Growth hormone deficiency in pre-pubertal children MedDRA version: 19.1 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Code: MOD-4023
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Somatrogon
CAS Number: 1663481-09-1
Current Sponsor code: MOD-4023
Concentration unit: mg/ml milligram(s)/millil
Sponsors
OPKO Biologics Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Pre-pubertal children aged =3 years , and not yet 11 years for girls (10 years and 364 days) or not yet 12 years (11 years and 364 days) for boys, (on the date of ICF signature), with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency. 2. Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of =10 ng/mL, determined by local or central laboratory using a validated assay . Global Medical Monitor may accept prior local laboratory results; subject to pre-approval and if the tests were conducted as recommended in the protocol 3. Bone age (BA) is not older than chronological age and should be less than 10 for girls and less than 11 for boys. 4. Without prior exposure to any rhGH therapy (naïve patients). 5. Impaired height velocity defined as: • Annualized height velocity (HV) below the 25th percentile for CA (HV =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Children with prior history of leukemia, lymphoma, sarcoma or any other forms of cancer. 2. History of radiation therapy or chemotherapy 3. Malnourished children defined as BMI < -2 SDS for age and sex 4. Children with psychosocial dwarfism 5. Children born small for gestational age (SGA – birth weight and/or birth length <-2 SDS for gestational age) 6. Presence of anti-hGH antibodies at screening 7. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc. 8. Type 1 and type 2 diabetic patients who, in the opinion of the investigator, are not receiving standard of care treatment, or are non-compliant with their prescribed treatment or who are in poor metabolic control 9. Chromosomal abnormalities including Turner’s syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias. 10. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids, with the exception of ADHD drugs or hormone replacement therapies (thyroxin, hydrocortisone, desmopressin [DDAVP]) 11. Children requiring glucocorticoid therapy (e.g. for asthma) that are taking chronically a dose greater than 400 µg/d of inhaled budesonide or equivalent 12. Major medical conditions and/or presence of contraindication to r-hGH treatment. 13. Closed epiphyses 14. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis. 15. Drug, substance, or alcohol abuse. 16. Known hypersensitivity to the components of study medication. 17. Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets. 18. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct. Participation in any other trial of an investigational agent within 30 days prior to ICF signature (including administration of investigational agent).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that weekly MOD-4023 administration is non-inferior to daily Genotropin administration in terms of safety and efficacy outcomes;Secondary Objective: To evaluate the effect of weekly MOD-4023 and daily Genotropin administration on quality of life, as measured by the QoLISSY (Quality of Life in Short Stature Youth) at specific number of countries.;Primary end point(s): Annual Height Velocity (HV) in cm/year;Timepoint(s) of evaluation of this end point: Baseline and after 12 months of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints (Auxology/Clinical): • Annualized height velocity after 6 months of treatment • Change in height SDS at 6 and 12 months, compared to baseline • Change in bone maturation (BM) at the end of 12 months, compared to Screening bone age (calculated as BA/CA) Secondary endpoints (Biochemical): • Absolute IGF-I levels on day 4(-1) after MOD-4023 dosing across study visits • IGF-I SDS on day 4(-1) after MOD-4023 dosing across study visits •IGFBP-3 levels and IGFBP-3 SDS on day 4(-1) after MOD-4023 dosing across study visits Additional Endpoints •QoL endpoint measured by the QoLISSY core questionnaire at baseline and month 12 or early termination in specific countries per Appendix L. ;Timepoint(s) of evaluation of this end point: as above | — |
Countries
Argentina, Australia, Belarus, Brazil, Bulgaria, Canada, Chile, Colombia, France, Georgia, Germany, Greece, India, Israel, Italy, Mexico, Netherlands, New Zealand, Poland, Romania, Russian Federation, Serbia, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States, Vietnam
Contacts
Public ContactDirector of Clinical Affairs
OPKO Biologics Ltd.
Outcome results
None listed