A Long-Acting Growth Hormone (Somavaratan, VRS-317) in Childre
- Conditions
- Growth hormone deficiencyMedDRA version: 19.0Level: PTClassification code 10056438Term: Growth hormone deficiencySystem Organ Class: 10014698 - Endocrine disordersTherapeutic area: Diseases [C] - Hormonal diseases [C19]
- Registration Number
- EUCTR2016-002780-34-BE
- Lead Sponsor
- Versartis, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- All
- Target Recruitment
- 300
Inclusion Criteria for Subjects Completing a Previous Somavaratan Study:
1.Completion of a somavaratan clinical study in pediatric subjects with GHD.
2.Willing and able to comply with all study procedures.
Inclusion Criteria for New Treatment Naïve Subjects:
1.Chronological Age = 3.0 years.
2.Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
3.Diagnosis of GHD as documented by two or more GH stimulation test results = 10.0 ng/mL. The use of prior GH stimulation test results is permitted providing the stimulatory agents, GH assay and test results are approved in writing by the Medical Monitor. In localities with different diagnostic criteria, any child meeting the GH stimulation test criteria described here will be considered eligible.
4.Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism. Subjects requiring thyroxine replacement must be considered adequately treated by the PI and Medical Monitor.
5.Normal adrenal function (morning cortisol and/or local stimulation test) 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.
6.Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
7.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: 300
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 for Subjects Completing a Previous Somavaratan Study:
1.Withdrawal from a somavaratan clinical study in pediatric subjects with GHD.
2.Use of certain medications with potential to alter responses to the test product including, but not limited to, IGF-I, GH-releasing hormone, and chronic anti-inflammatory doses of glucocorticoids.
3.Presence of a significant medical condition if such a condition or its treatment can influence the response to study drug (e.g., diabetes, renal failure).
Exclusion Criteria for New Treatment Naïve Subjects:
1.Prior/concomitant 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.Current, significant disease (e.g., diabetes, cystic fibrosis, renal insufficiency). In all cases of concurrent disease, screening must be approved in writing by the Medical Monitor.
3.Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome (e.g., Russell Silver, Prader Willi, Turner, etc.). Unconfirmed or suspected genetic variants will be considered individually.
4.Birth weight and/or birth length less than 5th percentile for gestational age using gestational age growth charts.
5.Prolonged daily (>14 days) use of anti-inflammatory doses of oral glucocorticoids.
6.Prior history of malignancy.
7.Treatment with an investigational drug in the 30 days prior to screening.
8.Known allergy to constituents of the study drug formulation.
9.Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.
10.Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants.
11.Significant abnormality in screening studies (as assessed by PI and Medical Monitor).
12.Current social conditions which would prevent completion of study activities (e.g., planned family move to a distant location).
13.History of pancreatitis or undiagnosed chronic abdominal pain.
14.History of spinal or total body irradiation.
15.Other pituitary hormone deficiencies that are not properly treated.
16.Unwillingness to provide consent for participation in all trial activities.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Evaluate the safety of somavaratan during long-term treatment ;Secondary Objective: Evaluate changes in pharmacodynamic responses (IGF-I and IGFBP-3), bone age, weight, height velocity, height standard deviation scores, metabolic parameters, pubertal development and anti-drug antibody responses during long-term somavaratan treatment ;Timepoint(s) of evaluation of this end point: End of study.;Primary end point(s): The primary endpoint is safety as assessed 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.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary growth endpoints are the annual assessment of height velocity, changes in height SDS, body weight, body mass index, bone age and pubertal status.<br>Secondary PD endpoints include changes in IGF-I and IGFBP-3 expressed as concentrations and/or SD scores. These secondary endpoints may be further evaluated to determine potential differences in response due to age and gender, baseline conditions, as well as other measures.<br>;Timepoint(s) of evaluation of this end point: End of study.