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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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:
Prader-Willi Syndrome
Syndrome
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Disease
Pathologic Processes

ClinicalTrials.gov processed this record on October 19, 2014