Clinical trial NCT02339090: Long-Acting Growth Hormone in Children Compared to Daily rhGH
Official title: Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Growth Disorders
Keywords: VRS-317
Growth Hormone Deficiency
Long Acting Recombinant Growth Hormone
Long Acting Growth Hormone
Pediatric Growth Hormone Deficiency
Growth Hormone Replacement Therapy
Versartis
Xten
Human Growth Hormone
somavaratan
growth failure
Lead sponsor: Aravive, Inc.
Sponsor class: INDUSTRY
First posted: 2015-01-15
Enrollment: 138
Sex: ALL
Minimum age (years): 3
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.

Detailed description: This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. The primary endpoint is height velocity at 12 months.

Eligibility criteria: Inclusion Criteria:

* Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
* Pre-pubertal status: Absent breast development in girls, testicular volume \< 4.0 mL in boys.
* Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
* Height standard deviation score (SDS) ≤ -2.0 at screening.
* Weight for Stature ≥ 10th percentile.
* Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening.
* Delayed bone age (≥ 6 months).

Exclusion Criteria:

* Prior treatment with any growth promoting agent
* History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
* Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
* A diagnosis of Attention Deficit Hyperactivity Disorder.
* Daily use of anti-inflammatory doses of glucocorticoid.
* Prior history of leukemia, lymphoma, sarcoma or cancer.
* Treatment with an investigational drug in the 30 days prior to screening.
* Known allergy to constituents of the study drug formulation.
* 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: Annual Height Velocity
timeFrame: 12 months
Secondary outcomes:
measure: Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
timeFrame: Baseline, Month 12
measure: Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
timeFrame: Baseline, Month 12
measure: Change From Baseline in Body Mass Index (BMI) at Month 12
timeFrame: Baseline, Month 12
measure: Change From Baseline in Body Weight at Month 12
timeFrame: Baseline, Month 12
measure: Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
timeFrame: Baseline, Month 12
measure: Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
timeFrame: Baseline, Month 12
measure: Number of Participants With Adverse Events (AEs)
timeFrame: Baseline up to Month 12
Other outcomes:
Locations:
Study officials:
affiliation: Sponsor GmbH
name: Will Charlton, MD
role: STUDY_DIRECTOR
References: