Controlled growth hormone (GH) study in children with Prader-Willi syndrome
| ISRCTN | ISRCTN49726762 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN49726762 |
| Secondary identifying numbers | NTR628 |
- Submission date
- 28/04/2006
- Registration date
- 28/04/2006
- Last edited
- 05/11/2012
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Dederieke Festen
Scientific
Scientific
Dutch Growth Foundation
Westzeedijk 106
Rotterdam
3016 AH
Netherlands
| Phone | +31 (0)10 2251533 |
|---|---|
| d.festen@erasmusmc.nl |
Study information
| 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 |
| Scientific title | Multicentre, randomised, controlled growth hormone study in children with Prader-Willi syndrome: effects on growth, body composition, activity level and psychosocial development |
| Study objectives | 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 |
| Health condition(s) or problem(s) studied | 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 |
| Completion date | 01/05/2007 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 6 Months |
| Upper age limit | 16 Years |
| Sex | Both |
| Target number of participants | 85 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 23/04/2002 |
| Date of final enrolment | 01/05/2007 |
Locations
Countries of recruitment
- Netherlands
Study participating centre
Dutch Growth Foundation
Rotterdam
3016 AH
Netherlands
3016 AH
Netherlands
Sponsor information
Dutch Growth Foundation (Netherlands)
Charity
Charity
Westzeedijk 106
Rotterdam
3016 AH
Netherlands
Funders
Funder type
Industry
Pfizer (Netherlands)
Government organisation / For-profit companies (industry)
Government organisation / For-profit companies (industry)
- Alternative name(s)
- Pfizer Inc., Pfizer Consumer Healthcare, Davis, Charles Pfizer & Company, Warner-Lambert, King Pharmaceuticals, Wyeth Pharmaceuticals, Seagen
- Location
- United States of America
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
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 |
