Safety and Efficacy of PrimeC in Amyotrophic Lateral Sclerosis: The PARADIGM Randomized Clinical Trial - PubMed
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- #ALS
- #clinical trial
- #neurodegenerative disease
- PrimeC is a fixed-dose oral combination of celecoxib and ciprofloxacin designed to target ALS-related mechanisms.
- The PARADIGM trial evaluated the safety, tolerability, and potential efficacy of PrimeC in ALS patients.
- The study was a randomized, double-blind, placebo-controlled, phase 2b trial with a 12-month open-label extension.
- 68 participants were included in the intent-to-treat population, with balanced demographics between PrimeC and placebo groups.
- PrimeC was well tolerated with a safety profile comparable to placebo, though drug-related adverse events were more frequent with PrimeC.
- At 6 months, the mean ALSFRS-R difference was 2.23 points between PrimeC and placebo, not statistically significant (P = .12).
- At 18 months, continuous PrimeC treatment showed a significant difference in ALSFRS-R scores (7.92 points; P = .007) and bulbar function (3.18 points; P = .001).
- Continuous PrimeC treatment was associated with a lower risk of ALS complications (HR, 0.36; P = .02).
- Biomarker analyses showed preserved transferrin levels, abolished negative ferritin-ALSFRS-R correlation, and downregulation of ALS-associated microRNAs with PrimeC.
- PrimeC was deemed safe and well tolerated over 18 months, with functional and biomarker findings supporting a confirmatory trial.