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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Versartis Inc.
ClinicalTrials.gov Identifier:
NCT01718041
First received: October 19, 2012
Last updated: March 10, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Pediatric Growth Hormone Deficiency
Drug: Experimental: VRS-317
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Versartis Inc.:

Primary Outcome Measures:
  • 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 [ Time Frame: 8 Months ] [ Designated as safety issue: Yes ]

    Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a).

    The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.



Secondary Outcome Measures:
  • Evaluate pharmacokinetic measures of VRS-317. [ Time Frame: 8 months ] [ Designated as safety issue: Yes ]
    Secondary outcome measures include evaluation of pharmacokinetics (PK) of VRS-317 including Cmax, Tmax and AUC. PK measures will be analysed at multiple timepoints after single dosing of VRS-317 during Phase 1b and after repeat dosing of VRS-317 during Phase 2a.


Other Outcome Measures:
  • Evaluate secondary efficacy measures after 6 months of VRS-317 dosing. [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
    Secondary efficacy measures during Phase 2a include change in height SDS and change in bone age after 6 months VRS-317 treatment.

  • Evaluate pharmacodynamic measures [ Time Frame: 8 Months ] [ Designated as safety issue: Yes ]
    Secondary outcome measures include evaluation of pharmacodynamics (PD) as measured by IGF-I and IGFBP-3 responses to single dose VRS-317 during Phase 1b and as measured by IGF-I and IGFBP-3 responses to multiple doses of VRS-317 after repeat dosing during Phase 2a. PD measures will be obtained at multiple timepoints during Phase 1b and Phase 2a.


Estimated Enrollment: 48
Study Start Date: October 2012
Estimated Study Completion Date: September 2014
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VRS-317
Active treatment arm
Drug: Experimental: VRS-317
Active treatment arm

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

Ages Eligible for Study:   3 Years to 11 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01718041

  Show 23 Study Locations
Sponsors and Collaborators
Versartis Inc.
  More Information

No publications provided

Responsible Party: Versartis Inc.
ClinicalTrials.gov Identifier: NCT01718041     History of Changes
Other Study ID Numbers: VRS-317 Protocol 12VR2
Study First Received: October 19, 2012
Last Updated: March 10, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Versartis Inc.:
Growth hormone
Growth hormone deficiency
Growth
Pediatric growth hormone deficiency
Long acting growth hormone
GHD
PGHD
VRS 317
Exten
Versartis

Additional relevant MeSH terms:
Endocrine System Diseases
Dwarfism, Pituitary
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 16, 2014