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

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

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

Study Type:	Observational
Study Design:	Retrospective
Official Title:	Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader Willi Syndrome (PWS)

Resource links provided by NLM:

Genetics Home Reference related topics: Prader-Willi syndrome

MedlinePlus related topics: Prader-Willi Syndrome

Drug Information available for: Somatropin Somatotropin

U.S. FDA Resources

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:

Enrollment:	41
Study Start Date:	March 2008
Study Completion Date:	November 2008
Primary Completion Date:	November 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

Locations

Denmark

Arhus N, Denmark, 8200
Germany

Hildesheim, Germany, 31134
Switzerland

Zürich, Switzerland, 8006

Sponsors and Collaborators

Novo Nordisk

Investigators

Study Director:

Hanne Jørgensen, Msc Pharm

Novo Nordisk

More Information

Additional Information:

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

No publications provided

Responsible Party:	Novo Nordisk A/S ( Public Access to Clinical Trials )
Study ID Numbers:	GHLIQUID-1961
Study First Received:	June 24, 2008
Last Updated:	January 30, 2009
ClinicalTrials.gov Identifier:	NCT00705172 History of Changes
Health Authority:	Germany: Ethics Committee

Additional relevant MeSH terms:

Obesity
Disease
Nervous System Diseases
Chromosome Disorders
Mental Retardation
Pathologic Processes
Genetic Diseases, Inborn
Syndrome

Abnormalities, Multiple
Neurologic Manifestations
Nutrition Disorders
Overnutrition
Prader-Willi Syndrome
Congenital Abnormalities
Neurobehavioral Manifestations

ClinicalTrials.gov processed this record on February 09, 2010