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Versartis Trial in Children to Assess Long-Acting Growth Hormone (VERTICAL)

Primary Purpose

Pediatric Growth Hormone Deficiency

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Experimental: VRS-317
Sponsored by
Versartis Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pediatric Growth Hormone Deficiency focused on measuring Growth hormone, Growth hormone deficiency, Growth, Pediatric growth hormone deficiency, Long acting growth hormone, GHD, PGHD, VRS 317, Exten, Versartis

Eligibility Criteria

3 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Chronological Age ≥ 3.0 years and ≤ 11.0
  • Diagnosis of GHD as documented by GH stimulation test
  • Below average height SDS at screening
  • Appropriate weight for Stature
  • Decreased IGF-I SDS at screening
  • Delayed bone age
  • Normal thyroid function test results at screening visit
  • Legally authorized representative informed consent.

Exclusion Criteria:

  • Prior treatment with any growth promoting agent
  • Documented history of, or current, significant disease
  • Chromosomal aneuploidy, significant gene mutations
  • Diagnosis of Attention Deficit Hyperactivity Disorder
  • Daily use of anti-inflammatory doses of glucocorticoid
  • Prior history of leukemia, lymphoma, sarcoma or cancer
  • Known allergy to constituents of the study drug formulation
  • Abnormal ocular findings at screening
  • Significant abnormality in screening laboratory studies

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VRS-317

Arm Description

Active treatment arm

Outcomes

Primary Outcome Measures

Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a). The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.

Secondary Outcome Measures

Evaluate pharmacokinetic measures of VRS-317.
Secondary outcome measures include evaluation of pharmacokinetics (PK) of VRS-317 including Cmax, Tmax and AUC. PK measures will be analysed at multiple timepoints after single dosing of VRS-317 during Phase 1b and after repeat dosing of VRS-317 during Phase 2a.

Full Information

First Posted
October 19, 2012
Last Updated
July 22, 2022
Sponsor
Versartis Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01718041
Brief Title
Versartis Trial in Children to Assess Long-Acting Growth Hormone
Acronym
VERTICAL
Official Title
A Long-acting Human Growth Hormone (VRS-317) in Pre-pubertal Children With Growth Hormone Deficiency: A Randomized, Open-label, Multi-center, Phase 1b/2a Study of Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Efficacy After Subcutaneous Administration for 6 Months
Study Type
Interventional

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
October 2012 (undefined)
Primary Completion Date
July 2014 (Actual)
Study Completion Date
July 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Versartis Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. During the Phase 2a stage, patients will receive 6 months of VRS-317 treatment at dose levels selected from the Phase 1b stage. The primary endpoints for the study are to determine the safety and efficacy of repeat dose VRS-317.
Detailed Description
In Phase 1b, separate cohorts of patients will be tested in a single ascending dose format. Safety review committee meetings will take place prior to escalating to each increasing dose level. Enrolled patients will be monitored for 60 days for safety with PK/PD and safety labs collected. Two dose levels will be selected after completion of Phase 1b to be tested during Phase 2a (6 months of continuous VRS-317 treatment). Safety and PK assessments will be made during the Phase 2a stage. Patient heights will be measured by stadiometer.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pediatric Growth Hormone Deficiency
Keywords
Growth hormone, Growth hormone deficiency, Growth, Pediatric growth hormone deficiency, Long acting growth hormone, GHD, PGHD, VRS 317, Exten, Versartis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
64 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VRS-317
Arm Type
Experimental
Arm Description
Active treatment arm
Intervention Type
Drug
Intervention Name(s)
Experimental: VRS-317
Intervention Description
Active treatment arm
Primary Outcome Measure Information:
Title
Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
Description
Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a). The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.
Time Frame
8 Months
Secondary Outcome Measure Information:
Title
Evaluate pharmacokinetic measures of VRS-317.
Description
Secondary outcome measures include evaluation of pharmacokinetics (PK) of VRS-317 including Cmax, Tmax and AUC. PK measures will be analysed at multiple timepoints after single dosing of VRS-317 during Phase 1b and after repeat dosing of VRS-317 during Phase 2a.
Time Frame
8 months
Other Pre-specified Outcome Measures:
Title
Evaluate secondary efficacy measures after 6 months of VRS-317 dosing.
Description
Secondary efficacy measures during Phase 2a include change in height SDS and change in bone age after 6 months VRS-317 treatment.
Time Frame
6 Months
Title
Evaluate pharmacodynamic measures
Description
Secondary outcome measures include evaluation of pharmacodynamics (PD) as measured by IGF-I and IGFBP-3 responses to single dose VRS-317 during Phase 1b and as measured by IGF-I and IGFBP-3 responses to multiple doses of VRS-317 after repeat dosing during Phase 2a. PD measures will be obtained at multiple timepoints during Phase 1b and Phase 2a.
Time Frame
8 Months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Chronological Age ≥ 3.0 years and ≤ 11.0 Diagnosis of GHD as documented by GH stimulation test Below average height SDS at screening Appropriate weight for Stature Decreased IGF-I SDS at screening Delayed bone age Normal thyroid function test results at screening visit Legally authorized representative informed consent. Exclusion Criteria: Prior treatment with any growth promoting agent Documented history of, or current, significant disease Chromosomal aneuploidy, significant gene mutations Diagnosis of Attention Deficit Hyperactivity Disorder Daily use of anti-inflammatory doses of glucocorticoid Prior history of leukemia, lymphoma, sarcoma or cancer Known allergy to constituents of the study drug formulation Abnormal ocular findings at screening Significant abnormality in screening laboratory studies
Facility Information:
City
Clovis
State/Province
California
Country
United States
City
Los Angeles
State/Province
California
Country
United States
City
San Diego
State/Province
California
Country
United States
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San Francisco
State/Province
California
Country
United States
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Greenwood Village
State/Province
Colorado
Country
United States
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Orlando
State/Province
Florida
Country
United States
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Tallahassee
State/Province
Florida
Country
United States
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Tampa
State/Province
Florida
Country
United States
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Atlanta
State/Province
Georgia
Country
United States
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Indianapolis
State/Province
Indiana
Country
United States
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Baltimore
State/Province
Maryland
Country
United States
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Springfield
State/Province
Massachusetts
Country
United States
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Minneapolis
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Minnesota
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United States
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Kansas City
State/Province
Missouri
Country
United States
City
Livingston
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New Jersey
Country
United States
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Morristown
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New Jersey
Country
United States
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Buffalo
State/Province
New York
Country
United States
City
Mineola
State/Province
New York
Country
United States
City
Sleepy Hollow
State/Province
New York
Country
United States
City
Cleveland
State/Province
Ohio
Country
United States
City
Columbus
State/Province
Ohio
Country
United States
City
San Antonio
State/Province
Texas
Country
United States
City
Seattle
State/Province
Washington
Country
United States

12. IPD Sharing Statement

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Versartis Trial in Children to Assess Long-Acting Growth Hormone

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