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Open-label Extension Study to Evaluate the Safety of Long-term Twice-monthly Administration of Somavaratan in Adult Growth Hormone Deficiency (AGHD)

Primary Purpose

Adult Growth Hormone Deficiency (AGHD)

Status
Terminated
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
somavaratan
Sponsored by
Versartis Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Adult Growth Hormone Deficiency (AGHD) focused on measuring VRS-317, Long Acting Growth Hormone, Versartis, somavaratan, Growth Hormone Deficiency, Adult Growth Hormone Deficiency, AGHD, Growth Hormone Replacement, Growth Hormone Replacement Therapy, XTEN

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Female subjects of childbearing potential must have negative pregnancy test and use appropriate contraceptive methods
  • Documented GHD during adulthood
  • Subjects naive to somavaratan must have an IGF-1 SDS value ≤ 0 at Screening
  • Subjects taking other hormone replacement therapy must have been on a stable course of treatment for at least 3 months
  • Underlying disorders responsible for the subject's GHD must have been clinically stable for at least 6 months
  • Subjects receiving daily rhGH injections must washout for ≥ 14 days
  • BMI (kg/m2) between 18.0 and 40.0

Exclusion Criteria:

  • Untreated adrenal insufficiency
  • Recently diagnosed thyroid dysfunction which is not being treated or has not been stable on therapy for at least 3 months
  • Currently taking anti-inflammatory dose of glucocorticoids that could potentially compromise safety or efficacy assessments
  • Currently taking a GHRH or IGF-I product
  • Current significant cardiovascular disease, heart insufficiency of New York Heart Association (NYHA) class > 2
  • Current significant disease thought to increase risk of receiving growth hormone or confound assessment of study outcomes
  • History of diabetes mellitus or inadequate glucose control
  • Current drug or alcohol abuse
  • Current HIV wasting syndrome (HIV testing not required)
  • History of malignancy in adulthood (subjects with a history of childhood malignancy that were subsequently treated with rhGH in childhood and remain GHD in adulthood may be enrolled)
  • Women who are pregnant or breastfeeding
  • Treatment with an investigational drug other than somavaratan within 30 days prior to Screening
  • A significant abnormality in Screening laboratory results

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Somavaratan

    Arm Description

    Long-acting recombinant human growth hormone therapy administered subcutaneously twice-monthly in adult participants with GHD

    Outcomes

    Primary Outcome Measures

    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'.

    Secondary Outcome Measures

    Average Dose Level During Titration/Maintenance
    Total average dose received by a participant during titration/maintenance has been reported.
    Number of Participants With Dose Adjustments
    The number of participants with a dose adjustment (titrated up/down) at each month are summarized.
    Number of Participants Who Were Anti-drug Antibody (ADA) Positive
    Number of Participants With Positive Neutralizing Antibodies (NABs)
    Change From Baseline in Mean Insulin-like Growth Factor 1 (IGF-I) Standard Deviation Score (SDS) at Specified Timepoints During Maintenance Period
    Changes in the IGF-I levels were assessed as Standard Deviation Scores (SDS). The SDS 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 standard deviation (SD) vary depending on the age and sex of the participant. Change in IGF-I level (SDS) at specified timepoints from baseline was assessed. A higher score reflects a better outcome.
    Mean Insulin-like Growth Factor-binding Protein 3 (IGFBP-3) SDS During Maintenance Period

    Full Information

    First Posted
    March 16, 2016
    Last Updated
    March 20, 2023
    Sponsor
    Versartis Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02719990
    Brief Title
    Open-label Extension Study to Evaluate the Safety of Long-term Twice-monthly Administration of Somavaratan in Adult Growth Hormone Deficiency (AGHD)
    Official Title
    An Open-Label, Long-Term Extension Study of the Safety of Somavaratan (VRS-317) in Adults With Growth Hormone Deficiency (GHD)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2023
    Overall Recruitment Status
    Terminated
    Why Stopped
    Phase 3 clinical trial of somavaratan in pediatric growth hormone deficiency (PGHD) did not meet its primary endpoint of non-inferiority.
    Study Start Date
    February 11, 2016 (Actual)
    Primary Completion Date
    January 11, 2018 (Actual)
    Study Completion Date
    January 11, 2018 (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
    Open-label extension study to evaluate the safety of long-term twice-monthly administration of somavaratan in adults with Growth Hormone Deficiency (GHD).
    Detailed Description
    An open-label extension study to evaluate the safety of long-term twice-monthly administration of somavaratan in adults with GHD. This multicenter study is open to participants completing a Versartis adult GHD study as well as approximately 40 new somavaratan naïve participants (either recombinant human growth hormone [rhGH] treatment naïve or currently receiving daily rhGH therapy). All participants will receive twice-monthly (every 15 days ± 2 days) subcutaneous (SC) somavaratan. Doses will be titrated to each participant's individual insulin-like growth factor-I (IGF-I) responses based on the IGF-I level 7 days post-dose until a maintenance dose is achieved. Participants receiving somavaratan in a previous somavaratan study will have their dose decreased by half (minimum dose of 20 milligrams [mg], 40 mg for women on estrogen, rounded down to the nearest even number) and will be titrated per the Dose Titration Plan. New participants enrolling in this study will be assigned to one of two cohorts based on sensitivity to rhGH and titrated per the Dose Titration Plan.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Adult Growth Hormone Deficiency (AGHD)
    Keywords
    VRS-317, Long Acting Growth Hormone, Versartis, somavaratan, Growth Hormone Deficiency, Adult Growth Hormone Deficiency, AGHD, Growth Hormone Replacement, Growth Hormone Replacement Therapy, XTEN

    7. Study Design

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

    8. Arms, Groups, and Interventions

    Arm Title
    Somavaratan
    Arm Type
    Experimental
    Arm Description
    Long-acting recombinant human growth hormone therapy administered subcutaneously twice-monthly in adult participants with GHD
    Intervention Type
    Drug
    Intervention Name(s)
    somavaratan
    Other Intervention Name(s)
    VRS-317
    Intervention Description
    Long-acting recombinant human growth hormone therapy administered subcutaneously twice-monthly
    Primary Outcome Measure Information:
    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
    From first dose of study drug up to approximately 2 years
    Secondary Outcome Measure Information:
    Title
    Average Dose Level During Titration/Maintenance
    Description
    Total average dose received by a participant during titration/maintenance has been reported.
    Time Frame
    Up to Month 12
    Title
    Number of Participants With Dose Adjustments
    Description
    The number of participants with a dose adjustment (titrated up/down) at each month are summarized.
    Time Frame
    Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12
    Title
    Number of Participants Who Were Anti-drug Antibody (ADA) Positive
    Time Frame
    Up to approximately 2 years
    Title
    Number of Participants With Positive Neutralizing Antibodies (NABs)
    Time Frame
    Up to approximately 2 years
    Title
    Change From Baseline in Mean Insulin-like Growth Factor 1 (IGF-I) Standard Deviation Score (SDS) at Specified Timepoints During Maintenance Period
    Description
    Changes in the IGF-I levels were assessed as Standard Deviation Scores (SDS). The SDS 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 standard deviation (SD) vary depending on the age and sex of the participant. Change in IGF-I level (SDS) at specified timepoints from baseline was assessed. A higher score reflects a better outcome.
    Time Frame
    Baseline, Month 2 Day 1, Month 2 Day 8, Month 3 Day 1, Month 3 Day 8, Month 4 Day 1, Month 4 Day 8, Month 5 Day 1, Month 5 Day 8, Month 6 Day 1, Month 6 Day 8, Month 7 Day 1, Month 7 Day 8, Month 8 Day 1, Month 8 Day 8, Month 10 Day 1, and Month 10 Day 8
    Title
    Mean Insulin-like Growth Factor-binding Protein 3 (IGFBP-3) SDS During Maintenance Period
    Time Frame
    Baseline up to Month 12

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    75 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Female participants of childbearing potential must have negative pregnancy test and use appropriate contraceptive methods Documented GHD during adulthood Participants naive to somavaratan must have an IGF-1 SDS value ≤ 0 at Screening Participants taking other hormone replacement therapy must have been on a stable course of treatment for at least 3 months Underlying disorders responsible for the participant's GHD must have been clinically stable for at least 6 months Participants receiving daily rhGH injections must washout for ≥ 14 days Body mass index (BMI) (kilograms [kg]/meter square [m^2]) between 18.0 and 40.0 Exclusion Criteria: Untreated adrenal insufficiency Recently diagnosed thyroid dysfunction which is not being treated or has not been stable on therapy for at least 3 months Currently taking anti-inflammatory dose of glucocorticoids that could potentially compromise safety or efficacy assessments Currently taking a GHRH or IGF-I product Current significant cardiovascular disease, heart insufficiency of New York Heart Association (NYHA) class > 2 Current significant disease thought to increase risk of receiving growth hormone or confound assessment of study outcomes History of diabetes mellitus or inadequate glucose control Current drug or alcohol abuse Current human immunodeficiency virus (HIV) wasting syndrome (HIV testing not required) History of malignancy in adulthood (participants with a history of childhood malignancy that were subsequently treated with rhGH in childhood and remain GHD in adulthood may be enrolled) Women who are pregnant or breastfeeding Treatment with an investigational drug other than somavaratan within 30 days prior to Screening A significant abnormality in Screening laboratory results
    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

    Learn more about this trial

    Open-label Extension Study to Evaluate the Safety of Long-term Twice-monthly Administration of Somavaratan in Adult Growth Hormone Deficiency (AGHD)

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