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

October 31, 2023 updated by: Novo Nordisk A/S

Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

41

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 15 years (Child)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Children with Prader-Willi Syndrome

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
in response to 12 months Norditropin® treatment in children with PWS (referenced to PWS population

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novo Nordisk A/S

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2008

Primary Completion (Actual)

November 1, 2008

Study Completion (Actual)

November 1, 2008

Study Registration Dates

First Submitted

June 24, 2008

First Submitted That Met QC Criteria

June 24, 2008

First Posted (Estimated)

June 25, 2008

Study Record Updates

Last Update Posted (Actual)

November 2, 2023

Last Update Submitted That Met QC Criteria

October 31, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GHLIQUID-1961

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe