Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome
- Registration Number
- NCT00705172
- Lead Sponsor
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 41
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®
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description A somatropin -
- Primary Outcome Measures
Name Time Method Primary objective is to investigate changes in height Standard Deviation Score (SDS) in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population
- Secondary Outcome Measures
Name Time Method Haematology at 12 months- and at last observation during Norditropin treatment Glycated Fraction of Haemoglobin (HbA1c) at 12 months- and at last observation during Norditropin treatment Changes in height SDS from start of treatment to last observation during Norditropin treatment (referenced to PWS population1) at 12 months- and at last observation during Norditropin treatment Change in body composition (DEXA, Bio impedance or stable isotope dilution) at 12 months- and at last observation during Norditropin treatment Height velocity (HV) and change in HV at 12 months- and at last observation during Norditropin treatment Adverse Events. at 12 months- and at last observation during Norditropin treatment Insulin-Like Growth Factor-I (IGF-I) at 12 months- and at last observation during Norditropin treatment Thyroid-stimulating hormone (TSH) and active form of free thyroxin at 12 months- and at last observation during Norditropin treatment