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

NCT03145831 | Terminated Phase 3 DMC

• 1 other identifier: J15VR6
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Growth Hormone Deficiency

Keywords

Pediatrics

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Incidence and severity of adverse events

  12 months

Secondary Outcomes (3)

• Height velocity

  12 months

• IGF-I expression

  12 months

• Immunogenicity

  12 months

Study Arms (1)

Somavaratan

EXPERIMENTAL

fusion protein, subcutaneous bolus injection, 3.5 mg/kg twice monthly

Drug: Somavaratan

Interventions

Somavaratan DRUG

All subjects will receive somavaratan 3.5 mg/kg twice monthly (every 15 days ± 2 days). Administered as a subcutaneous bolus injection.

Also known as: VRS 317

Somavaratan

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Chronological Age ≥ 3.0 years.
• Pre-pubertal status: Absent breast development in girls, testicular volume \< 4.0 mL in boys.
• Subjects with GHD (diagnosed according to the current diagnostic guidelines) who are receiving treatment with daily rhGH.
• 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.
• 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.
• Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
• Willingness to discontinue daily rhGH therapy.
• Legally authorized representatives must be willing and able to give informed consent

You may not qualify if:

• \. Prior (in the last 12 months) or concomitant treatment with a growth promoting agent other than rhGH \[e.g., IGF-I, GH releasing hormone (GHRH), sex steroids (except when used as primer for GH stimulation test), aromatase inhibitors and/or GnRH agonist\].
• \. 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.
• \. 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.).
• \. Birth weight and/or birth length less than 5th percentile for gestational age using local gestational age growth charts.
• \. Prolonged daily (\> 14 days) use of anti-inflammatory doses of oral glucocorticoids.
• \. 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.
• \. Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants.
• \. Significant abnormality in screening laboratory studies (as assessed by PI and medical monitor).
• \. Current social conditions which would prevent completion of study activities (e.g., planned family move to a distant location).
• \. 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.
• \. Unwillingness to provide consent for participation in all trial activities

Sponsors & Collaborators

• Versartis Inc.: lead

Study Sites (1)

Eric Humphriss

Menlo Park, California, 94025, United States

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

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; Endocrine System Diseases

Study Officials

• Will Charlton, MD

  Vesrartis

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 5, 2017
• First Posted: May 9, 2017
• Study Start: March 31, 2017
• Primary Completion: November 30, 2017
• Study Completion: November 30, 2017
• Last Updated: March 9, 2018

Record last verified: 2018-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)