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Controlled growth hormone study in children with Prader Willi Syndrome.


- candidate number2018
- NTR NumberNTR628
- ISRCTNISRCTN49726762
- Date ISRCTN created28-apr-2006
- date ISRCTN requested25-apr-2006
- Date Registered NTR15-mrt-2006
- Secondary IDsN/A 
- Public TitleControlled growth hormone study in children with Prader Willi Syndrome.
- Scientific TitleMulticenter, randomized, controlled growth hormone study in children with Prader Willi Syndrome: effects on growth, body composition, activity level and psychosocial development.
- ACRONYMN/A
- hypothesisGH treatment improves height, weight, body composition, muscle strength, activity level, psychosocial development, psychomotor development in infants, metabolism and respiratory function versus no GH treatment in children with Prader Willi Syndrome.
- Healt Condition(s) or Problem(s) studiedPrader-Willi syndrome
- Inclusion criteria1. Genetically confirmed diagnosis of Prader Willi Syndrome;
2. Age between 6 months and 16 years at start of the study;
3. Bone age less than 16 years.
- Exclusion criteria1. Extremely low dietary intake;
2. Severe scoliosis (consult spinal surgeon);
3. BMI SDS > +3SDS;
4. In children > 3 years, height SDS < 0 unless weight for height> +2SDS.
- mec approval receivedyes
- multicenter trialyes
- randomisedyes
- masking/blindingNone
- controlActive
- groupParallel
- Type2 or more arms, randomized
- Studytypeintervention
- planned startdate 23-apr-2002
- planned closingdate1-mei-2007
- Target number of participants85
- InterventionsTreatment with GH: Genotropin ® 1mg/m2/d s.c. vs. no GH-treatment.
Dietary and exercise advice.
- Primary outcomeTo asses effects of GH-treatment vs. no GH-treatment in children with PWS on:
height, weight, body composition, muscle mass, muscle strength and daily life activity.
Cognition, behaviour and social emotional development. Resting Energy Expenditure.
Psychomotor development in infants.
- Secondary outcomeTo study the effect of additional dietary advise and physical exercise on body composition in children with PWS treated with GH vs, not treated with GH.
- TimepointsN/A
- Trial web siteN/A
- statusinclusion stopped: follow-up
- CONTACT FOR PUBLIC QUERIESMD. Dederieke Festen
- CONTACT for SCIENTIFIC QUERIESProf. Dr. A.C.S. Hokken-Koelega
- Sponsor/Initiator Dutch Growth Foundation
- Funding
(Source(s) of Monetary or Material Support)
Pfizer
- PublicationsN/A
- Brief summarySummary of the Dutch National Growth Hormone Study in Children with Prader Willi Syndrome.

Title:
Multicenter, randomized, controlled growth hormone study in children with Prader Willi Syndrome: effects on growth, body composition, activity level and psychosocial development” MEC 2001/230.

Short title:
Controlled growth hormone study in children with Prader Willi Syndrome.

Background:
Children with Prader Willi Syndrome have often short stature and have an abnormal body composition (Increased fatpercentage and decreased lean body mass). Physical activity level is therefore decreased in children with Prader Willi Syndrome. Children with Prader Willi syndrome often have mental retardation and behavioral problems.
Recent studies showed an improvement in height and body composition during growth hormone (GH) treatment in children with Prader Willi Syndrome. Preliminary data showed also an improvement in activity level, metabolism, respiratory function, behaviour and social-emotional development.

Aim:
To study the effects of GH-treatment versus no GH treatment in children with Prader Willi Syndrome on changes in height, weight, body composition, muscle strength, activity level, psychosocial development, metabolism and respiratory function.

Patients:
85 children with PWS, aged 6 mo. to 16 years at start of the study.

Intervention:
Treatment with GH: Genotropin ® 1mg/m2/d s.c.

Design:
Children will be randomly divided into 3 subgroups: infants, prepubertal children and adolescents. Infants and prepubertal children will be treated in a controlled design. (Start GH-treatment at start of the study or after 1 or two years resp.) Adolescents will be treated in a non-controlled design. All children over the age of 3 will receive dietary advice and an exercise program.
Anthropometric assessments will be performed every 3 months.
Yearly assessment of body composition (DEXA), metabolism (Indirect calorimetry), muscle strength (quadriceps dynamometry) and activity level (Activity monitoring) will be performed.
Cognition (subtests of WISC, WPPSI or BOS), behaviour (DBC), social emotional development (VABS) and quality of life (DUX 25) will be measured yearly.
In a subgroup of children pulmonary CO2 responsiveness will be measured.

Objectives:
Primary:
To asses effects of GH-treatment vs. no GH-treatment in children with PWS on Height, weight, body composition Muscle mass, muscle strength and daily life activity Cognition, behaviour and social emotional development Resting Energy Expenditure CO2 responsiveness.
Secondary:
To study the effect of additional dietary advise and physical exercise on body composition in children with PWS treated with GH vs, not treated with GH.
- Main changes (audit trail)
- RECORD15-mrt-2006 - 1-dec-2009


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