Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome

• Status: Completed
• Trial end date: 2018-07-12
• Target enrollment: 0
• Participant gender: All

Summary

This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novo Nordisk A/S

Criteria

Inclusion Criteria:

• Japanese children with Noonan syndrome clinically diagnosed in one of the following ways: 1. Clinically diagnosed by at least two medical experts using van der Burgt score list, 2. Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by result of genetic testing for Noonan syndrome, 3. Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by the same medical expert based on the results of centralised evaluation of facial change using van der Burgt score list

• Height SDS (standard deviation score): -2 SDS or below (according to the Japanese reference data)

• Age: boys 3 to below 11 years, girls 3 to below 10 years

• Height records must be available within the period between 40 and 64 weeks prior to Visit 1 (screening)

• Prepubertal children (definition for girls breast and pubes of Tanner stage is I, and none of menses, and for boys testicular volume below 4 mL, and pubes and penis of Tanner stage is I)

Exclusion Criteria:

• Children with known or suspected hypersensitivity against human growth hormone (hGH) or related products (including any components of the trial products)

• Children with diabetic type diagnosed with the Japanese Diabetes Society Classification

• Children with history or presence of active malignancy

• Children who have received GH (growth hormone) treatment

• Children who have received systemic administration of the following medications within two years prior to Visit 1 (screening): Thyroid hormone (except replacement therapy), antithyroid hormone, androgen, oestrogen, progesterone, anabolic steroid, adrenocortical steroid treatment period for at least 13 weeks), derivative of gonadotropin releasing hormone and somatomedin C (IGF-I)