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

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ClinicalTrials.gov Identifier: NCT00705172
Recruitment Status : Completed
First Posted : June 25, 2008
Last Update Posted : December 23, 2014
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
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 or disease Intervention/treatment
Genetic Disorder Prader-Willi Syndrome Drug: somatropin

Study Type : Observational
Actual Enrollment : 41 participants
Time Perspective: Retrospective
Official Title: Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS)
Study Start Date : November 2008
Actual Primary Completion Date : November 2008
Actual Study Completion Date : November 2008

Resource links provided by the National Library of Medicine

Drug Information available for: Somatropin
U.S. FDA Resources

Group/Cohort Intervention/treatment
A Drug: somatropin
Prader-Willi syndrome children treated with at least one dose of Norditropin®
Other Name: Norditropin®



Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Change in body composition (DEXA, Bio impedance or stable isotope dilution) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]
  3. Height velocity (HV) and change in HV [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]
  4. Glycated Fraction of Haemoglobin (HbA1c) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]
  5. Insulin-Like Growth Factor-I (IGF-I) [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]
  6. Haematology [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]
  7. Thyroid-stimulating hormone (TSH) and active form of free thyroxin [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]
  8. Adverse Events. [ Time Frame: at 12 months- and at last observation during Norditropin treatment ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 15 Years   (Child)
Sexes Eligible for Study:   All
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®

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Additional Information:
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
First Posted: June 25, 2008    Key Record Dates
Last Update Posted: December 23, 2014
Last Verified: December 2014

Additional relevant MeSH terms:
Syndrome
Prader-Willi Syndrome
Hyperphagia
Genetic Diseases, Inborn
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Obesity
Overnutrition
Nutrition Disorders
Signs and Symptoms, Digestive
Signs and Symptoms