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

Primary Purpose

Pediatric Growth Hormone Deficiency, Growth Disorders

Status

Terminated

Phase

Phase 2

Locations

Japan

Study Type

Interventional

Intervention

Somavaratan (VRS-317)

About this trial

This is an interventional treatment trial for Pediatric Growth Hormone Deficiency focused on measuring GHD

Eligibility Criteria

3 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Hokkaido University Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Phase 2: Somavaratan (VRS-317)

Phase 3: Somavaratan (VRS-317)

Arm Description

Active treatment arm

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

Outcomes

Primary Outcome Measures

Efficacy (Annual Height velocity)

Annual Height velocity.

Secondary Outcome Measures

Pharmacodynamics (IGF-I responses to study drug administration)

IGF-I responses to study drug administration.

Pharmacodynamics (IGFBP-3 responses to study drug administration)

IGFBP-3 responses to study drug administration.

Safety (Number of subjects with adverse events )

Number of subjects with adverse events (including repeat dose immunogenicity).

Safety (Concomitant medications)

Concomitant medications

Safety (Safety labs)

Safety labs

Safety (Vital signs)

Vital signs

Safety (Physical Exams)

Physical Exams

Full Information

NCT ID

NCT02413138

First Posted

March 23, 2015

Last Updated

July 22, 2022

Sponsor

Versartis Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02413138

Brief Title

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

Official Title

A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)

Study Type

Interventional

2. Study Status

Record Verification Date

July 2022

Overall Recruitment Status

Terminated

Why Stopped

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

Study Start Date

August 8, 2015 (Actual)

Primary Completion Date

November 30, 2017 (Actual)

Study Completion Date

November 30, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Versartis Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pediatric Growth Hormone Deficiency, Growth Disorders

Keywords

GHD

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

41 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Phase 2: Somavaratan (VRS-317)

Arm Type

Experimental

Arm Description

Active treatment arm

Arm Title

Phase 3: Somavaratan (VRS-317)

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Somavaratan (VRS-317)

Other Intervention Name(s)

Long acting recombinant human growth hormone

Intervention Description

Long acting recombinant human growth hormone

Primary Outcome Measure Information:

Title

Efficacy (Annual Height velocity)

Description

Annual Height velocity.

Time Frame

12 months

Secondary Outcome Measure Information:

Title

Pharmacodynamics (IGF-I responses to study drug administration)

Description

IGF-I responses to study drug administration.

Time Frame

12 months

Title

Pharmacodynamics (IGFBP-3 responses to study drug administration)

Description

IGFBP-3 responses to study drug administration.

Time Frame

12 months

Title

Safety (Number of subjects with adverse events )

Description

Number of subjects with adverse events (including repeat dose immunogenicity).

Time Frame

12 months

Title

Safety (Concomitant medications)

Description

Concomitant medications

Time Frame

12 months

Title

Safety (Safety labs)

Description

Safety labs

Time Frame

12 months

Title

Safety (Vital signs)

Description

Vital signs

Time Frame

12 months

Title

Safety (Physical Exams)

Description

Physical Exams

Time Frame

12 months

Other Pre-specified Outcome Measures:

Title

Secondary Efficacy (Change in height SDS)

Description

Change in height SDS.

Time Frame

12 months

Title

Secondary Efficacy

Description

Change in body weight

Time Frame

12 months

Title

Secondary Efficacy

Description

Change in body mass index.

Time Frame

12 months

Title

Secondary Efficacy (Change in bone age)

Description

Change in bone age.

Time Frame

12 months

Title

Secondary Efficacy (Change in pubertal staging.)

Description

Change in pubertal staging.

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Will Charlton, MD

Organizational Affiliation

Vesrartis

Official's Role

Study Director

Facility Information:

Facility Name

Hokkaido University Hospital

City

Sapporo

Country

Japan

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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