Noonan syndrome associated short stature MedDRA version: 7.1 Level: PT Classification code 10029748
Conditions
Interventions
Trade Name: Norditropin 24 IU
Product Name: Norditropin 24 IU
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Somatropin
Concentration unit: IU international un
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The present inclusion criteria are • Informed consent before any trial related activities • Participation in the original Norditropin® study (S/GHD/004/NOO) or following the protocol for S/GHD/004/NOO without being randomised in the trial The original inclusion criteria were • Verbal informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.) • Children with Noonan Syndrome. The diagnostic criteria will be o Short stature o Hypertelorism o Low set ears o And one of the following criteria: ? Ptosis ? Cubiti valga ? Testicular dysplasia ? Hypogonadotroph hypogonadism • Bone age determination showing no significant acceleration (more than one year above chronological age) according to Greulich and Pyle (14). Patients showing an accelerated bone age (> chronological age) at the first examination will be reevaluated within 6 weeks before treatment start in order to evaluate the development process • Age at start of the treatment Age: 3.00-11.99 years • Prepubertal according to Tanner standards: =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The original exclusion criteria were • Earlier or ongoing treatment with growth hormone, anabolic steroids or corticosteroids • Subjects with congenital heart defect perceived to possibly have a major impact on growth • History of or actual endocrine, metabolic or other serious chronic disease perceived to possibly have a major impact on growth • Fasting blood glucose consistently above 6.7 mmol/l • Growth hormone deficiency defined as o GH levels below 10 µg/l during two consecutive GH stimulation tests
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the effect of Norditropin® on final height in children with Noonan syndrome;Secondary Objective: -;Primary end point(s): Primary Endpoint: Change in height SDS from start of treatment to final height (referenced to normal population) Secondary Endpoints: Efficacy • Final height SDS (referenced to normal population and Noonan population, respectively) • Change in height SDS from start of treatment to final height (referenced to Noonan population) • Height velocity • Change in height velocity from start of treatment • Sitting height • Number and proportion of subjects with final height SDS above -2 SDS Safety • Adverse events (AEs) • HbA1c, Free Thyroxin and TSH • Fasting blood glucose and insulin • Haematology, serum biochemistry and urinalysis laboratory variables • Physical examinations, BP and pulse • Bone age • ECG • Echocardiography • IGF-I, and IGFBP-3 levels and SDS • PqCT for assessment of bone mineral status • DXA scan for assessment of body composition and bone mineral status • Plasma leptin, ghrelin and adiponectin for assessment of these growth hormone dependent hormones | — |
Countries
Sweden
Outcome results
None listed