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

Growth Disorders Clinical Trial

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02413138
• Other study ID #: J14VR5
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: August 8, 2015
• Est. completion date: November 30, 2017

Study information

• Verified date: July 2022
• Source: Aravive, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 41
• Est. completion date: November 30, 2017
• Est. primary completion date: November 30, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 10 Years

Eligibility

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

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Disorders
• Pediatric Growth Hormone Deficiency

Intervention

Drug:
• Somavaratan (VRS-317)
Long acting recombinant human growth hormone

Locations

Country	Name	City	State
Japan	Hokkaido University Hospital	Sapporo

Sponsors (1)

Lead Sponsor	Collaborator
Versartis Inc.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Secondary Efficacy (Change in height SDS)	Change in height SDS.	12 months
Other	Secondary Efficacy	Change in body weight	12 months
Other	Secondary Efficacy	Change in body mass index.	12 months
Other	Secondary Efficacy (Change in bone age)	Change in bone age.	12 months
Other	Secondary Efficacy (Change in pubertal staging.)	Change in pubertal staging.	12 months
Primary	Efficacy (Annual Height velocity)	Annual Height velocity.	12 months
Secondary	Pharmacodynamics (IGF-I responses to study drug administration)	IGF-I responses to study drug administration.	12 months
Secondary	Pharmacodynamics (IGFBP-3 responses to study drug administration)	IGFBP-3 responses to study drug administration.	12 months
Secondary	Safety (Number of subjects with adverse events )	Number of subjects with adverse events (including repeat dose immunogenicity).	12 months
Secondary	Safety (Concomitant medications)	Concomitant medications	12 months
Secondary	Safety (Safety labs)	Safety labs	12 months
Secondary	Safety (Vital signs)	Vital signs	12 months
Secondary	Safety (Physical Exams)	Physical Exams	12 months