Contact information
Type
Scientific
Contact name
Dr Dederieke Festen
ORCID ID
Contact details
Dutch Growth Foundation
Westzeedijk 106
Rotterdam
3016 AH
Netherlands
+31 (0)10 2251533
d.festen@erasmusmc.nl
Additional identifiers
EudraCT/CTIS number
IRAS number
ClinicalTrials.gov number
Protocol/serial number
NTR628
Study information
Scientific title
Multicentre, randomised, controlled growth hormone study in children with Prader-Willi syndrome: effects on growth, body composition, activity level and psychosocial development
Acronym
Study hypothesis
Growth hormone (GH) 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.
Ethics approval(s)
Local medical ethics committee gave approval
Study design
Multicentre randomised active-controlled parallel group trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Study setting(s)
Hospital
Study type
Treatment
Patient information sheet
Condition
Prader-Willi syndrome
Intervention
Treatment with GH: Genotropin® 1 mg/m^2/day subcutaneously (sc) versus no GH-treatment. Dietary and exercise advice.
Intervention type
Drug
Pharmaceutical study type(s)
Phase
Not Applicable
Drug/device/biological/vaccine name(s)
Genotropin®
Primary outcome measure
To asses effects of GH-treatment versus no GH-treatment in children with Prader-Willi syndrome on:
1. Height, weight, body composition, muscle mass, muscle strength and daily life activity
2. Cognition, behaviour and social emotional development
3. Resting energy expenditure
4. Psychomotor development in infants
Secondary outcome measures
To study the effect of additional dietary advice and physical exercise on body composition in children with Prader-Willi syndrome treated with GH versus not treated with GH.
Overall study start date
23/04/2002
Overall study end date
01/05/2007
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. 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
Participant type(s)
Patient
Age group
Child
Lower age limit
6 Months
Upper age limit
16 Years
Sex
Both
Target number of participants
85
Participant exclusion criteria
1. Extremely low dietary intake
2. Severe scoliosis (consult spinal surgeon)
3. Body mass index (BMI) SDS greater than +3
4. In children greater than 3 years, height SDS less than 0 unless weight for height greater than +2SDS
Recruitment start date
23/04/2002
Recruitment end date
01/05/2007
Locations
Countries of recruitment
Netherlands
Study participating centre
Dutch Growth Foundation
Rotterdam
3016 AH
Netherlands
Funders
Funder type
Industry
Funder name
Pfizer (Netherlands)
Alternative name(s)
Pfizer Inc., Pfizer Consumer Healthcare, Davis
Funding Body Type
government organisation
Funding Body Subtype
For-profit companies (industry)
Location
United States of America
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Individual participant data (IPD) sharing plan
IPD sharing plan summary
Not provided at time of registration
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results on effect of GH-treatment on incidence of scoliosis | 01/04/2009 | Yes | No | |
| Results article | results on effect of GH-treatment on bone density | 01/10/2009 | Yes | No | |
| Results article | ovarian function results | 01/09/2012 | Yes | No |