Pediatric growth hormon deficiency
Conditions
Interventions
Lonapegsomatropin will be administered as once weekly subcutaneous (SC) injections of 0.24 mg hGH/kg/week using GH Auto-Injector.
Genotropin will be administered as daily SC injections in a standard d
Sponsors
Ikle Jennifer
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Main period 1) Prepubertal Japanese children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 aged (at randomization): Boys: 3 - 12 years, inclusive Girls: 3 - 11 years, inclusive 2) Impaired HT: A. Defined as at least 2.0 standard deviation score (SDS) below the mean HT for chronological age and sex (HT SDS -2.0, who have benign, organic, intracranial lesions (such as Rathke's cleft cyst) causing deficiency of at least one pituitary hormone in addition to GH, if a. the growth velocity is 1) Children who have completed main period 2) Children who have not permanently discontinued investigational product in the main period 3) Written, signed informed consent of
Exclusion criteria
Exclusion criteria: Main period 1) Children with a weight below 5.5 kg 2) Prior exposure to recombinant hGH or IGF-1 therapy 3) Children with a history of benign intracranial tumors unless there is documentation(within 6 Month(M) prior to Screening)to confirm no growth within the past 2 years. Children with non-malignant meningiomas are not eligible 4) Children born small for gestational age(i.e., birth weight = 8.0%)or diabetic complications 12) Known chromosomal abnormalities and other named medical syndromes known to impact growth(e.g., Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias)with the exception of septo optic dysplasia 13) Closed epiphyses 14) Tanner stage > 1 (scant pubic hair alone does not exclude the subject) 15) Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and including methylphenidate and other stimulant medications used for ADHD. With the exception of hormone replacement therapies(e.g., levothyroxine or hydrocortisone when used for hormone replacement). Medication for ADHD will be allowed for subjects to start after enrollment. Glucocorticoids not given for hormone replacement therapy are discussed separately below. 16) Children requiring systemic glucocorticoids for reasons other than as hormone replacement therapy, inhaled, or mid- to high-potency topical glucocorticoids as follows: A. Children requiring inhaled glucocorticoid therapy(e.g., asthma)who used a dose of greater than 400 microgram/d of inhaled budesonide or equivalent for more than 28 days cumulatively over the course of the 12M prior to screening(or who will likely require treatment with more than 400 microgram/d of inhaled budesonide or equivalent for more than 28 days cumulatively in a calendar year during the trial). Chronic use of lower doses of inhaled glucocorticoids is not a reason for exclusion. (Note: Approximately equivalent doses to budesonide DPI 400 microgram/d: beclomethasone[HFA; extra fine particles], 240 microgram/d; beclomethasone[HFA, standard particles], 400 microgram/day; ciclesonide[HFA], 240 microgram/d; flunisolide[HFA], 240 microgram/d; fluticasone propionate[HFA], 220 microgram/d; fluticasone propionate
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized Height Velocity at 52 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| - Annualized HV for the lonapegsomatropin and the daily Genotropin over 52 weeks - Change in HT SDS from baseline over 52 weeks for the lonapegsomatropin and the daily Genotropin - Incidence of AEs - Local tolerability (assessed by the subjects, the parents/legally acceptable representatives and the investigator) - Incidence of anti-hGH antibodies including neutralizing antibodies as needed (both cohorts) and incidence of anti-PEG and anti lonapegsomatropin antibody formation (in lonapegsomatropin subjects) - Parameters of glucose metabolism [fasting glucose and hemoglobin A1c (HbA1c)] and lipid parameters - Hormone levels: thyroid status and morning cortisol - All other hematology and biochemistry blood parameters - Electrocardiogram (ECG) at Visit 5 (main period) near time to maximum concentration (Tmax) of hGH - Results of the physical examinations, vital sign measurements - Bone age at 52 weeks - Serum hGH levels - Serum lonapegsomatropin, mPEG levels (in lonapegsomatropin subjects) - Serum IGF-1, IGFBP-3 levels, IGF-1 SDS and IGFBP-3 SDS - Proportion of subjects with IGF-1 SDS level 0-2 Data collected in the extension period will support the following secondary efficacy, safety, and PK/PD endpoints. - Annualized HV over 156 weeks - Change in HT SDS from baseline over 156 weeks - Incidence of AEs - Local tolerability (assessed by the subjects, the parents/legally acceptable representatives and the investigator) - Incidence of anti-hGH antibodies including neutralizing antibodies as needed and incidence of anti-PEG and anti-lonapegsomatropin antibody formation - Parameters of glucose metabolism (HbA1c) and lipid parameters - Hormone levels: thyroid status and morning cortisol - All other hematology and biochemistry blood parameters - Electrocardiogram (ECG) - Results of the physical examinations, vital sign measurements - Bone age at 156 weeks - Serum IGF-1, IGFBP-3 levels, IGF-1 SDS and IGFBP-3 SDS - Proportion of subjects with IGF-1 SDS level 0-2 - Serum mP | — |
Contacts
Public ContactClinical contact
ICON Clinical Research GK
Outcome results
None listed