Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency

14VR4

The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.

2015-08-26

2017-08-23

2017-08-23

Completed

Early phase I

138

Inclusion Criteria: * Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys). * Pre-pubertal status: Absent breast development in girls, testicular volume \< 4.0 mL in boys. * Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL. * Height standard deviation score (SDS) ≤ -2.0 at screening. * Weight for Stature ≥ 10th percentile. * Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening. * Delayed bone age (≥ 6 months). Exclusion Criteria: * Prior treatment with any growth promoting agent * History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency). * Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome. * A diagnosis of Attention Deficit Hyperactivity Disorder. * Daily use of anti-inflammatory doses of glucocorticoid. * Prior history of leukemia, lymphoma, sarcoma or cancer. * Treatment with an investigational drug in the 30 days prior to screening. * Known allergy to constituents of the study drug formulation. * Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening. * Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants. * Significant abnormality in screening laboratory studies

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 138, 'type': 'ACTUAL'}}

2022-12-30