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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01718041
Recruitment Status : Completed
First Posted : October 31, 2012
Last Update Posted : August 9, 2016
Information provided by (Responsible Party):
Versartis Inc.

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.

Condition or disease Intervention/treatment Phase
Pediatric Growth Hormone Deficiency Drug: Experimental: VRS-317 Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : October 2012
Actual Primary Completion Date : July 2014
Actual Study Completion Date : July 2014

Arm Intervention/treatment
Experimental: VRS-317
Active treatment arm
Drug: Experimental: VRS-317
Active treatment arm

Primary Outcome Measures :
  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 [ Time Frame: 8 Months ]

    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 :
  1. Evaluate pharmacokinetic measures of VRS-317. [ Time Frame: 8 months ]
    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.

Other Outcome Measures:
  1. Evaluate secondary efficacy measures after 6 months of VRS-317 dosing. [ Time Frame: 6 Months ]
    Secondary efficacy measures during Phase 2a include change in height SDS and change in bone age after 6 months VRS-317 treatment.

  2. Evaluate pharmacodynamic measures [ Time Frame: 8 Months ]
    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.

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01718041

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United States, California
Clovis, California, United States
Los Angeles, California, United States
San Diego, California, United States
San Francisco, California, United States
United States, Colorado
Greenwood Village, Colorado, United States
United States, Florida
Orlando, Florida, United States
Tallahassee, Florida, United States
Tampa, Florida, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, Maryland
Baltimore, Maryland, United States
United States, Massachusetts
Springfield, Massachusetts, United States
United States, Minnesota
Minneapolis, Minnesota, United States
United States, Missouri
Kansas City, Missouri, United States
United States, New Jersey
Livingston, New Jersey, United States
Morristown, New Jersey, United States
United States, New York
Buffalo, New York, United States
Mineola, New York, United States
Sleepy Hollow, New York, United States
United States, Ohio
Cleveland, Ohio, United States
Columbus, Ohio, United States
United States, Texas
San Antonio, Texas, United States
United States, Washington
Seattle, Washington, United States
Sponsors and Collaborators
Versartis Inc.
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Responsible Party: Versartis Inc. Identifier: NCT01718041    
Other Study ID Numbers: VRS-317 Protocol 12VR2
First Posted: October 31, 2012    Key Record Dates
Last Update Posted: August 9, 2016
Last Verified: August 2016
Keywords provided by Versartis Inc.:
Growth hormone
Growth hormone deficiency
Pediatric growth hormone deficiency
Long acting growth hormone
VRS 317
Additional relevant MeSH terms:
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Dwarfism, Pituitary
Endocrine System Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases