How the study was designed
RADIANT-LMNS was a Phase 3, multicenter, double-blind study. Participants received Veylora 40 mg once daily or placebo in addition to stable supportive care. Neither participants nor investigators knew which treatment was assigned during the first 24 weeks.
adults enrolled at 42 study centers
weeks of total observation
participants assigned Veylora
participants assigned placebo
Primary endpoint at Week 24
The primary endpoint was the percentage of participants achieving a clinically meaningful response, defined as at least a 4-point improvement on the 30-point LMNS Functional Response Score (LFRS).
68% responded with Veylora vs 29% with placebo
Difference: 39 percentage points; 95% confidence interval: 25–52; p<0.001. Individual results may vary.
Additional outcomes
| Outcome at Week 24 | Veylora | Placebo |
|---|---|---|
| Mean change in LFRS | +6.8 points | +2.1 points |
| Median reduction in monthly episodes | 41% | 12% |
| Improvement in timed hand task | 2.4 seconds | 0.9 seconds |
| Patient-reported fatigue improvement | 55% | 26% |
Secondary endpoints were tested in a prespecified statistical hierarchy. The timed hand-task result is presented descriptively and was not adjusted for multiple comparisons.
Who participated?
- Average age was 39 years; ages ranged from 19 to 67.
- 58% identified as female and 42% as male.
- Average time since diagnosis was 4.7 years.
- Participants had at least 2 neuro-inflammatory episodes during the previous 6 months.
- All participants had a confirmed pathogenic or likely pathogenic NRT7 variant.
Understanding the results
Important study limitations
The trial enrolled a relatively small rare-disease population. People with severe liver disease, active serious infection, recent blood clots, or pregnancy were excluded. Results may not apply to people unlike those who participated. Longer-term benefits and risks continue to be evaluated.
Considering treatment?
Learn about the daily dose, baseline testing, ongoing monitoring, and medicine interactions.
How to take Veylora →