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

NCT02413138 | Terminated Phase 2 DMC

• 1 other identifier: J14VR5
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

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.

Conditions

Pediatric Growth Hormone Deficiency; Growth Disorders

Keywords

GHD

Outcome Measures

Primary Outcomes (1)

• Efficacy (Annual Height velocity)

  Annual Height velocity.

  12 months

Secondary Outcomes (7)

• Pharmacodynamics (IGF-I responses to study drug administration)

  12 months

• Pharmacodynamics (IGFBP-3 responses to study drug administration)

  12 months

• Safety (Number of subjects with adverse events )

  12 months

• Safety (Concomitant medications)

  12 months

• Safety (Safety labs)

  12 months

Other Outcomes (5)

• Secondary Efficacy (Change in height SDS)

  12 months

• Secondary Efficacy

  12 months

• Secondary Efficacy

  12 months

Study Arms (2)

Phase 2: Somavaratan (VRS-317)

EXPERIMENTAL

Active treatment arm

Drug: Somavaratan (VRS-317)

Phase 3: Somavaratan (VRS-317)

EXPERIMENTAL

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

Drug: Somavaratan (VRS-317)

Interventions

Somavaratan (VRS-317) DRUG

Long acting recombinant human growth hormone

Also known as: Long acting recombinant human growth hormone

Phase 2: Somavaratan (VRS-317); Phase 3: Somavaratan (VRS-317)

Eligibility Criteria

• Age: 3 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Versartis Inc.: lead

Study Sites (1)

Hokkaido University Hospital

Sapporo, Japan

Location

MeSH Terms

Conditions

Growth Disorders

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Will Charlton, MD

  Vesrartis

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2015
• First Posted: April 9, 2015
• Study Start: August 8, 2015
• Primary Completion: November 30, 2017
• Study Completion: November 30, 2017
• Last Updated: July 25, 2022

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)