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

Last updated: July 22, 2022
Sponsor: Versartis Inc.
Overall Status: Terminated

Phase

2/3

Condition

Growth Hormone Deficiencies/abnormalities

Severe Short Stature

Treatment

N/A

Clinical Study ID

NCT02413138
J14VR5
  • Ages 3-10
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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

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 atscreening
  • Significant spinal abnormalities including scoliosis, kyphosis and spina bifidavariants
  • Significant abnormality in screening laboratory studies

Study Design

Total Participants: 41
Study Start date:
August 08, 2015
Estimated Completion Date:
November 30, 2017

Study 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.

Connect with a study center

  • Akita University Hospital

    Akita,
    Japan

    Site Not Available

  • Ehime University Hospital

    Ehime,
    Japan

    Site Not Available

  • Fukuoka University Hospital

    Fukuoka,
    Japan

    Site Not Available

  • Gunma University Hospital

    Gunma,
    Japan

    Site Not Available

  • Hamamatsu University Hospital

    Hamamatsu,
    Japan

    Site Not Available

  • Kure Medical Center

    Hiroshima,
    Japan

    Site Not Available

  • Hokkaido University Hospital

    Hokkaido,
    Japan

    Site Not Available

  • Morioka Children's Hospital

    Iwate,
    Japan

    Site Not Available

  • Kanagawa Prefectural Hospital

    Kanagawa,
    Japan

    Site Not Available

  • Kanazawa Medical University Hospital

    Kanazawa,
    Japan

    Site Not Available

  • Kumamoto University Hospital

    Kumamoto,
    Japan

    Site Not Available

  • University of Miyazaki Hospital

    Miyazaki,
    Japan

    Site Not Available

  • Aichi Children's Health and Medical Center

    Nagoya,
    Japan

    Site Not Available

  • Niigata University Medical and Dental Hospital

    Niigata,
    Japan

    Site Not Available

  • Oita University Hospital

    Oita,
    Japan

    Site Not Available

  • Okayama Saiseikai General Hospital

    Okayama,
    Japan

    Site Not Available

  • Osaka Police Hospital

    Osaka,
    Japan

    Site Not Available

  • Saitama Children's Medical Center

    Saitama,
    Japan

    Site Not Available

  • Hokkaido University Hospital

    Sapporo,
    Japan

    Site Not Available

  • Shiga University of Medical Science Hospital

    Shiga,
    Japan

    Site Not Available

  • Tanaka Growth Clinic

    Tokyo,
    Japan

    Site Not Available

  • Tottori University Hosptial

    Tottori,
    Japan

    Site Not Available

  • Iwakuni Medical Center

    Yamaguchi,
    Japan

    Site Not Available

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