Trial reportJournal of neurology2026
Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy.
Trial report in Journal of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies
A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
Who cites it
1 citing paper in PubMed.
- Trial
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundVamorolone is a dissociative glucocorticoid receptor agonist for treating Duchenne muscular dystrophy (DMD). The VBP15-006 study assessed vamorolone safety, tolerability, and pharmacokinetics in 2- to < 4 and 7- to < 18-year-old boys with DMD. Results for 7- to < 18-year-old boys, either corticosteroid (CS)-untreated or switching from prior CS treatment, are reported here. Exploratory objectives included efficacy and pharmacodynamic biomarkers related to safety.
methodsIn this phase II, open-label, multiple-dose study, participants received 2 or 6 mg/kg/day vamorolone for 12 weeks, followed by treatment in an expanded access protocol (EAP Canada).
results34 participants (12 CS-untreated, 22 CS-treated) aged 7- to < 18 years were enrolled. Most treatment-emergent adverse events were mild. All 34 participants completed VBP15-006 and entered EAP Canada. During EAP Canada, CS-untreated participants maintained stable linear growth, while CS-treated participants exhibited catch-up growth consistent with serum bone biomarkers (median total vamorolone exposure 1.3 and 1.7 years, respectively). Some weight gain occurred, especially in CS-untreated participants receiving 6 mg/kg/day. Dose-dependent adrenal suppression occurred in CS-untreated and CS-treated participants; 2 individuals who switched from deflazacort to vamorolone 2 mg/kg/day had generalized weakness consistent with adrenal insufficiency. Vamorolone showed dose-dependent pharmacokinetics, rapid clearance, and no accumulation. There were no relevant efficacy changes in CS-treated and CS-untreated groups at either dose.
conclusionsVamorolone demonstrated a consistent safety profile in 7- to < 18-year-old boys with DMD. Switching to 6 mg/kg/day vamorolone appeared to mitigate adrenal insufficiency risk. There was no negative effect on growth, and catch-up growth occurred in previously CS-treated individuals switching to vamorolone.
trial registrationClinicalTrials.gov: NCT05185622, NCT03863119. First submitted 09-11-2021.
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