Pre-Pubertal Children with Idiopathic Growth Hormone Deficiency MedDRA version: 12.1 Level: LLT Classification code 10056438 Term: Growth hormone deficiency
Conditions
Interventions
Trade Name: NutropinAq 10 mg/2 ml
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Somatropin
CAS Number: 12629-01-5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration typ
Sponsors
Ipsen Pharma
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Treatment with rhGH at a minimum starting dose of 0.025 mg/kg/day (without dose reduction) to a maximum dose of 0.035 mg/kg/day for at least 12 months and a maximum of 18 months based on the diagnosis of IGHD with a pre-treatment GH stimulation test/spontaneous maximum of GH =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Evidence (more than 20% rhGH injections missed) or suspicion of poor compliance during the first year of rhGH treatment • Hypothalamic-pituitary tumours diagnosed or treated prior to screening • Evidence of any active malignancy or intracranial tumours • Co-morbidity known to affect linear growth including, but not limited to, skeletal dysplasia • Chronic illness including but not limited to diabetes, inborn errors of metabolism, osteochondrodystrophy, disorders of genitourinary, cardiopulmonary, gastrointestinal, or central nervous system • Any named syndrome known to be associated with short stature but not limited to Prader-Willi syndrome, Russel Silver syndrome, etc. • Neurological disease and mental disabilities that may interfere with compliance [Attention Deficit Hyperactivity Disorders (ADHD), autism, Asperger] • Ongoing drug treatment known to alter growth, including but not limited to high dose glucocorticoids • Multiple hormonal deficiencies except hypothyroidism if corrected and well controlled • Abnormal findings at previous fundoscopy or echocardiogram • Significant abnormality in clinical screening laboratories, as determined by the Investigator • Syndromes that predispose the subject to cancer (e.g. Fanconi syndrome, Bloom syndrome, ataxia telangectasia) • Active seizure disorders, defined as one or more seizures per month irrespective of anticonvulsant therapy • Known allergy or hypersensitivity to any components of NutropinAq® (somatropin) or Increlex® (mecasermin) • Acute critical illness due to complications following open heart or abdominal surgery, multiple accidental traumas or acute respiratory failure • Any current or previous exposure to therapeutic spinal irradiation • Prior bone marrow transplantation • Evidence of clinical malnutrition or growth deficit attributable to emotional deprivation • Participation in a clinical trial within the last 12 weeks • Any social or medical condition that, in the opinion of the Investigator, would be detrimental to either the subject or the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate a superior efficacy on height velocity (HV) of treatment with a combination of flexible rhIGF-1 dose and 0.035 mg/kg/day of rhGH, as compared to treatment with 0.035 mg/kg/day of rhGH only, after one year of treatment in pre-pubertal children with IGHD, showing a poor response to rhGH treatment during the first treatment year.;Secondary Objective: • To assess: - effect on HV, delta height SDS, HV SDS of flexible rhIGF-1 dose and fixed rhGH dose combination therapy vs. fixed rhGH dose therapy - safety of flexible rhIGF-1 dose and fixed rhGH dose combination vs. fixed rhGH dose therapy - effect on serum concentrations of GH and IGF-1 and their binding proteins during flexible rhIGF-1 dose and fixed rhGH dose combination therapy vs. fixed rhGH dose therapy - effect on serum concentrations of IGF-1 SDS during flexible rhIGF-1 dose and fixed rhGH dose combination therapy vs. fixed rhGH dose therapy at each visit - effect on pubertal status - change in bone age • To identify prognostic factors of therapeutic response to flexible rhIGF-1 dose and fixed rhGH combination therapy, based on historical data (pre rhGH treatment and at study start), on GH stimulation tests, IGF-1, auxology and diagnosis • To assess changes in metabolic parameters;Primary end point(s): Difference in mean HV between the two treatment groups after 1 year of treatment. A difference of = 1.5 cm/year is considered to be clinically relevant. | — |
Countries
Finland, Spain, Sweden, United Kingdom
Outcome results
None listed