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

NCT ID: NCT02413138

Last Updated: 2022-07-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 41 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-08-08
• Study Completion Date: 2017-11-30

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.

Conditions

Pediatric Growth Hormone Deficiency; Growth Disorders

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Phase 2: Somavaratan (VRS-317)

Active treatment arm

Group Type: EXPERIMENTAL

Somavaratan (VRS-317)

Intervention Type: DRUG

Long acting recombinant human growth hormone

Phase 3: Somavaratan (VRS-317)

Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly

Group Type: EXPERIMENTAL

Somavaratan (VRS-317)

Intervention Type: DRUG

Long acting recombinant human growth hormone

Interventions

Somavaratan (VRS-317)

Long acting recombinant human growth hormone

Intervention Type: DRUG

Other Intervention Names

Long acting recombinant human growth hormone

Eligibility Criteria

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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Versartis Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Will Charlton, MD

Role: STUDY_DIRECTOR

Vesrartis

Locations

Hokkaido University Hospital

Sapporo, , Japan

Countries

Japan

Other Identifiers

J14VR5

Identifier Type: -

Identifier Source: org_study_id