Versartis Trial in Children to Assess Long-Acting Growth Hormone (VERTICAL)

Versartis

Status and phase: Completed; Phase 2; Phase 1

Conditions: Pediatric Growth Hormone Deficiency

Treatments: Drug: Experimental: VRS-317

Study type: Interventional

Funder types: Industry

Identifiers

NCT01718041

VRS-317 Protocol 12VR2

Details and patient eligibility

About

This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. During the Phase 2a stage, patients will receive 6 months of VRS-317 treatment at dose levels selected from the Phase 1b stage. The primary endpoints for the study are to determine the safety and efficacy of repeat dose VRS-317.

Full description

In Phase 1b, separate cohorts of patients will be tested in a single ascending dose format. Safety review committee meetings will take place prior to escalating to each increasing dose level. Enrolled patients will be monitored for 60 days for safety with PK/PD and safety labs collected.

Two dose levels will be selected after completion of Phase 1b to be tested during Phase 2a (6 months of continuous VRS-317 treatment). Safety and PK assessments will be made during the Phase 2a stage. Patient heights will be measured by stadiometer.

Enrollment: 64 patients

Sex: All

Ages: 3 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Chronological Age ≥ 3.0 years and ≤ 11.0
• Diagnosis of GHD as documented by GH stimulation test
• Below average height SDS at screening
• Appropriate weight for Stature
• Decreased IGF-I SDS at screening
• Delayed bone age
• Normal thyroid function test results at screening visit
• Legally authorized representative informed consent.

Exclusion criteria

• Prior treatment with any growth promoting agent
• Documented history of, or current, significant disease
• Chromosomal aneuploidy, significant gene mutations
• Diagnosis of Attention Deficit Hyperactivity Disorder
• Daily use of anti-inflammatory doses of glucocorticoid
• Prior history of leukemia, lymphoma, sarcoma or cancer
• Known allergy to constituents of the study drug formulation
• Abnormal ocular findings at screening
• Significant abnormality in screening laboratory studies

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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64 participants in 1 patient group

VRS-317

Experimental group

Description:

Active treatment arm

Treatment:

Drug: Experimental: VRS-317