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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 136

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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