Condition category
Nutritional, Metabolic, Endocrine
Date applied
28/04/2006
Date assigned
28/04/2006
Last edited
05/11/2012
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

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 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

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

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

Prader-Willi syndrome

Intervention

Treatment with GH: GenotropinĀ® 1 mg/m^2/day subcutaneously (sc) versus no GH-treatment. Dietary and exercise advice.

Intervention type

Drug

Phase

Not Applicable

Drug names

GenotropinĀ®

Primary outcome measures

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 trial start date

23/04/2002

Overall trial end date

01/05/2007

Reason abandoned

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

Patient

Age group

Child

Gender

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

Trial participating centre

Dutch Growth Foundation
Rotterdam
3016 AH
Netherlands

Sponsor information

Organisation

Dutch Growth Foundation (Netherlands)

Sponsor details

Westzeedijk 106
Rotterdam
3016 AH
Netherlands

Sponsor type

Charity

Website

Funders

Funder type

Industry

Funder name

Pfizer (Netherlands)

Alternative name(s)

Pfizer Inc.

Funding Body Type

private sector organisation

Funding Body Subtype

corporate

Location

United States of America

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

1. 2009 results on effect of GH-treatment on incidence of scoliosis in http://www.ncbi.nlm.nih.gov/pubmed/19158197
2. 2009 results on effect of GH-treatment on bone density in http://www.ncbi.nlm.nih.gov/pubmed/19622627
3. 2012 ovarian function results in http://www.ncbi.nlm.nih.gov/pubmed/22723315

Publication citations

  1. Ovarian function results

    Siemensma EP, van Alfen-van der Velden AA, Otten BJ, Laven JS, Hokken-Koelega AC, Ovarian function and reproductive hormone levels in girls with Prader-Willi syndrome: a longitudinal study., J. Clin. Endocrinol. Metab., 2012, 97, 9, E1766-73, doi: 10.1210/jc.2012-1595.

  2. de Lind van Wijngaarden RF, de Klerk LW, Festen DA, Duivenvoorden HJ, Otten BJ, Hokken-Koelega AC, Randomized controlled trial to investigate the effects of growth hormone treatment on scoliosis in children with Prader-Willi syndrome., J. Clin. Endocrinol. Metab., 2009, 94, 4, 1274-1280, doi: 10.1210/jc.2008-1844.

  3. de Lind van Wijngaarden RF, Festen DA, Otten BJ, van Mil EG, Rotteveel J, Odink RJ, van Leeuwen M, Haring DA, Bocca G, Mieke Houdijk EC, Hokken-Koelega AC, Bone mineral density and effects of growth hormone treatment in prepubertal children with Prader-Willi syndrome: a randomized controlled trial., J. Clin. Endocrinol. Metab., 2009, 94, 10, 3763-3771, doi: 10.1210/jc.2009-0270.

Additional files

Editorial Notes