Clinical trial NCT01718041: Versartis Trial in Children to Assess Long-Acting Growth Hormone
Official title: A Long-acting Human Growth Hormone (VRS-317) in Pre-pubertal Children With Growth Hormone Deficiency: A Randomized, Open-label, Multi-center, Phase 1b/2a Study of Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Efficacy After Subcutaneous Administration for 6 Months
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Pediatric Growth Hormone Deficiency
Keywords: Growth hormone
Growth hormone deficiency
Growth
Pediatric growth hormone deficiency
Long acting growth hormone
GHD
PGHD
VRS 317
Exten
Versartis
Lead sponsor: Aravive, Inc.
Sponsor class: INDUSTRY
First posted: 2012-10-31
Enrollment: 64
Sex: ALL
Minimum age (years): 3
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
timeFrame: 8 Months
Secondary outcomes:
measure: Evaluate pharmacokinetic measures of VRS-317.
timeFrame: 8 months
Other outcomes:
measure: Evaluate secondary efficacy measures after 6 months of VRS-317 dosing.
timeFrame: 6 Months
measure: Evaluate pharmacodynamic measures
timeFrame: 8 Months
Locations:
city: Clovis
country: United States
state: California
city: Los Angeles
country: United States
state: California
city: San Diego
country: United States
state: California
city: San Francisco
country: United States
state: California
city: Greenwood Village
country: United States
state: Colorado
city: Orlando
country: United States
state: Florida
city: Tallahassee
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
city: Indianapolis
country: United States
state: Indiana
city: Baltimore
country: United States
state: Maryland
city: Springfield
country: United States
state: Massachusetts
city: Minneapolis
country: United States
state: Minnesota
city: Kansas City
country: United States
state: Missouri
city: Livingston
country: United States
state: New Jersey
city: Morristown
country: United States
state: New Jersey
city: Buffalo
country: United States
state: New York
city: Mineola
country: United States
state: New York
city: Sleepy Hollow
country: United States
state: New York
city: Cleveland
country: United States
state: Ohio
city: Columbus
country: United States
state: Ohio
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
Study officials:
References: