Health Condition 1: E20-E35- Disorders of other endocrine glands
Conditions
Interventions
Intervention1: Somapacitan
: Somapacitan will be administered subcutaneously (s.c.
under the skin) once weekly by PDS290 pen-injector. Somapacitan can be injected any time during the once weekly dosing day. The dose will be calculated based on the subjects current body weight.
Cont
Sponsors
Novo Nordisk AS
Eligibility
Inclusion criteria
Inclusion criteria: Subjects are eligible to be included in the trial only if all of the following criteria apply: 1. Informed consent of parent or legally acceptable representative of subject and child assent, as age-appropriate must be obtained before any trial-related activities a) The parent or legally acceptable representative of the child must sign and date the Informed Consent Form (according to local requirements) b) The child must sign and date the Child Assent Form or provide oral assent (if required according to local requirements) 2. Prepubertal children: a) Boys: o Age = 2 years and 26 weeks and o Testis volume b) Girls: o Age = 2 years and 26 weeks and o Tanner stage 1 for breast development (no palpable glandular breast tissue)16 3. Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of = 10.0 ng/ml using the WHO International Somatropin 98/574 standard a) If only one growth hormone stimulation test is available before screening, then confirmation of growth hormone deficiency by second and different growth hormone stimulation test must be done b) For children with at least 2 additional pituitary hormone deficiencies (other than growth hormone deficiency) only one growth hormone stimulation test is needed For Japan: see Appendix 9 4. Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention17 5. Impaired height velocity, defined as annualised height velocity below the 25th percentile for chronological age and gender according to the standards of Prader18 calculated over a time span of minimum 6 months and maximum 18 months prior to screening 6. No prior exposure to growth hormone therapy or IGF-I treatment 7. Bone age less than chronological age at screening 8. Body Mass Index >5th and Prevention17, Body Mass Index-for-age growth charts. 9. IGF-I central laboratory 10. Hormone replacement therapies for any other hormone deficiency should be adequate and stable for at least 90 days prior to randomisation 11. No intracranial tumour confirmed by magnetic resonance imaging or computer tomography scan. An image or scan taken within 9 months prior to screening can be used as screening data if the medical evaluation and conclusion is available.
Exclusion criteria
Exclusion criteria: Subjects are excluded from the trial if any of the following criteria apply: 1. Known or suspected hypersensitivity to trial product(s) or related products 2. Previous participation in this trial. Participation is defined as randomisation 3. Receipt of any investigational medicinal product within 3 months before screening or participation in another clinical trial at time of randomisation 4. Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements: a) Turner Syndrome (including mosaicisms) b) Chromosomal aneuploidy and significant gene mutations causing medical “syndromes ? with short stature, including but not limited to Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors c) Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants d) Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome or skeletal dysplasias e) Family history of skeletal dysplasia 5. Children born small for gestational age (birth weight and/or birth length gestational age according to national standards) 6. Children diagnosed with diabetes mellitus or screening values from central laboratory of a) fasting plasma glucose =126 mg/dl (7.0 mmol/L) or b) HbA1c = 6.5 % 7. Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening 8. Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 µg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening 9. Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD) 10. Diagnosis of attention deficit hyperactivity disorder 11. Prior history or presence of malignancy including intracranial tumours 12. Prior history or known presence of active Hepatitis B or Hepatitis C (exceptions to this exclusion criterion is the presence of antibodies due to vaccination against Hepatitis B) 13. Any disorder which, in the opinion of the investigator, might jeopardise subject’s safety or compliance with the protocol For France, Spain, and UK: see Appendix 9 14. The subject or the parent/legally acceptable representative is likely to be non-compliant in respect to trial conduct, as judged by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Height velocityTimepoint: 52 weeks visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in bone ageTimepoint: Week 52;Change in height standard deviation scoreTimepoint: Week 52;Change in height velocity standard deviation scoreTimepoint: Week 52;Change in fasting plasma glucoseTimepoint: Week 52, 104, 156 and 208;Change in homeostatic model assessmentTimepoint: Week 52, 104, 156 and 208;Change in glycated haemoglobinTimepoint: Week 52, 104, 156 and 208;Change in insulin-like growth factor-ITimepoint: Week 52, 104, 156 and 208;Change in insulin-like growth factor binding protein 3Timepoint: Week 52, 104, 156 and 208 | — |
Countries
Algeria, Austria, Canada, Denmark, France, Germany, India, Israel, Italy, Japan, Republic of Korea, Russian Federation, Slovenia, Switzerland, Thailand, Ukraine, United Kingdom, United States of America
Contacts
Public ContactDr Maya Sharma
Novo Nordisk India Private Ltd.
Outcome results
None listed