NCT02068521

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
385

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Mar 2014

Typical duration for phase_2

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 6, 2014

Completed
15 days until next milestone

First Posted

Study publicly available on registry

February 21, 2014

Completed
10 days until next milestone

Study Start

First participant enrolled

March 3, 2014

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 17, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 17, 2017

Completed
Last Updated

March 9, 2018

Status Verified

March 1, 2018

Enrollment Period

3.7 years

First QC Date

February 6, 2014

Last Update Submit

March 7, 2018

Conditions

Pediatric Growth Hormone Deficiency

Keywords

Growth Hormone DeficiencyPediatric Growth Hormone DeficiencyGHDPGHDVRS-317VersartisGrowth disorderLong acting growth hormoneGrowth hormoneWeekly growth hormone dosingSemi-monthly growth hormone dosingMonthly growth hormone dosingIGF-IShort statureHeight velocityAnnual height velocityGrowth rateSomavaratan

Outcome Measures

Primary Outcomes (1)

  • Safety

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

    Up to 4 years

Other Outcomes (1)

  • 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

    Up to 4 years

Study Arms (2)

Treatment naive subjects with GHD

EXPERIMENTAL

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

Drug: somavaratan

Subjects who have completed a somavaratan study

EXPERIMENTAL

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

Drug: somavaratan

Interventions

somavaratanDRUG

Subcutaneous injection

Also known as: VRS-317
Subjects who have completed a somavaratan studyTreatment naive subjects with GHD

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • 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.
  • 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.
  • 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.
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Links

MeSH Terms

Conditions

Dwarfism, PituitaryGrowth DisordersDwarfism

Condition Hierarchy (Ancestors)

Bone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesBone Diseases, EndocrineHypopituitarismPituitary DiseasesHypothalamic DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEndocrine System DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Will Charlton, MD

    Versartis Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 6, 2014

First Posted

February 21, 2014

Study Start

March 3, 2014

Primary Completion

November 17, 2017

Study Completion

November 17, 2017

Last Updated

March 9, 2018

Record last verified: 2018-03