A Phase 3 interventional study of Somavaratan and rhGH in Growth Disorders, sponsored by Versartis Inc.. Completed. Open to participants aged 3 Years to 11 Years. Per ClinicalTrials.gov, last updated 2022-12-30.
Sponsored by Versartis Inc. · Phase 3, Interventional, and Treatment
The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. The primary endpoint is height velocity at 12 months.
172 studies on the registry are indexed under Growth Disorders; 24 are open to participants now.
This study's enrollment of 138 is below the median of 283 across 98 interventional studies indexed under Growth Disorders.
Browse Growth Disorders studies →Versartis Inc. is the lead sponsor of 8 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Participants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months.
Drug: Somavaratan
Participants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months.
Drug: rhGH
Somavaratan will be administered per dose and schedule specified in the arm description.
Also known as: Long-acting recombinant human growth hormone, VRS-317
rhGH will be administered per dose and schedule specified in the arm description.
Also known as: daily growth hormone, recombinant growth hormone therapy
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.
Time frame: 12 months
Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
Height SDS was determined using the Center for Disease Control (CDC) Clinical Growth Charts; 2000. The SD score was calculated as the participant's height value minus the mean divided by the standard deviation (SD). The mean and the SD vary depending on the age and sex of the participant. Mean change from baseline in height SDS at Month 12 is presented.
Time frame: Baseline, Month 12
Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
Bone age was assessed from a radiograph of the left hand and wrist by central reader.
Time frame: Baseline, Month 12
Change From Baseline in Body Mass Index (BMI) at Month 12
The BMI is a person's weight in kilograms (kg) divided by the square of height in meters.
Time frame: Baseline, Month 12
Change From Baseline in Body Weight at Month 12
Body weight measured in light clothing and without shoes.
Time frame: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
The SD score was calculated as the actual value of IGF-I minus mean reference value of IGF-1 divided by reference standard deviation of IGF-I. The mean and the SD vary depending on the age and sex of the participant. Change in IGF-I level (SD score) at Month 12 from Baseline was assessed. A higher score reflects a better outcome.
Time frame: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
Time frame: Baseline, Month 12
Number of Participants With Adverse Events (AEs)
An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Time frame: Baseline up to Month 12
| Milestone | Somavaratan | rhGH |
|---|---|---|
| Started | 104 | 34 |
| Received a least 1 dose of study drug | 104 | 32 |
| Completed | 98 | 29 |
| Not completed | 6 | 5 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Lost to follow-up | 0 | 1 |
| Withdrew: Non-compliance with study drug | 0 | 1 |
| Withdrew: Withdrawal by subject | 2 | 1 |
| Withdrew: Poor growth | 3 | 0 |
| Withdrew: Randomized but not treated | 0 | 2 |
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.
| cm/year | Somavaratan | rhGH |
|---|---|---|
| Annual Height Velocity | 9.43 ± 0.28 | 10.70 ± 0.48 |
Height SDS was determined using the Center for Disease Control (CDC) Clinical Growth Charts; 2000. The SD score was calculated as the participant's height value minus the mean divided by the standard deviation (SD). The mean and the SD vary depending on the age and sex of the participant. Mean change from baseline in height SDS at Month 12 is presented.
| SD score | Somavaratan | rhGH |
|---|---|---|
| Change From Baseline in Height Standard Deviation Score (SDS) at Month 12 | 0.8 ± 0.53 | 1.0 ± 0.51 |
Bone age was assessed from a radiograph of the left hand and wrist by central reader.
| months | Somavaratan | rhGH |
|---|---|---|
| Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader | 1.1 ± 0.47 | 1.3 ± 0.55 |
The BMI is a person's weight in kilograms (kg) divided by the square of height in meters.
| kg/m^2 | Somavaratan | rhGH |
|---|---|---|
| Change From Baseline in Body Mass Index (BMI) at Month 12 | 1.1 ± 0.86 | -0.1 ± 0.95 |
Body weight measured in light clothing and without shoes.
| kg | Somavaratan | rhGH |
|---|---|---|
| Change From Baseline in Body Weight at Month 12 | 4.9 ± 1.87 | 3.8 ± 1.64 |
The SD score was calculated as the actual value of IGF-I minus mean reference value of IGF-1 divided by reference standard deviation of IGF-I. The mean and the SD vary depending on the age and sex of the participant. Change in IGF-I level (SD score) at Month 12 from Baseline was assessed. A higher score reflects a better outcome.
| SD score | Somavaratan | rhGH |
|---|---|---|
| Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12 | 0.9 ± 0.99 | 1.8 ± 0.72 |
| nanomoles (nmol)/milliliter (mL) | Somavaratan | rhGH |
|---|---|---|
| Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12 | 32.2 ± 30.2 | 49.8 ± 19.1 |
An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
| Participants | Somavaratan | rhGH |
|---|---|---|
| Number of Participants With Adverse Events (AEs) | 80 | 22 |
Collected over Baseline up to Month 12. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Somavaratan | 0/104 (0%) | 6/104 (5.8%) | 59/104 (56.7%) |
| rhGH | 0/32 (0%) | 0/32 (0%) | 16/32 (50%) |
| Event | Somavaratan | rhGH |
|---|---|---|
| Arnold-Chiari malformationCongenital, familial and genetic disorders | 2/104 | 0/32 |
| Adrenal insufficiencyEndocrine disorders | 1/104 | 0/32 |
| Otitis mediaInfections and infestations | 1/104 | 0/32 |
| SyringomyeliaNervous system disorders | 1/104 | 0/32 |
| GlomerulonephritisRenal and urinary disorders | 1/104 | 0/32 |
| Event | Somavaratan | rhGH |
|---|---|---|
| PyrexiaGeneral disorders | 18/104 | 4/32 |
| Injection site painGeneral disorders | 18/104 | 3/32 |
| VomitingGastrointestinal disorders | 12/104 | 5/32 |
| HeadacheNervous system disorders | 16/104 | 4/32 |
| CoughRespiratory, thoracic and mediastinal disorders | 15/104 | 2/32 |
| Upper respiratory tract infectionInfections and infestations | 12/104 | 3/32 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 12/104 | 1/32 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 11/104 | 3/32 |
| NauseaGastrointestinal disorders | 5/104 | 3/32 |
| NasopharyngitisInfections and infestations | 7/104 | 2/32 |
Safety Population included all participants who received any amount of study drug.
| Age, Continuous(years) | Somavaratan | rhGH | Total |
|---|---|---|---|
| Mean | 7.0 ± 2.03 | 6.9 ± 2.38 | 7.0 ± 2.11 |
| Sex: Female, Male(Participants) | Somavaratan | rhGH | Total |
|---|---|---|---|
| Female | 46 | 10 | 56 |
| Male | 58 | 22 | 80 |
| Ethnicity (NIH/OMB)(Participants) | Somavaratan | rhGH | Total |
|---|---|---|---|
| Hispanic or Latino | 8 | 3 | 11 |
| Not Hispanic or Latino | 96 | 29 | 125 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Somavaratan | rhGH | Total |
|---|---|---|---|
| American Indian or Alaska Native | 1 | 0 | 1 |
| Asian | 5 | 0 | 5 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 0 | 1 |
| White | 91 | 31 | 122 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 6 | 1 | 7 |
No study locations are listed for this record.
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
Ran from Aug 2015 to primary completion in Aug 2017, 23 months.
Results were first posted in Dec 2022, 5 years 4 months after primary completion.
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Studies of the drug tested here: Somavaratan
Versartis Inc.