Phase 3
N=138
Long-Acting Growth Hormone in Children Compared to Daily rhGH
Growth Disorders
Bottom Line
View on ClinicalTrials.gov: NCT02339090 ↗Enrolled (actual)
138
Serious AEs
4.4%
Results posted
Dec 2022
Primary outcome: Primary: Annual Height Velocity — 9.43; 10.70 cm/year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Somavaratan (Drug); rhGH (Drug)
- Age
- Pediatric · 3+ yrs
- Sex
- All
- Sponsor
- Versartis Inc.
- Primary completion
- Aug 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annual Height Velocity |
9.43; 10.70 | — |
| SECONDARY Change From Baseline in Height Standard Deviation Score (SDS) at Month 12 |
0.8; 1.0 | — |
| SECONDARY Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader |
1.1; 1.3 | — |
| SECONDARY Change From Baseline in Body Mass Index (BMI) at Month 12 |
1.1; -0.1 | — |
| SECONDARY Change From Baseline in Body Weight at Month 12 |
4.9; 3.8 | — |
| SECONDARY Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12 |
0.9; 1.8 | — |
| SECONDARY Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12 |
32.2; 49.8 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) |
80; 22 | — |
Summary
The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
Eligibility Criteria
Inclusion Criteria
- 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).
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT02339090). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.