Clinical trial NCT02413138: Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)
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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Pediatric Growth Hormone Deficiency
Growth Disorders
Keywords: GHD
Lead sponsor: Aravive, Inc.
Sponsor class: INDUSTRY
First posted: 2015-04-09
Enrollment: 41
Sex: ALL
Minimum age (years): 3
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility 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

Primary outcomes:
measure: Efficacy (Annual Height velocity)
timeFrame: 12 months
Secondary outcomes:
measure: Pharmacodynamics (IGF-I responses to study drug administration)
timeFrame: 12 months
measure: Pharmacodynamics (IGFBP-3 responses to study drug administration)
timeFrame: 12 months
measure: Safety (Number of subjects with adverse events )
timeFrame: 12 months
measure: Safety (Concomitant medications)
timeFrame: 12 months
measure: Safety (Safety labs)
timeFrame: 12 months
measure: Safety (Vital signs)
timeFrame: 12 months
measure: Safety (Physical Exams)
timeFrame: 12 months
Other outcomes:
measure: Secondary Efficacy (Change in height SDS)
timeFrame: 12 months
measure: Secondary Efficacy
timeFrame: 12 months
measure: Secondary Efficacy
timeFrame: 12 months
measure: Secondary Efficacy (Change in bone age)
timeFrame: 12 months
measure: Secondary Efficacy (Change in pubertal staging.)
timeFrame: 12 months
Locations:
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
Study officials:
affiliation: Vesrartis
name: Will Charlton, MD
role: STUDY_DIRECTOR
References: