Trial results for the Phase 2 study (NCT05306873) investigating voclosporin in combination with mycophenolate mofetil for Systemic Lupus Erythematosus were posted on ClinicalTrials.gov on 2025-09-09. The study, which was terminated, showed that 0 participants in the voclosporin combination arm experienced treatment failure in Stage 3, compared to 1 participant in the placebo combination arm.
Background
The study, titled "Examining Distinct Immunophenotypes to Validate and Enhance Rational Treatment in Systemic Lupus," aimed to evaluate the potential effectiveness of mycophenolate mofetil (MMF) within immunologically defined subsets of Systemic Lupus Erythematosus (SLE) patients. It also explored and compared gene expression patterns and immunologic changes that accompany treatment-induced disease improvement with MMF and MMF plus voclosporin.
Trial design
The study (NCT05306873) was a Phase 2 trial that enrolled 12 participants with Systemic Lupus Erythematosus. The overall status of the trial is TERMINATED. Interventions included mycophenolate mofetil (MMF), placebo for MMF, voclosporin, and placebo for voclosporin. The study design involved evaluating treatment effects within immunologically-homogenous subsets of SLE patients, with a focus on MMF and MMF plus voclosporin in different stages, including Stage 2 and Stage 3 re-randomization.
Key results
Key results from the terminated Phase 2 study include several measurements related to treatment failure and clinical response across different stages of the trial.
- For the outcome "The Percentage of Participants Who Experience a Stage 2 Treatment Failure at or Before the Stage 2 Week 24 Visit":
- In the Stage 2 MMF group, 2 participants experienced treatment failure.
- In the Stage 2 Placebo for MMF group, 2 participants experienced treatment failure.
- For the outcome "Clinical Response in Stage 2, Defined by the BILAG-based Combined Lupus Assessment (BICLA) at Stage 2 Week 24":
- In one measurement, 1 participant in the Stage 2 MMF group showed clinical response, compared to 0 participants in the Stage 2 Placebo for MMF group.
- In another measurement for the same outcome, 2 participants in the Stage 2 MMF group showed clinical response, compared to 3 participants in the Stage 2 Placebo for MMF group.
- For the outcome "The Proportion of Participants Who Experience a Stage 3 Treatment Failure, After Stage 3 Re-randomization at or Before Completing Stage 3 Week 24":
- In the Stage 3 MMF + Voclosporin group, 0 participants experienced treatment failure.
- In the Stage 3 MMF + Placebo for Voclosporin group, 1 participant experienced treatment failure.
- For the outcome "Time to Treatment Failure in Stage 3, Defined as the Interval From the Day of Stage 3 Randomization Until the Day of Treatment Failure":
- In the Stage 3 MMF + Placebo for Voclosporin group, the mean time to treatment failure was 69 Days.
- For the outcome "Clinical Response in Stage 3, Defined by the BILAG-based Combined Lupus Assessment (BICLA) at Stage 3 Week 24":
- In one measurement, 0 participants in the Stage 3 MMF + Voclosporin group showed clinical response, compared to 0 participants in the Stage 3 MMF + Placebo for Voclosporin group.
- In another measurement for the same outcome, 1 participant in the Stage 3 MMF + Voclosporin group showed clinical response.
What this means
The results from this terminated Phase 2 trial, with its small enrollment of 12 participants, provide very preliminary insights into the use of voclosporin in combination with mycophenolate mofetil for Systemic Lupus Erythematosus. While the data for Stage 3 showed 0 participants experiencing treatment failure in the MMF + Voclosporin group compared to 1 participant in the MMF + Placebo for Voclosporin group, and 1 participant achieving clinical response in the MMF + Voclosporin group in one measurement, these findings are from a terminated study with limited statistical power. Further research would be needed to confirm any potential benefits suggested by these early results.
Source
The information for these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05306873, titled "Examining Distinct Immunophenotypes to Validate and Enhance Rational Treatment in Systemic Lupus," were posted on 2025-09-09 on clinicaltrials.gov.