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Versartis Long-Term Safety Study of Somavaratan (VISTA)

Primary Purpose

Pediatric Growth Hormone Deficiency

Status

Terminated

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

somavaratan

About this trial

This is an interventional treatment trial for Pediatric Growth Hormone Deficiency focused on measuring Growth Hormone Deficiency, Pediatric Growth Hormone Deficiency, GHD, PGHD, VRS-317, Versartis, Growth disorder, Long acting growth hormone, Growth hormone, Weekly growth hormone dosing, Semi-monthly growth hormone dosing, Monthly growth hormone dosing, IGF-I, Short stature, Height velocity, Annual height velocity, Growth rate, Somavaratan

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria for Subjects Completing a Previous Somavaratan Study:

Completion of a somavaratan clinical study.
Willing and able to comply with all study procedures.

Exclusion Criteria for Subjects Completing a Previous somavaratan Study:

Withdrawal from a somavaratan clinical study.
Use of certain medications with potential to alter responses to the test product.
Presence of a significant medical condition.

Inclusion Criteria for New Treatment Naïve Subjects:

Chronological Age ≥ 3.0 years.
Pre-pubertal status.
Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL.
Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism.
Normal adrenal function at Screening Visit or within 6 months of the Screening Visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration.
Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
Legally authorized representatives must be willing and able to give informed consent.

Exclusion Criteria for New Treatment Naïve Subjects:

Prior/concomitant treatment with any growth promoting agent.
Current, significant disease.
Chromosomal aneuploidy, significant gene mutations or confirmed diagnosis of a named syndrome.
Birth weight and/or birth length less than 5th percentile for gestational age.
Prolonged daily use of anti-inflammatory doses of oral glucocorticoids.
Prior history of malignancy.
Treatment with an investigational drug in the 30 days prior to screening.
Known allergy to constituents of the study drug formulation.
Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.
Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants.
Significant abnormality in screening studies.
History of pancreatitis or undiagnosed chronic abdominal pain.
History of spinal or total body irradiation.
Other pituitary hormone deficiencies that are not properly treated.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Treatment naive subjects with GHD

Subjects who have completed a somavaratan study

Arm Description

Up to 100 new treatment naïve subjects with GHD will receive somavaratan 3.5mg/kg twice monthly.

All subjects after participation in (12VR2) or participation in the 14VR4 protocols have the option to receive somavaratan 3.5mg/kg twice monthly.

Outcomes

Primary Outcome Measures

Safety

Safety assessments include recording of adverse events and concomitant medications, monitoring of injection sites, vital signs and clinical laboratory determinations.

Secondary Outcome Measures

Full Information

NCT ID

NCT02068521

First Posted

February 6, 2014

Last Updated

March 7, 2018

Sponsor

Versartis Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02068521

Brief Title

Versartis Long-Term Safety Study of Somavaratan

Acronym

VISTA

Official Title

An Open-Label, Long-Term Safety Study of A Long-acting Human Growth Hormone Somavaratan (VRS-317) in Children With Growth Hormone Deficiency

Study Type

Interventional

2. Study Status

Record Verification Date

March 2018

Overall Recruitment Status

Terminated

Why Stopped

The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved

Study Start Date

March 3, 2014 (Actual)

Primary Completion Date

November 17, 2017 (Actual)

Study Completion Date

November 17, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Versartis Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study. Safety will be monitored by physical examination, inspection of injection sites, vital signs, clinical laboratory determinations (including fasting glucose, insulin, and lipids), 12-lead ECGs (for new treatment naïve subjects and subjects not previously exposed to somavaratan), PK/PD assessments, and immunogenicity assessments. Adverse events (AEs) and concomitant medications will be captured. AEs will be coded using CTCAE v 4.0. AEs will be coded using the MedDRA dictionary and CMs using the WHO Drug dictionary.

Detailed Description

Protocol 13VR3 is a multi-center, open-label study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan study in children with growth hormone deficiency (GHD), as well as up to 100 new treatment naïve subjects with GHD. All subjects will receive somavaratan 3.5 mg/kg twice monthly. The study will be conducted at approximately 70 Pediatric Endocrinology centers in the United States, Canada, and Europe.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pediatric Growth Hormone Deficiency

Keywords

Growth Hormone Deficiency, Pediatric Growth Hormone Deficiency, GHD, PGHD, VRS-317, Versartis, Growth disorder, Long acting growth hormone, Growth hormone, Weekly growth hormone dosing, Semi-monthly growth hormone dosing, Monthly growth hormone dosing, IGF-I, Short stature, Height velocity, Annual height velocity, Growth rate, Somavaratan

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

385 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment naive subjects with GHD

Arm Type

Experimental

Arm Description

Up to 100 new treatment naïve subjects with GHD will receive somavaratan 3.5mg/kg twice monthly.

Arm Title

Subjects who have completed a somavaratan study

Arm Type

Experimental

Arm Description

All subjects after participation in (12VR2) or participation in the 14VR4 protocols have the option to receive somavaratan 3.5mg/kg twice monthly.

Intervention Type

Drug

Intervention Name(s)

somavaratan

Other Intervention Name(s)

VRS-317

Intervention Description

Subcutaneous injection

Primary Outcome Measure Information:

Title

Safety

Description

Safety assessments include recording of adverse events and concomitant medications, monitoring of injection sites, vital signs and clinical laboratory determinations.

Time Frame

Up to 4 years

Other Pre-specified Outcome Measures:

Title

Evaluate the changes in pharmacodynamics (PD) responses, bone age, weight, height velocity, height standard deviation scores, metabolic parameters, pubertal development and anti-drug antibody responses during long-term somavaratan treatment

Description

PK/PD peak and trough measurements will be assessed throughout the study with assessment of plasma VRS-317 concentrations and IGF-I and its binding proteins measured at pre-specified time points.

Time Frame

Up to 4 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria for Subjects Completing a Previous Somavaratan Study: Completion of a somavaratan clinical study. Willing and able to comply with all study procedures. Exclusion Criteria for Subjects Completing a Previous somavaratan Study: Withdrawal from a somavaratan clinical study. Use of certain medications with potential to alter responses to the test product. Presence of a significant medical condition. Inclusion Criteria for New Treatment Naïve Subjects: Chronological Age ≥ 3.0 years. Pre-pubertal status. Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL. Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism. Normal adrenal function at Screening Visit or within 6 months of the Screening Visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration. Pathology relating to cause of GHD must be stable for at least 6 months prior to screening. Legally authorized representatives must be willing and able to give informed consent. Exclusion Criteria for New Treatment Naïve Subjects: Prior/concomitant treatment with any growth promoting agent. Current, significant disease. Chromosomal aneuploidy, significant gene mutations or confirmed diagnosis of a named syndrome. Birth weight and/or birth length less than 5th percentile for gestational age. Prolonged daily use of anti-inflammatory doses of oral glucocorticoids. Prior history of malignancy. Treatment with an investigational drug in the 30 days prior to screening. Known allergy to constituents of the study drug formulation. Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening. Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants. Significant abnormality in screening studies. History of pancreatitis or undiagnosed chronic abdominal pain. History of spinal or total body irradiation. Other pituitary hormone deficiencies that are not properly treated.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Will Charlton, MD

Organizational Affiliation

Versartis Inc.

Official's Role

Study Director

12. IPD Sharing Statement

Links:

URL

http://versartis.com

Description

Versartis website

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Versartis Long-Term Safety Study of Somavaratan

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