Clinical Trial: VRS-317 in Adult Subjects With Growth Hormone Deficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Blinded Placebo Controlled Single Ascending Dose Phase 1 for Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics After Subcutaneous Administration of VRS-317 in Adults With The purpose of this research study is to determine the safety and tolerability of up to five doses of VRS-317 in Adult Growth Hormone Deficient patients.

• Patients will be evaluated for evidence of activity of VRS-317 by measurement of changes from baseline in insulin-like growth factor-1 (IGF-I) and binding protein (IGFBP-3), and bone turnover (bone alkaline phosphatase)
• Descriptive pharmacokinetic (PK) and pharmacodynamic (PD) parameters (IGF-I and IGFBP-3) will be determined by standard model independent methods based on the plasma concentration-time data of each subject. These parameters include: Cmax, Tmax, AUCavg, AUC0-inf, and t1/2.
• The purpose is to determine the appropriate dose of VRS-317 to maintain a normal range (for appropriate age/gender) for IGF-I levels in adult patients for up to one month after administration of a single dose

Detailed Summary:

The study is a placebo controlled single ascending dose (SAD) study in adult GHD patients currently receiving daily rhGH therapy. After screening patients are withdrawn from daily rhGH therapy for a minimum of 7 days (maximum of 60 days) prior to randomization for treatment. Patients will be randomised within a treatment group that is currently being enrolled once the patient has passed the pre-dose screening criteria.

Documented confirmation (medical history) of GHD during adulthood by a minimum of one or more GH stimulation tests is required such as:

• insulin tolerance test (ITT; peak hGH ≤ 5.0 ng/mL),
• arginine alone (peak hGH ≤ 0.4 ng/mL);
• arginine + growth-hormone-releasing hormone* (see below);
• or glucagon stimulation test (peak hGH ≤ 3.0 ng/mL) OR
• at least 3 other pituitary hormone deficiencies and a low IGF-I for age/gender appropriate normal range

Each patient will be randomised to receive either the investigational product, VRS-317 (Cohorts A-E), or placebo (Cohort F) in a 4:1 ratio.

Subjects will be monitored for safety throughout their participation in the study. To ensure patient safety, two patients (1 active, 1 placebo) in the first treatment group, one from Cohort A and one from Cohort F1, will be dosed in a blinded manner and monitored for 48 hr prior to dosing the remaining 8 patients. The 8 remaining patients will be blinded and randomized to the first treatment group. Vital signs, clinical lab values, adverse events (AEs) and concomitant medications (CMs) will be captured. AEs will be graded using the Common Termi
Sponsor: Versartis Inc.

Current Primary Outcome: Safety and Tolerability of single dose of VRS-317 [ Time Frame: 30 days ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Determine the pharmacokinetic (PK) profile of VRS-317 administered SC [ Time Frame: 30 days ]

Original Secondary Outcome: Same as current

Information By: Versartis Inc.

Dates:
Date Received: May 13, 2011
Date Started: March 2011
Date Completion:
Last Updated: July 19, 2012
Last Verified: July 2012