Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)

• Sponsor: Versartis Inc. (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT02413138
• Collaborator: (none)
• Enrollment: 41
• Location: 1
• Arms: 2
• Actual Duration (Months): 27.8
• Patients Per Site Per Month: 1.5

Study Details

Study Description

Brief Summary

The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.

Condition or Disease	Intervention/Treatment	Phase
Pediatric Growth Hormone Deficiency; Growth Disorders	Drug: Somavaratan (VRS-317)	Phase 2/Phase 3

Detailed Description

The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.

Study Design

• Study Type: Interventional
• Actual Enrollment: 41 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)
• Actual Study Start Date: Aug 8, 2015
• Actual Primary Completion Date: Nov 30, 2017
• Actual Study Completion Date: Nov 30, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Phase 2: Somavaratan (VRS-317) Active treatment arm	Drug: Somavaratan (VRS-317); Long acting recombinant human growth hormone; Other Names: Long acting recombinant human growth hormone
Experimental: Phase 3: Somavaratan (VRS-317) Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly	Drug: Somavaratan (VRS-317); Long acting recombinant human growth hormone; Other Names: Long acting recombinant human growth hormone

Outcome Measures

Primary Outcome Measures

1. Efficacy (Annual Height velocity) [12 months]

   Annual Height velocity.

Secondary Outcome Measures

1. Pharmacodynamics (IGF-I responses to study drug administration) [12 months]

   IGF-I responses to study drug administration.

2. Pharmacodynamics (IGFBP-3 responses to study drug administration) [12 months]

   IGFBP-3 responses to study drug administration.

3. Safety (Number of subjects with adverse events ) [12 months]

   Number of subjects with adverse events (including repeat dose immunogenicity).

4. Safety (Concomitant medications) [12 months]

   Concomitant medications

5. Safety (Safety labs) [12 months]

   Safety labs

6. Safety (Vital signs) [12 months]

   Vital signs

7. Safety (Physical Exams) [12 months]

   Physical Exams

Other Outcome Measures

1. Secondary Efficacy (Change in height SDS) [12 months]

   Change in height SDS.

2. Secondary Efficacy [12 months]

   Change in body weight

3. Secondary Efficacy [12 months]

   Change in body mass index.

4. Secondary Efficacy (Change in bone age) [12 months]

   Change in bone age.

5. Secondary Efficacy (Change in pubertal staging.) [12 months]

   Change in pubertal staging.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 3 Years to 10 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)

• Pre-pubertal status

• Diagnosis of GHD as documented by two or more GH stimulation test results

• Height SD score ≤ -2.0 at screening

• Weight for Stature ≥ 10th percentile

• IGF-I SD score ≤ -1.0 at screening

• Delayed bone age

Exclusion Criteria:

• Prior treatment with any growth promoting agent

• History of, or current, significant disease

• Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome

• Birth weight and/or birth length less than 5th percentile for gestational age

• A diagnosis of Attention Deficit Hyperactivity Disorder

• Daily use of anti-inflammatory doses of glucocorticoid

• Prior history of leukemia, lymphoma, sarcoma or cancer

• Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening

• Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants

• Significant abnormality in screening laboratory studies

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hokkaido University Hospital	Sapporo		Japan

Sponsors and Collaborators

• Versartis Inc.

Investigators

• Study Director: Will Charlton, MD, Vesrartis

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Versartis Inc.
• ClinicalTrials.gov Identifier: NCT02413138
• Other Study ID Numbers: J14VR5
• First Posted: Apr 9, 2015
• Last Update Posted: Jul 25, 2022
• Last Verified: Jul 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Versartis Inc.: GHD
• Additional relevant MeSH terms: Dwarfism, Pituitary; Endocrine System Diseases; Growth Disorders; Hormones

Study Results

No Results Posted as of Jul 25, 2022