Overview

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

Status:
Terminated
Trial end date:
2017-11-30
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 2/Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Versartis Inc.
Treatments:
Hormones
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