Long-Acting Growth Hormone in Children Compared to Daily rhGH

NCT02339090 | Completed Phase 3 Results DMC

• 1 other identifier: 14VR4
• Study Type: interventional
• Target: 138
• Locations: 0 countries
• Sites: N/A

Brief Summary

The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.

Conditions

Growth Disorders

Keywords

VRS-317; Growth Hormone Deficiency; Long Acting Recombinant Growth Hormone; Long Acting Growth Hormone; Pediatric Growth Hormone Deficiency; Growth Hormone Replacement Therapy; Versartis; Xten; Human Growth Hormone; somavaratan; growth failure

Outcome Measures

Primary Outcomes (1)

• Annual Height Velocity

  Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) \* 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.

  12 months

Secondary Outcomes (7)

• Change From Baseline in Height Standard Deviation Score (SDS) at Month 12

  Baseline, Month 12

• Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader

  Baseline, Month 12

• Change From Baseline in Body Mass Index (BMI) at Month 12

  Baseline, Month 12

• Change From Baseline in Body Weight at Month 12

  Baseline, Month 12

• Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12

  Baseline, Month 12

Study Arms (2)

Somavaratan

EXPERIMENTAL

Participants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months.

Drug: Somavaratan

rhGH

ACTIVE COMPARATOR

Participants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months.

Drug: rhGH

Interventions

Somavaratan DRUG

Somavaratan will be administered per dose and schedule specified in the arm description.

Also known as: Long-acting recombinant human growth hormone, VRS-317

Somavaratan

rhGH DRUG

rhGH will be administered per dose and schedule specified in the arm description.

Also known as: daily growth hormone, recombinant growth hormone therapy

rhGH

Eligibility Criteria

• Age: 3 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
• Pre-pubertal status: Absent breast development in girls, testicular volume \< 4.0 mL in boys.
• Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
• Height standard deviation score (SDS) ≤ -2.0 at screening.
• Weight for Stature ≥ 10th percentile.
• Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening.
• Delayed bone age (≥ 6 months).

You may not qualify if:

• Prior treatment with any growth promoting agent
• History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
• Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
• A diagnosis of Attention Deficit Hyperactivity Disorder.
• Daily use of anti-inflammatory doses of glucocorticoid.
• Prior history of leukemia, lymphoma, sarcoma or cancer.
• Treatment with an investigational drug in the 30 days prior to screening.
• Known allergy to constituents of the study drug formulation.
• 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

Sponsors & Collaborators

• Versartis Inc.: lead

MeSH Terms

Conditions

Growth Disorders; Dwarfism, Pituitary; Failure to Thrive

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms; Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases; Signs and Symptoms

Results Point of Contact

• Title: Chief Operating Officer
• Organization: Aravive, Inc.

clinicaltrials@aravive.com

(936) 355-1910

Study Officials

• Will Charlton, MD

  Sponsor GmbH

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2015
• First Posted: January 15, 2015
• Study Start: August 26, 2015
• Primary Completion: August 23, 2017
• Study Completion: August 23, 2017
• Last Updated: December 30, 2022
• Results First Posted: December 30, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share