Adrenal Suppression From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial
Authors' conclusion
AS after vamorolone and prednisone was frequent and vamorolone-associated AS appeared dose-dependent. A lower stimulated cortisol threshold may be appropriate when using a monoclonal assay. We recommend hydrocortisone for glucocorticoid stress dosing in patients receiving vamorolone.
Study context
- Population
- Boys with pediatric Duchenne muscular dystrophy (DMD); mean age at enrolment 5.41 ± 0.86 years
- Participants
- 118 participants
- Comparison
- Vamorolone 6 or 2 mg/kg/day vs prednisone 0.75 mg/kg/day vs placebo
- Prednisone 0.75 mg/kg/dayn = 25
- Vamorolone 6 mg/kg/dayn = 21
- Vamorolone 2 mg/kg/dayn = 19
- Placebon = 20reference
- Primary endpoint
- Adrenal suppression (AS): stimulated cortisol <500 nmol/L (historical threshold) and <400 nmol/L (revised threshold), Elecsys II immunoassay
- Follow-up
- Week 24
Methodological detailsdouble-blind, placebo- and prednisone-controlled, 24-week crossover extension, phase 2b
- Double-blind
- Placebo- and prednisone-controlled
- 24-week crossover extension
- Phase 2b
Primary result
As reported in the abstract
AS, historical threshold (<500 nmol/L)
Further findings
AS, revised threshold (<400 nmol/L) · week 24
Correlation of morning and peak ACTH-stimulated cortisol in steroid-treated boys · week 48
- Spearman correlation
- 0.83
Original publication
Adrenal Suppression From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial. J Clin Endocrinol Metab. Published 4 August 2024. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →