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Long-Acting Growth Hormone in Children Compared to Daily rhGH (VELOCITY)

Primary Purpose

Growth Disorders

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Somavaratan
rhGH
Sponsored by
Versartis Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Growth Disorders focused on measuring VRS-317, Growth Hormone Deficiency, Long Acting Recombinant Growth Hormone, Long Acting Growth Hormone, Pediatric Growth Hormone Deficiency, Growth Hormone Replacement Therapy, Versartis, Xten, Human Growth Hormone, somavaratan, growth failure

Eligibility Criteria

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

Inclusion Criteria:

  • Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
  • Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
  • Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
  • Height standard deviation score (SDS) ≤ -2.0 at screening.
  • Weight for Stature ≥ 10th percentile.
  • Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening.
  • Delayed bone age (≥ 6 months).

Exclusion Criteria:

  • Prior treatment with any growth promoting agent
  • History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
  • Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
  • A diagnosis of Attention Deficit Hyperactivity Disorder.
  • Daily use of anti-inflammatory doses of glucocorticoid.
  • Prior history of leukemia, lymphoma, sarcoma or cancer.
  • Treatment with an investigational drug in the 30 days prior to screening.
  • Known allergy to constituents of the study drug formulation.
  • 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

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Active Comparator

    Arm Label

    Somavaratan

    rhGH

    Arm Description

    Participants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months.

    Participants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months.

    Outcomes

    Primary Outcome Measures

    Annual Height Velocity
    Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) * 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.

    Secondary Outcome Measures

    Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
    Height SDS was determined using the Center for Disease Control (CDC) Clinical Growth Charts; 2000. The SD score was calculated as the participant's height value minus the mean divided by the standard deviation (SD). The mean and the SD vary depending on the age and sex of the participant. Mean change from baseline in height SDS at Month 12 is presented.
    Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
    Bone age was assessed from a radiograph of the left hand and wrist by central reader.
    Change From Baseline in Body Mass Index (BMI) at Month 12
    The BMI is a person's weight in kilograms (kg) divided by the square of height in meters.
    Change From Baseline in Body Weight at Month 12
    Body weight measured in light clothing and without shoes.
    Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
    The SD score was calculated as the actual value of IGF-I minus mean reference value of IGF-1 divided by reference standard deviation of IGF-I. The mean and the SD vary depending on the age and sex of the participant. Change in IGF-I level (SD score) at Month 12 from Baseline was assessed. A higher score reflects a better outcome.
    Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
    Number of Participants With Adverse Events (AEs)
    An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.

    Full Information

    First Posted
    January 7, 2015
    Last Updated
    November 28, 2022
    Sponsor
    Versartis Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02339090
    Brief Title
    Long-Acting Growth Hormone in Children Compared to Daily rhGH
    Acronym
    VELOCITY
    Official Title
    Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    November 2022
    Overall Recruitment Status
    Completed
    Study Start Date
    August 26, 2015 (Actual)
    Primary Completion Date
    August 23, 2017 (Actual)
    Study Completion Date
    August 23, 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 compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
    Detailed Description
    This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. 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
    Growth Disorders
    Keywords
    VRS-317, Growth Hormone Deficiency, Long Acting Recombinant Growth Hormone, Long Acting Growth Hormone, Pediatric Growth Hormone Deficiency, Growth Hormone Replacement Therapy, Versartis, Xten, Human Growth Hormone, somavaratan, growth failure

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    138 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Somavaratan
    Arm Type
    Experimental
    Arm Description
    Participants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months.
    Arm Title
    rhGH
    Arm Type
    Active Comparator
    Arm Description
    Participants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months.
    Intervention Type
    Drug
    Intervention Name(s)
    Somavaratan
    Other Intervention Name(s)
    Long-acting recombinant human growth hormone, VRS-317
    Intervention Description
    Somavaratan will be administered per dose and schedule specified in the arm description.
    Intervention Type
    Drug
    Intervention Name(s)
    rhGH
    Other Intervention Name(s)
    daily growth hormone, recombinant growth hormone therapy
    Intervention Description
    rhGH will be administered per dose and schedule specified in the arm description.
    Primary Outcome Measure Information:
    Title
    Annual Height Velocity
    Description
    Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) * 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.
    Time Frame
    12 months
    Secondary Outcome Measure Information:
    Title
    Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
    Description
    Height SDS was determined using the Center for Disease Control (CDC) Clinical Growth Charts; 2000. The SD score was calculated as the participant's height value minus the mean divided by the standard deviation (SD). The mean and the SD vary depending on the age and sex of the participant. Mean change from baseline in height SDS at Month 12 is presented.
    Time Frame
    Baseline, Month 12
    Title
    Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
    Description
    Bone age was assessed from a radiograph of the left hand and wrist by central reader.
    Time Frame
    Baseline, Month 12
    Title
    Change From Baseline in Body Mass Index (BMI) at Month 12
    Description
    The BMI is a person's weight in kilograms (kg) divided by the square of height in meters.
    Time Frame
    Baseline, Month 12
    Title
    Change From Baseline in Body Weight at Month 12
    Description
    Body weight measured in light clothing and without shoes.
    Time Frame
    Baseline, Month 12
    Title
    Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
    Description
    The SD score was calculated as the actual value of IGF-I minus mean reference value of IGF-1 divided by reference standard deviation of IGF-I. The mean and the SD vary depending on the age and sex of the participant. Change in IGF-I level (SD score) at Month 12 from Baseline was assessed. A higher score reflects a better outcome.
    Time Frame
    Baseline, Month 12
    Title
    Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
    Time Frame
    Baseline, Month 12
    Title
    Number of Participants With Adverse Events (AEs)
    Description
    An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
    Time Frame
    Baseline up to Month 12

    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 ≤ 10.0 (girls) and ≤ 11.0 (boys). Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys. Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL. Height standard deviation score (SDS) ≤ -2.0 at screening. Weight for Stature ≥ 10th percentile. Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening. Delayed bone age (≥ 6 months). Exclusion Criteria: Prior treatment with any growth promoting agent History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency). Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome. A diagnosis of Attention Deficit Hyperactivity Disorder. Daily use of anti-inflammatory doses of glucocorticoid. Prior history of leukemia, lymphoma, sarcoma or cancer. Treatment with an investigational drug in the 30 days prior to screening. Known allergy to constituents of the study drug formulation. 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
    Sponsor GmbH
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Plan to Share IPD
    No

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    Long-Acting Growth Hormone in Children Compared to Daily rhGH

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