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

NCT00705172 | Completed

• 1 other identifier: GHLIQUID-1961
• Study Type: observational
• Target: 41
• Locations: 3 countries
• Sites: 3

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.

Conditions

Genetic Disorder; Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• 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

• Glycated Fraction of Haemoglobin (HbA1c)

  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

Study Arms (1)

A

Drug: somatropin

Interventions

somatropin DRUG

Prader-Willi syndrome children treated with at least one dose of Norditropin®

Also known as: Norditropin®

A

Eligibility Criteria

• Age: Up to 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Children with Prader-Willi Syndrome

You may qualify if:

• 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)

You may not qualify if:

• Pre-treatment with other Growth Hormone preparation prior to treatment with Norditropin®

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (3)

Unknown Facility

Arhus N, 8200, Denmark

Location

Unknown Facility

Hildesheim, 31134, Germany

Location

Unknown Facility

Zurich, 8006, Switzerland

Location

Related Publications (1)

• 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.

  PMID: 23632904 DERIVED

Related Links

• Clinical Trials at Novo Nordisk

MeSH Terms

Conditions

Genetic Diseases, Inborn; Prader-Willi Syndrome

Interventions

Human Growth Hormone

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Global Clinical Registry (GCR, 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 24, 2008
• First Posted: June 25, 2008
• Study Start: November 1, 2008
• Primary Completion: November 1, 2008
• Study Completion: November 1, 2008
• Last Updated: November 2, 2023

Record last verified: 2023-10

Locations

CH (1); DK (1); DE (1)