Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader Willi Syndrome - Full Text View

Purpose

This study is conducted in Europe.

The aim of this observational study is to collect data from children with Prader Willi Syndrome, who have been treated off-label with Norditropin® for more than 12 months to seek approval for Norditropin treatment with Prader Willi Syndrome

Condition	Intervention
Prader-Willi Syndrome	Drug: No treatment given

Genetics Home Reference related topics:

Prader-Willi syndrome

MedlinePlus related topics:

Prader-Willi Syndrome

ChemIDplus related topics:

Somatotropin Somatropin

U.S. FDA Resources

Study Type:	Observational
Study Design:	Other, Retrospective

Official Title:	Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader Willi Syndrome (PWS)

Further study details as provided by Novo Nordisk:

Primary Outcome Measures:

Primary objective is to investigate changes in height Standard Deviation Score (SDS) [ Time Frame: in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Changes in height SDS from start of treatment to last observation during Norditropin treatment (referenced to PWS population1) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Change in body composition (DEXA, Bio impedance or stable isotope dilution) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Height velocity (HV) and change in HV [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Glycated Fraction of Haemoglobin (HbA1c) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Insulin-Like Growth Factor-I (IGF-I) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Haematology [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Thyroid-stimulating hormone (TSH) and active form of free thyroxin [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]
Adverse Events. [ Time Frame: at 12 months- and at last observation during Norditropin treatment ] [ Designated as safety issue: No ]

Biospecimen Retention: None Retained

Biospecimen Description:

Estimated Enrollment:	34
Study Start Date:	March 2008
Estimated Study Completion Date:	July 2008
Estimated Primary Completion Date:	July 2008 (Final data collection date for primary outcome measure)

Groups/Cohorts	Assigned Interventions
A	Drug: No treatment given Prader-Willi syndrome children treated with at least one dose of Norditropin®

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Children with Prader Willi Syndrome

Criteria

Inclusion Criteria:

Informed consent obtained before any trial-related activities
Genetically diagnosed Prader Willi Syndrome
Received at least one dose of Norditropin® treatment
Pre-pubertal at start of treatment; assessed by Tanner stage 1, or

Exclusion Criteria:

Pre-treatment with other Growth Hormone preparation prior to treatment with Norditropin®

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00705172

Contacts


Contact: Public Access to Clinical Trials - Novo Nordisk	Please Contact NN via email	clinicaltrials@novonordisk.com

Locations


Denmark
		Recruiting
	Arhus N, Denmark, 8200

Germany
		Not yet recruiting
	Hildesheim, Germany, 31134

Switzerland
		Not yet recruiting
	Zürich, Switzerland, 8006

Sponsors and Collaborators

Novo Nordisk

Investigators


Study Director:	Hanne Jørgensen, Msc Pharm	Novo Nordisk A/S

More Information

Clinical Trials at Novo Nordisk This link exits the ClinicalTrials.gov site

Responsible Party:	Novo Nordisk A/S ( Public Access to Clinical Trials )
Study ID Numbers:	GHLIQUID-1961
First Received:	June 24, 2008
Last Updated:	June 24, 2008
ClinicalTrials.gov Identifier:	NCT00705172
Health Authority:	Germany: Ethics Committee

Study placed in the following topic categories:

Chromosomal abnormalities

Obesity

Chromosome Disorders

Prader-Willi syndrome

Mental Retardation

Genetic Diseases, Inborn

Abnormalities, Multiple

Neurologic Manifestations

Nutrition Disorders

Overnutrition

Prader-Willi Syndrome

Congenital Abnormalities

Neurobehavioral Manifestations

Additional relevant MeSH terms:

Pathologic Processes

Disease

Syndrome

Nervous System Diseases

ClinicalTrials.gov processed this record on September 23, 2008

Sponsored by:	Novo Nordisk
Information provided by:	Novo Nordisk
ClinicalTrials.gov Identifier:	NCT00705172