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:	Time Perspective: 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: 17q21.31 microdeletion syndrome tetrasomy 18p

MedlinePlus related topics: Diabetes Medicines Prader-Willi Syndrome

Drug Information available for: Somatropin

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 ]

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

Additional relevant MeSH terms:

Prader-Willi Syndrome
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple

Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders

ClinicalTrials.gov processed this record on May 23, 2012