (audit trail)|
|- RECORD||16-aug-2007 - 24-sep-2007|