Growth Hormone Deficiency in adults MedDRA version: 18.1 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Name: Somavaratan
Product Code: VRS-317
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Somavaratan
CAS Number: 1448335-08-7
Other descriptive name: VRS-317
Concentration unit
Sponsors
Versartis Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Female or male subjects with age 23 to 70 years (inclusive). 2. Female subjects of childbearing potential must agree to use appropriate contraceptive methods during the study and continue this method for at least one month after their study completion. 3. Female subjects of childbearing potential must have a negative serum pregnancy test at Screening. 4. Subjects must have a documented medical history of GHD during adulthood, according to established guidelines (confirmatory testing should be done to document persistence of childhood onset GHD into adulthood if necessary). Acceptable diagnostic criteria include: - insulin tolerance test (ITT): peak hGH = 5.0 ng/mL - arginine alone: peak hGH = 1.4 ng/mL - arginine + growth-hormone-releasing hormone - glucagon stimulation test: peak hGH = 3.0 ng/mL OR - at least 3 pituitary hormone deficiencies and a low IGF-I for age/gender For arginine + GHRH stimulation test, peak hGH limit is based upon body mass index as follows: = 11.0 ng/mL for BMI =65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1.Subjects with a documented history of diabetes mellitus or inadequate glucose control as defined by a historical or Screening value of: •FPG > 126 mg/dL (7 mM), or •HbA1c = 6.5% 2.Subjects with untreated adrenal insufficiency. Adrenal insufficiency will be screened by morning serum cortisol followed by ACTH stimulation test for subjects without documented history of adrenal insufficiency. 3.Subjects with free thyroxine outside the normal reference range. 4.Subjects currently taking oral glucocorticoids, except for physiological maintenance doses of oral glucocorticoids in subjects with multiple pituitary hormone deficiencies. 5.Subjects with current significant cardiovascular disease, heart insufficiency of NYHA class > 2. 6.Subjects with current significant cerebrovascular, pulmonary, neurological (not considered related to GHD), renal, inflammatory, or hepatobiliary disease. 7.Subjects with current papilledema. 8.Subjects with a history of persistent (unresolved without medical intervention) or recurring migraines. 9.Subjects with current edema (= CTCAE Grade 2). 10.Subjects with current drug or alcohol abuse. 11.Subjects with a documented history of HIV, current HBV or HCV infection (testing not required). 12.Subjects with a prior history of malignancy or abnormal results of any routine cancer screening tests, excluding adequately treated non-melanoma skin cancers or adequately treated in situ carcinoma of the cervix. 13.Women who are pregnant or breastfeeding. 14.Subjects treated with an investigational drug within 30 days prior to Screening. 15.Subjects with a significant abnormality in Screening laboratory results as interpreted by the Investigator and/or the Medical Monitor.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To evaluate the safety of individualized dosing of Somavaratan for up to five months as assessed by review of the accumulated safety data in each treatment cohort •To evaluate the starting doses of Somavaratan for each cohort as measured by the proportion of subjects who achieve normalization of IGF-I SDS response (reference range of -2.00 to 2.00 SDS) during the first dosing interval (one month after the first dose) •To evaluate the dose titration plan of Somavaratan for each cohort as measured by the proportion of subjects who achieve a mean IGF-I SDS (based on pre-dose and 7 days after dosing values) within the defined target range of 0 to 1.50 after each dose titration •To determine whether IGF-I response over the 30 day dosing interval can be determined by using the SDS of the mean of IGF-I values from pre-dose and 7 days after dosing ;Secondary Objective: •To evaluate the immunogenicity of Somavaratan by measurement of serum anti-drug antibody (ADA) titers and detection of neutralizing antibodies (NAb) ;Primary end point(s): 1. IGF-I SDS levels within therapeutic range after 1st dose 2. IGF-I SDS levels within target range following each dose 3. Compare mean IGF-I from 2 values vs mean IGF-I from 4 values ;Timepoint(s) of evaluation of this end point: 1. Predose and Day 7 following 1st dose 2. Predose and Day 7 following each dose 3. Predose and Days 7, 21 and 30 following each dose | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Serum anti-drug antibody titers 2. Serum neutralising antibodies ;Timepoint(s) of evaluation of this end point: 1. Prior to each dose and at end of study 2. Prior to each dose and at end of study | — |
Countries
Australia, Germany, United Kingdom, United States
Contacts
Public ContactJessica Merryfield
Premier Research
Outcome results
None listed