VELOCITY: Versartis Long-Acting Growth Hormone in Children Compared to Daily rhGH

Sponsor

Versartis Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT02339090

Collaborator

(none)

136

Enrollment

Arms

Actual Duration (Months)

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Growth Disorders	Drug: somavaratan Drug: Daily rhGH	Phase 3

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

136 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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.

Actual Study Start Date :

Aug 24, 2015

Actual Primary Completion Date :

Aug 23, 2017

Actual Study Completion Date :

Aug 23, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: somavaratan somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly	Drug: somavaratan long acting growth hormone therapy Other Names: Long acting recombinant human growth hormone
Active Comparator: Daily rhGH Daily recombinant growth hormone therapy administered subcutaneously every day	Drug: Daily rhGH daily growth hormone therapy Other Names: daily growth hormone recombinant growth hormone therapy

Arm

Intervention/Treatment

Experimental: somavaratan

somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly

Drug: somavaratan

long acting growth hormone therapy

Other Names:

Long acting recombinant human growth hormone

Active Comparator: Daily rhGH

Daily recombinant growth hormone therapy administered subcutaneously every day

Drug: Daily rhGH

daily growth hormone therapy

Other Names:

daily growth hormone

recombinant growth hormone therapy

Outcome Measures

Primary Outcome Measures

Efficacy (Annual height velocity) [12 months]
Annual height velocity

Secondary Outcome Measures

Pharmacodynamics (IGF-I and IGFBP-3 responses to study drug administration) [12 months]
IGF-I and IGFBP-3 responses to study drug administration
Safety as measured by he number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, and repeat dose immunogenicity. [12 Months]
Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, and repeat dose immunogenicity.

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 11 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL.
Height SD score ≤ -2.0 at screening.
Weight for Stature ≥ 10th percentile.
IGF-I SD score ≤ -1.0 at screening.
Delayed bone age (≥ 6 months).

Exclusion Criteria:

Prior treatment with any growth promoting agent
History of or concurrent significant disease (e.g. 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