A Long-Acting Growth Hormone, Somavaratan (VRS-317), in Children compared to Daily rhGH

• Register: EUCTR
• Last refreshed on: 30 October 2017
• Main ID: EUCTR2014-004525-41-SE
• Date of registration: 12/02/2015
• Prospective Registration: Yes
• Primary sponsor: Versartis, Inc.
• Public title: A Long-Acting Growth Hormone, Somavaratan (VRS-317), in Children compared to Daily rhGH
• Scientific title: Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children with Growth Hormone Deficiency. - VELOCITY
• Date of first enrolment: 10/04/2015
• Target sample size: 136
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-004525-41
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Canada, Netherlands, Poland, Sweden, United States

Contacts

• Name: Global Project Manager
• Affiliation: ResearchPoint Global

• Name: Global Project Manager
• Affiliation: ResearchPoint Global

Key inclusion & exclusion criteria

Inclusion criteria:
1. Chronological Age = 3.0 years and = 10.0 (girls) and = 11.0 (boys).
2. Pre-pubertal status: Absent breast development in girls, testicular volume <4.0 mL in boys.
3. Diagnosis of GHD as documented two or more GH stimulation test results = 10.0 ng/mL.
4. Height SDS = -2.0 at screening.
5. Weight for Stature = 10th percentile.
6. IGF-I SD score = -1.0 at screening.
7. Delayed bone age (= 6 months as determined by the central reader). Left hand X-Ray must be obtained within 90 days of screening visit or during screening.
8. Normal thyroid function test results at screening visit (or a minimum of four weeks of thyroxine replacement therapy prior to study drug administration).
9. Available adrenal function test results at screening visit (or in the preceding 6 months) in all subjects without a minimum of four weeks glucocorticoid replacement therapy prior to study drug administration.
10. Pathology relating to cause of GH deficiency must be stable for at least 6 months prior to screening.
11. Legally authorized representatives must be willing and able to give informed
consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 136
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Prior treatment with any growth promoting agent (e.g., GH, IGF-I, GH releasing hormone (GHRH), gonadotrophins, sex steroids). Up to 10 day exposures to a growth promoting agent for diagnostic purposes are permitted if administered 30 or more days prior to screening.
2. Documented history of, or current, significant disease.
3. Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
4. Birth weight and/or birth length less than 5th percentile for gestational age using gestational age growth charts.
5. A diagnosis of Attention Deficit Hyperactivity Disorder (ADHD), use of ADHD
medications or a likelihood of starting ADHD medications during study participation.
6. Daily use of anti-inflammatory doses of glucocorticoid.
7. Prior history of leukemia, lymphoma, sarcoma or cancer.
8. Treatment with an investigational drug in the 30 days prior to screening.
9. Known allergy to constituents of the study drug formulation.
10. Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.
11. Significant spinal abnormalities including scoliosis, kyphosis and spina bifida
variants.
12. Significant abnormality in screening laboratory studies.
13. Current social conditions which would prevent completion of study activities
(e.g., planned family move to a distant location).
14. History of pancreatitis or undiagnosed chronic abdominal pain.
15. History of spinal or total body irradiation.
16. Subjects with other pituitary hormone deficiency who are not treated properly.
17. Unwillingness to provide consent for participation in all trial activities.
18. Unwillingness to accept dose assignments.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Growth hormone deficiency
MedDRA version: 19.0 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders

Intervention(s)

Product Name: Somavaratan (VRS-317)
Pharmaceutical Form: Injection
INN or Proposed INN: VRS-317
CAS Number: 1448335-08-7
Other descriptive name: VRS-317
Concentration unit: µg/kg microgram(s)/kilogram
Concentration type: range
Concentration number: 25-34

Pharmaceutical Form: Injection
INN or Proposed INN: Recombinat human growth hormone
Other descriptive name: RECOMBINANT HUMAN GROWTH HORMONE
Concentration unit: µg/kg microgram(s)/kilogram
Concentration type: equal
Concentration number: 34-

Primary Outcome(s)

Main Objective: Compare the safety and efficacy of subcutaneous VRS-317 and daily rhGH during 12 months of treatment.

Timepoint(s) of evaluation of this end point: End of study.

Secondary Objective: Evaluate and compare changes in pharmacodynamic responses (IGF-I, IGF binding protein-3 (IGFBP-3), growth hormone binding protein (GHBP) and acid labile subunit (ALS)), bone age, weight, body mass index, height standard deviation scores, pubertal development and anti-drug antibody responses.

Primary end point(s): Annual height velocity after 12 months continous treatment with either VRS-317 or daily rhGH.

Secondary Outcome(s)

Secondary end point(s): - IGF-1 and IGFBP-3 responses to study drug administration.
- Change in height SDS.
- Change in bone age.
- Change in body weight.
- Change in body mass index.

Timepoint(s) of evaluation of this end point: End of study.

Secondary ID(s)

14VR4

Source(s) of Monetary Support

Versartis, Inc.