Versartis Trial in Children to Assess Long-Acting Growth Hormone - Full Text View

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:	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

Resource links provided by NLM:

Genetics Home Reference related topics: isolated growth hormone deficiency metatropic dysplasia pseudoachondroplasia

MedlinePlus related topics: Hormones

Genetic and Rare Diseases Information Center resources: Growth Hormone Deficiency Isolated Growth Hormone Deficiency Hypopituitarism

U.S. FDA Resources

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 ]
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 ]
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 ]
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 ]
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.


Enrollment:	64
Study Start Date:	October 2012
Study Completion Date:	July 2014
Primary Completion Date:	July 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 (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

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


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:	August 5, 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

ClinicalTrials.gov processed this record on August 23, 2017

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