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

Versartis

Status and phase

Terminated

Phase 3

Phase 2

Conditions

Growth Disorders

Pediatric Growth Hormone Deficiency

Treatments

Drug: Somavaratan (VRS-317)

Study type

Interventional

Funder types

Industry

Identifiers

NCT02413138

J14VR5

Details and patient eligibility

About

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.

Full 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.

Enrollment

41 patients

Sex

All

Ages

3 to 10 years old

Volunteers

No Healthy Volunteers

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