We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

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.
ClinicalTrials.gov Identifier: NCT02413138
Recruitment Status : Active, not recruiting
First Posted : April 9, 2015
Last Update Posted : November 24, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
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 Design

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
Estimated Primary Completion Date : November 30, 2017
Estimated Study Completion Date : November 30, 2017


Arms and Interventions

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


Outcome Measures

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.


Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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
Contacts and Locations

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


Locations
Japan
Akita University Hospital
Akita, Japan
Ehime University Hospital
Ehime, Japan
Fukuoka Children's Hospital
Fukuoka, Japan
Fukuoka University Hospital
Fukuoka, Japan
Gunma University Hospital
Gunma, Japan
Hamamatsu University Hospital
Hamamatsu, Japan
Kure Medical Center
Hiroshima, Japan
Hokkaido University Hospital
Hokkaido, Japan
Morioka Children's Hospital
Iwate, Japan
Kanagawa Prefectural Hospital
Kanagawa, Japan
Kanazawa Medical University Hospital
Kanazawa, Japan
Kumamoto University Hospital
Kumamoto, Japan
University of Miyazaki Hospital
Miyazaki, Japan
Aichi Children's Health and Medical Center
Nagoya, Japan
Nagoya City University Hospital
Nagoya, Japan
Niigata University Medical and Dental Hospital
Niigata, Japan
Oita University Hospital
Oita, Japan
Okayama Saiseikai General Hospital
Okayama, Japan
Japan Community Health care Organization Osaka Hospital
Osaka, Japan
Osaka City General Hospital
Osaka, Japan
Osaka Medical Center and Reserach Institute for Maternal and Child Health
Osaka, Japan
Osaka Police Hospital
Osaka, Japan
Osaka University Hospital
Osaka, Japan
Saitama Children's Medical Center
Saitama, Japan
Shiga University of Medical Science Hospital
Shiga, Japan
Keio University Hospital
Tokyo, Japan
National Center for Child Health and Development
Tokyo, Japan
Nihon University Hospital
Tokyo, Japan
Saitama Medical University Hospital
Tokyo, Japan
Tanaka Growth Clinic
Tokyo, Japan
The Jikei University Hospital
Tokyo, Japan
Tohoku University Hospital
Tokyo, Japan
Tokyo Medical and Dental University
Tokyo, Japan
Tokyo Metropolitan Children's Medical Center
Tokyo, Japan
Toranomon Hospital
Tokyo, Japan
Tottori University Hosptial
Tottori, Japan
Iwakuni Medical Center
Yamaguchi, Japan
Sponsors and Collaborators
Versartis Inc.
Investigators
Principal Investigator: Tomonobu Hasegawa, MD Keio University Hospital
More Information

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: November 24, 2017
Last Verified: November 2017
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