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Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)

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ClinicalTrials.gov Identifier: NCT02413138
Recruitment Status : Terminated (The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved)
First Posted : April 9, 2015
Last Update Posted : March 9, 2018
Sponsor:
Information provided by (Responsible Party):
Versartis Inc.

Brief Summary:
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.

Condition or disease Intervention/treatment Phase
Pediatric Growth Hormone Deficiency Growth Disorders Drug: Somavaratan (VRS-317) Phase 2 Phase 3

Detailed Description:
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)
Actual Study Start Date : August 8, 2015
Actual Primary Completion Date : November 30, 2017
Actual Study Completion Date : November 30, 2017


Arm Intervention/treatment
Experimental: Phase 2: Somavaratan (VRS-317)
Active treatment arm
Drug: Somavaratan (VRS-317)
Long acting recombinant human growth hormone
Other Name: Long acting recombinant human growth hormone
Experimental: Phase 3: Somavaratan (VRS-317)
Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly
Drug: Somavaratan (VRS-317)
Long acting recombinant human growth hormone
Other Name: Long acting recombinant human growth hormone



Primary Outcome Measures :
  1. Efficacy (Annual Height velocity) [ Time Frame: 12 months ]
    Annual Height velocity.


Secondary Outcome Measures :
  1. Pharmacodynamics (IGF-I responses to study drug administration) [ Time Frame: 12 months ]
    IGF-I responses to study drug administration.

  2. Pharmacodynamics (IGFBP-3 responses to study drug administration) [ Time Frame: 12 months ]
    IGFBP-3 responses to study drug administration.

  3. Safety (Number of subjects with adverse events ) [ Time Frame: 12 months ]
    Number of subjects with adverse events (including repeat dose immunogenicity).

  4. Safety (Concomitant medications) [ Time Frame: 12 months ]
    Concomitant medications

  5. Safety (Safety labs) [ Time Frame: 12 months ]
    Safety labs

  6. Safety (Vital signs) [ Time Frame: 12 months ]
    Vital signs

  7. Safety (Physical Exams) [ Time Frame: 12 months ]
    Physical Exams


Other Outcome Measures:
  1. Secondary Efficacy (Change in height SDS) [ Time Frame: 12 months ]
    Change in height SDS.

  2. Secondary Efficacy [ Time Frame: 12 months ]
    Change in body weight

  3. Secondary Efficacy [ Time Frame: 12 months ]
    Change in body mass index.

  4. Secondary Efficacy (Change in bone age) [ Time Frame: 12 months ]
    Change in bone age.

  5. Secondary Efficacy (Change in pubertal staging.) [ Time Frame: 12 months ]
    Change in pubertal staging.



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Ages Eligible for Study:   3 Years to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)
  • Pre-pubertal status
  • Diagnosis of GHD as documented by two or more GH stimulation test results
  • Height SD score ≤ -2.0 at screening
  • Weight for Stature ≥ 10th percentile
  • IGF-I SD score ≤ -1.0 at screening
  • Delayed bone age

Exclusion Criteria:

  • Prior treatment with any growth promoting agent
  • History of, or current, significant disease
  • Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome
  • Birth weight and/or birth length less than 5th percentile for gestational age
  • A diagnosis of Attention Deficit Hyperactivity Disorder
  • Daily use of anti-inflammatory doses of glucocorticoid
  • Prior history of leukemia, lymphoma, sarcoma or cancer
  • 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

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 ClinicalTrials.gov identifier (NCT number): NCT02413138


Sponsors and Collaborators
Versartis Inc.
Investigators
Study Director: Will Charlton, MD Vesrartis

Responsible Party: Versartis Inc.
ClinicalTrials.gov Identifier: NCT02413138     History of Changes
Other Study ID Numbers: J14VR5
First Posted: April 9, 2015    Key Record Dates
Last Update Posted: March 9, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Versartis Inc.:
GHD

Additional relevant MeSH terms:
Endocrine System Diseases
Dwarfism, Pituitary
Growth Disorders
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Pathologic Processes
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs