A Long-Acting Growth Hormone (Somavaratan, VRS-317) in Childre

Phase 1

Conditions: Growth hormone deficiency
MedDRA version: 20.0Level: PTClassification code 10056438Term: Growth hormone deficiencySystem Organ Class: 10014698 - Endocrine disorders
Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Registration Number: EUCTR2016-002780-34-SE

Lead Sponsor: Versartis, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 400

Inclusion Criteria

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 and Switch 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.In Sweden, a brain MRI must be obtained during Screening to rule out any contraindications to rhGH treatment (must be obtained within 6 months prior to enrollment).
5.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.
6.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.
7.Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
8.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: 400
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

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 and Switch 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.