Versartis Trial in Pre-pubertal Japanese Children With Gr... | NCT02413138

Status Module

Record Verification Date

Jul 2022

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved

Expanded Access Info

Start Date

Aug 8, 2015Actual

Primary Completion Date

Nov 30, 2017Actual

Completion Date

Nov 30, 2017Actual

First Submitted Date

Mar 23, 2015

First Submission Date that Met QC Criteria

Apr 8, 2015

First Posted Date

Apr 9, 2015Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 22, 2022

Last Update Posted Date

Jul 25, 2022Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

41Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Somavaratan (VRS-317)	Drug	Long acting recombinant human growth hormone	Phase 2: Somavaratan (VRS-317) Phase 3: Somavaratan (VRS-317)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Efficacy (Annual Height velocity)	Annual Height velocity.	12 months

Secondary Outcomes

Measure	Description	Time Frame
Pharmacodynamics (IGF-I responses to study drug administration)	IGF-I responses to study drug administration.	12 months
Pharmacodynamics (IGFBP-3 responses to study drug administration)	IGFBP-3 responses to study drug administration.

Other Outcomes

Measure	Description	Time Frame
Secondary Efficacy (Change in height SDS)	Change in height SDS.	12 months
Secondary Efficacy	Change in body weight	12 months

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

3 Years

Maximum Age

10 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Will Charlton, MD	Vesrartis	Study Director

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Derived Section

Miscellaneous Info Module

Version Holder

Jul 10, 2026

Removed Countries

Not provided

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

MeSH Terms

Intervention Browse Module

No data available

No data is available for this block.

ID	Term
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms