Adult Growth Hormone Deficiency
Conditions
Keywords
VRS-317, Long Acting Growth Hormone, Versartis, somavaratan, Growth Hormone Deficiency, Adult Growth Hormone Deficiency, AGHD, Growth Hormone Replacement, Growth Hormone Replacement Therapy, XTEN
Brief summary
A Phase 2, open-label dose-finding safety study of individualized monthly VRS-317 dosing for five months in adults with GHD.
Detailed description
A Phase 2, dose finding safety study to evaluate an individualized monthly VRS-317 dosing regimen in adults with GHD. This is an open-label, international, multicenter study with VRS-317 treatment for five months. This treatment period will include monthly dose titrations until a subject's mean IGF-I SDS value is within a target range for two consecutive months. Subjects will be stratified into three cohorts based on sensitivity to rhGH.
Interventions
DRUGsomavaratan
Long-acting recombinant human growth hormone therapy administered subcutaneously once monthly
Sponsors
Premier Research
Versartis Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
23 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Female subjects of childbearing potential must have negative pregnancy test and use appropriate contraceptive methods * Subjects must have documented GHD during adulthood. * Subjects taking other hormone replacement therapy must have been on a stable course of treatment for at least 3 months. * Subjects with underlying disorders responsible for the subject's GHD must have been clinically stable for at least 6 months. * Subjects receiving daily rhGH injections must washout for 14 days. * Subjects must provide signed informed consent. * Subjects must have a BMI (kg/m2) between 19.0 and 35.0.
Exclusion criteria
* Subjects with diabetes mellitus or inadequate glucose control * Subjects with untreated adrenal insufficiency. * Subjects with free thyroxine outside the normal reference range. * Subjects currently taking oral glucocorticoids, except for physiological maintenance doses of oral glucocorticoids in subjects with multiple pituitary hormone deficiencies. * Subjects with current significant cardiovascular disease, heart insufficiency of NYHA class \> 2. * Subjects with current significant cerebrovascular, pulmonary, neurological, renal, inflammatory, or hepatobiliary disease. * Subjects with current papilledema. * Subjects with a history of persistent or recurring migraines. * Subjects with current edema (≥ CTCAE Grade 2). * Subjects with current drug or alcohol abuse. * Subjects with a documented history of HIV, current HBV or HCV infection * Subjects with a prior history of malignancy excluding adequately treated non-melanoma skin cancers or in situ carcinoma of the cervix. * Women who are pregnant or breastfeeding. * Subjects with a significant abnormality in Screening laboratory results
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety as measured by the number of subjects with adverse events, concomitant medications, safety labs, vital signs and physical exams | 5 months | Safety observations include adverse events, concomitant medications, safety labs, vital signs and physical exams. |
| Starting doses (proportion of subjects who achieve normalization of IGF-I SDS response during the first dosing interval ) | 5 months | To evaluate the starting doses of VRS-317 for each cohort as measured by the proportion of subjects who achieve normalization of IGF-I SDS response during the first dosing interval (one month after the first dose) |
| Dose titration plan (proportion of subjects who achieve a mean IGF-I SDS within the defined target range after each dose titration) | 5 months | To evaluate the dose titration plan of VRS-317 for each cohort as measured by the proportion of subjects who achieve a mean IGF-I SDS within the defined target range after each dose titration |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Immunogenicity of VRS-317 by measurement of serum anti-drug antibody (ADA) titers | 5 months | To evaluate the immunogenicity of VRS-317 by measurement of serum anti-drug antibody (ADA) titers |
| Immunogenicity of VRS-317 by detection of neutralizing antibodies (NAbs) | 5 months | To evaluate the immunogenicity of VRS-317 by detection of neutralizing antibodies (NAb) |
Countries
Australia, Germany, United Kingdom, United States
Outcome results
None listed