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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT00705172
First received: June 24, 2008
Last updated: April 14, 2014
Last verified: April 2014
  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
Genetic Disorder
Prader-Willi Syndrome
Drug: somatropin

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:


Further study details as provided by Novo Nordisk A/S:

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: somatropin
Prader-Willi syndrome children treated with at least one dose of Norditropin®
Other Name: Norditropin®

  Eligibility

Ages Eligible for Study:   up to 15 Years
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 testicular volume below 4ml (according to Tanner 1976)

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 A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
No publications provided by Novo Nordisk A/S

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: 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: April 14, 2014
Health Authority: Germany: Ethics Committee

Additional relevant MeSH terms:
Genetic Diseases, Inborn
Prader-Willi Syndrome
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Obesity
Overnutrition
Nutrition Disorders

ClinicalTrials.gov processed this record on April 17, 2014