NCT01718041

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
64

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Oct 2012

Geographic Reach
1 country

23 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2012

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

October 19, 2012

Completed
12 days until next milestone

First Posted

Study publicly available on registry

October 31, 2012

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2014

Completed
Last Updated

July 26, 2022

Status Verified

July 1, 2022

Enrollment Period

1.7 years

First QC Date

October 19, 2012

Last Update Submit

July 22, 2022

Conditions

Keywords

Growth hormoneGrowth hormone deficiencyGrowthPediatric growth hormone deficiencyLong acting growth hormoneGHDPGHDVRS 317ExtenVersartis

Outcome Measures

Primary Outcomes (1)

  • 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.

    8 Months

Secondary Outcomes (1)

  • Evaluate pharmacokinetic measures of VRS-317.

    8 months

Other Outcomes (2)

  • Evaluate secondary efficacy measures after 6 months of VRS-317 dosing.

    6 Months

  • Evaluate pharmacodynamic measures

    8 Months

Study Arms (1)

VRS-317

EXPERIMENTAL

Active treatment arm

Drug: Experimental: VRS-317

Interventions

Active treatment arm

VRS-317

Eligibility Criteria

Age3 Years - 11 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (23)

Unknown Facility

Clovis, California, United States

Location

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

San Diego, California, United States

Location

Unknown Facility

San Francisco, California, United States

Location

Unknown Facility

Greenwood Village, Colorado, United States

Location

Unknown Facility

Orlando, Florida, United States

Location

Unknown Facility

Tallahassee, Florida, United States

Location

Unknown Facility

Tampa, Florida, United States

Location

Unknown Facility

Atlanta, Georgia, United States

Location

Unknown Facility

Indianapolis, Indiana, United States

Location

Unknown Facility

Baltimore, Maryland, United States

Location

Unknown Facility

Springfield, Massachusetts, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

Kansas City, Missouri, United States

Location

Unknown Facility

Livingston, New Jersey, United States

Location

Unknown Facility

Morristown, New Jersey, United States

Location

Unknown Facility

Buffalo, New York, United States

Location

Unknown Facility

Mineola, New York, United States

Location

Unknown Facility

Sleepy Hollow, New York, United States

Location

Unknown Facility

Cleveland, Ohio, United States

Location

Unknown Facility

Columbus, Ohio, United States

Location

Unknown Facility

San Antonio, Texas, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

DwarfismBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesBone Diseases, EndocrineHypopituitarismPituitary DiseasesHypothalamic DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEndocrine System Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 19, 2012

First Posted

October 31, 2012

Study Start

October 1, 2012

Primary Completion

July 1, 2014

Study Completion

July 1, 2014

Last Updated

July 26, 2022

Record last verified: 2022-07

Locations