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A Long-Term Safety Study of Somavaratan in Japanese Children With Growth Hormone Deficiency

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ClinicalTrials.gov Identifier: NCT03145831
Recruitment Status : Terminated (The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved)
First Posted : May 9, 2017
Last Update Posted : March 9, 2018
Sponsor:
Information provided by (Responsible Party):
Versartis Inc.

Brief Summary:
This study is a multi-center, open-label safety study assessing long-term somavaratan administration.

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency Drug: Somavaratan Phase 3

Detailed Description:
This study is a multi-center, open-label safety study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan Japanese Phase 2/3 study (Protocol J14VR5) in children with growth hormone deficiency (GHD), as well as approximately 20 new children currently receiving daily rhGH therapy for GHD (switch subjects). For switch subjects, the first dose of somavaratan will be administered approximately 48 hours after the last dose of the daily rhGH. All subjects will receive somavaratan 3.5mg/kg twice-monthly. The study will be conducted at approximately 40 medical institutions in Japan.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Long-Term Safety Study of Long-acting Human Growth Hormone Somavaratan (VRS-317) in Japanese Children With Growth Hormone Deficiency
Actual Study Start Date : March 31, 2017
Actual Primary Completion Date : November 30, 2017
Actual Study Completion Date : November 30, 2017


Arm Intervention/treatment
Experimental: Somavaratan
fusion protein, subcutaneous bolus injection, 3.5 mg/kg twice monthly
Drug: Somavaratan
All subjects will receive somavaratan 3.5 mg/kg twice monthly (every 15 days ± 2 days). Administered as a subcutaneous bolus injection.
Other Name: VRS 317




Primary Outcome Measures :
  1. Adverse Events [ Time Frame: 12 months ]
    Incidence and severity of adverse events


Secondary Outcome Measures :
  1. Height velocity [ Time Frame: 12 months ]
    Comparison of Height Velocity (HV) and HV-SDS before and after switching therapy

  2. IGF-I expression [ Time Frame: 12 months ]
    Change from Day 1

  3. Immunogenicity [ Time Frame: 12 months ]
    Evaluated by anti-drug antibody response



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Chronological Age ≥ 3.0 years.
  2. Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
  3. Subjects with GHD (diagnosed according to the current diagnostic guidelines) who are receiving treatment with daily rhGH.
  4. Normal thyroid function at screening visit in subjects not being treated for hypothyroidism. Subjects requiring thyroxine replacement must be considered adequately treated by the PI and Medical Monitor.
  5. Normal adrenal function (morning cortisol and/or local stimulation test) at screening visit or within 6 months of the screening visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration.
  6. Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
  7. Willingness to discontinue daily rhGH therapy.
  8. Legally authorized representatives must be willing and able to give informed consent

Exclusion Criteria:

  • 1. Prior (in the last 12 months) or concomitant treatment with a growth promoting agent other than rhGH [e.g., IGF-I, GH releasing hormone (GHRH), sex steroids (except when used as primer for GH stimulation test), aromatase inhibitors and/or GnRH agonist].

    2. Current significant disease (e.g., diabetes, cystic fibrosis, renal insufficiency). In all cases of concurrent disease, screening must be approved in writing by the medical monitor.

    3. Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome (e.g., Russell Silver, Prader Willi, Turner, etc.).

    4. Birth weight and/or birth length less than 5th percentile for gestational age using local gestational age growth charts.

    5. Prolonged daily (> 14 days) use of anti-inflammatory doses of oral glucocorticoids.

    6. Prior history of malignancy. 7. Treatment with an investigational drug in the 30 days prior to screening. 8. Known allergy to constituents of the study drug formulation. 9. Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.

    10. Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants.

    11. Significant abnormality in screening laboratory studies (as assessed by PI and medical monitor).

    12. Current social conditions which would prevent completion of study activities (e.g., planned family move to a distant location).

    13. History of pancreatitis or undiagnosed chronic abdominal pain. 14. History of spinal or total body irradiation. 15. Presence of other pituitary hormone deficiencies that are not properly treated.

    16. Unwillingness to provide consent for participation in all trial activities


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): NCT03145831


Locations
United States, California
Eric Humphriss
Menlo Park, California, United States, 94025
Sponsors and Collaborators
Versartis Inc.
Investigators
Study Director: Will Charlton, MD Vesrartis

Responsible Party: Versartis Inc.
ClinicalTrials.gov Identifier: NCT03145831     History of Changes
Other Study ID Numbers: J15VR6
First Posted: May 9, 2017    Key Record Dates
Last Update Posted: March 9, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Versartis Inc.:
Pediatrics

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