Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)

Phase 2

Terminated

• Conditions: Pediatric Growth Hormone Deficiency; Growth Disorders
• Interventions: Drug: Somavaratan (VRS-317)

• Registration Number: NCT02413138
• Lead Sponsor: Versartis Inc.

Brief Summary

The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.

Detailed Description

The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.

Study Timeline

• Study First Submitted: 2015-03-23
• Study First Submitted QC: 2015-04-08
• Study First Posted: 2015-04-09
• Study Start: 2015-08-08
• Primary Completion: 2017-11-30
• Study Completion: 2017-11-30
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-22
• Last Update Posted: 2022-07-25

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 41

Inclusion Criteria

• Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)
• Pre-pubertal status
• Diagnosis of GHD as documented by two or more GH stimulation test results
• Height SD score ≤ -2.0 at screening
• Weight for Stature ≥ 10th percentile
• IGF-I SD score ≤ -1.0 at screening
• Delayed bone age

Exclusion Criteria

• Prior treatment with any growth promoting agent
• History of, or current, significant disease
• Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome
• Birth weight and/or birth length less than 5th percentile for gestational age
• A diagnosis of Attention Deficit Hyperactivity Disorder
• Daily use of anti-inflammatory doses of glucocorticoid
• Prior history of leukemia, lymphoma, sarcoma or cancer
• 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Phase 3: Somavaratan (VRS-317)	Somavaratan (VRS-317)	Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly
Phase 2: Somavaratan (VRS-317)	Somavaratan (VRS-317)	Active treatment arm

Primary Outcome Measures

Name	Time	Method
Efficacy (Annual Height velocity)	12 months	Annual Height velocity.

Secondary Outcome Measures

Name	Time	Method
Safety (Concomitant medications)	12 months	Concomitant medications
Safety (Safety labs)	12 months	Safety labs
Safety (Vital signs)	12 months	Vital signs
Safety (Physical Exams)	12 months	Physical Exams
Safety (Number of subjects with adverse events )	12 months	Number of subjects with adverse events (including repeat dose immunogenicity).
Pharmacodynamics (IGF-I responses to study drug administration)	12 months	IGF-I responses to study drug administration.
Pharmacodynamics (IGFBP-3 responses to study drug administration)	12 months	IGFBP-3 responses to study drug administration.

Trial Locations

Locations (1)

Hokkaido University Hospital; 🇯🇵 Sapporo, Japan