Clinical trial NCT00705172: Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome
Official title: Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Genetic Disorder
Prader-Willi Syndrome
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2008-06-25
Enrollment: 41
Sex: ALL
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with Prader-Willi Syndrome

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.


Eligibility 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®

Primary outcomes:
measure: Primary objective is to investigate changes in height Standard Deviation Score (SDS)
timeFrame: in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population
Secondary outcomes:
measure: Changes in height SDS from start of treatment to last observation during Norditropin treatment (referenced to PWS population1)
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Change in body composition (DEXA, Bio impedance or stable isotope dilution)
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Height velocity (HV) and change in HV
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Glycated Fraction of Haemoglobin (HbA1c)
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Insulin-Like Growth Factor-I (IGF-I)
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Haematology
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Thyroid-stimulating hormone (TSH) and active form of free thyroxin
timeFrame: at 12 months- and at last observation during Norditropin treatment
measure: Adverse Events.
timeFrame: at 12 months- and at last observation during Norditropin treatment
Other outcomes:
Locations:
city: Arhus N
country: Denmark
city: Hildesheim
country: Germany
city: Zürich
country: Switzerland
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Meinhardt U, Christiansen JS, Farholt S, Lammer C, Ostergaard JR, Schmidt F, Kappelgaard AM, Eiholzer U. The efficacy and safety of long-term Norditropin(R) treatment in children with Prader-Willi syndrome. Horm Metab Res. 2013 Jul;45(7):532-6. doi: 10.1055/s-0033-1343449. Epub 2013 Apr 30.