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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Pediatric Growth Hormone Deficiency	Drug: Experimental: VRS-317	Phase 1; Phase 2

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	64 participants
Intervention Model:	Single Group Assignment
Masking:	None (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
Study Start Date :	October 2012
Actual Primary Completion Date :	July 2014
Actual Study Completion Date :	July 2014

Arms and Interventions

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Arm	Intervention/treatment
Experimental: VRS-317; Active treatment arm	Drug: Experimental: VRS-317; Active treatment arm

Outcome Measures

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Primary Outcome Measures :

1. 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 ]

   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 :

1. Evaluate pharmacokinetic measures of VRS-317. [ Time Frame: 8 months ]
   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:

1. Evaluate secondary efficacy measures after 6 months of VRS-317 dosing. [ Time Frame: 6 Months ]
   Secondary efficacy measures during Phase 2a include change in height SDS and change in bone age after 6 months VRS-317 treatment.
2. Evaluate pharmacodynamic measures [ Time Frame: 8 Months ]
   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.

Eligibility Criteria

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	3 Years to 11 Years (Child)
Sexes Eligible for Study:	All
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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

  Show 23 Study Locations

Sponsors and Collaborators

Versartis Inc.

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Versartis Inc.
ClinicalTrials.gov Identifier:	NCT01718041 History of Changes
Other Study ID Numbers:	VRS-317 Protocol 12VR2
First Posted:	October 31, 2012
Last Update Posted:	August 9, 2016
Last Verified:	August 2016

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