Versartis Trial in Children to Assess Long-Acting Growth Hormone

Phase 1

Completed

• Conditions: Pediatric Growth Hormone Deficiency

• Registration Number: NCT01718041
• Lead Sponsor: Versartis Inc.

Brief Summary

This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. During the Phase 2a stage, patients will receive 6 months of VRS-317 treatment at dose levels selected from the Phase 1b stage. The primary endpoints for the study are to determine the safety and efficacy of repeat dose VRS-317.

Detailed Description

In Phase 1b, separate cohorts of patients will be tested in a single ascending dose format. Safety review committee meetings will take place prior to escalating to each increasing dose level. Enrolled patients will be monitored for 60 days for safety with PK/PD and safety labs collected.

Two dose levels will be selected after completion of Phase 1b to be tested during Phase 2a (6 months of continuous VRS-317 treatment). Safety and PK assessments will be made during the Phase 2a stage. Patient heights will be measured by stadiometer.

Study Timeline

• Study Start: 2012-10
• Study First Submitted: 2012-10-19
• Study First Submitted QC: 2012-10-26
• Study First Posted: 2012-10-31
• Primary Completion: 2014-07
• Study Completion: 2014-07
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-22
• Last Update Posted: 2022-07-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 64

Inclusion Criteria

• Chronological Age ≥ 3.0 years and ≤ 11.0
• Diagnosis of GHD as documented by GH stimulation test
• Below average height SDS at screening
• Appropriate weight for Stature
• Decreased IGF-I SDS at screening
• Delayed bone age
• Normal thyroid function test results at screening visit
• Legally authorized representative informed consent.

Exclusion Criteria

• Prior treatment with any growth promoting agent
• Documented history of, or current, significant disease
• Chromosomal aneuploidy, significant gene mutations
• Diagnosis of Attention Deficit Hyperactivity Disorder
• Daily use of anti-inflammatory doses of glucocorticoid
• Prior history of leukemia, lymphoma, sarcoma or cancer
• Known allergy to constituents of the study drug formulation
• Abnormal ocular findings at screening
• Significant abnormality in screening laboratory studies

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317	8 Months	Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a).; The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.

Secondary Outcome Measures

Name	Time	Method
Evaluate pharmacokinetic measures of VRS-317.	8 months	Secondary outcome measures include evaluation of pharmacokinetics (PK) of VRS-317 including Cmax, Tmax and AUC. PK measures will be analysed at multiple timepoints after single dosing of VRS-317 during Phase 1b and after repeat dosing of VRS-317 during Phase 2a.