A Phase IIb, Multi-Center, Multinational, Double-Blind, Placebo-Controlled Study, With an Open Label Extension, to Evaluate Safety, Tolerability and Efficacy of PrimeC in Subjects With ALS

Summary

69 subjects with ALS will be enrolled in the study and randomized at a 2:1 ratio to receive the study drug or placebo tablets. Randomization sequences will be in random block sizes and stratified for ENCALS risk category \[high risk ≥ -4.5 vs. low risk \< -4.5\], and for background ALS treatment (riluzole and/or edaravone and/or sodium phenylbutyrate and/or taurursodiol) vs. no background ALS treatment. All subjects will be administered the drug/placebo twice daily (BID), two tablets each time, for 6 months. Subjects will be allowed to receive standard of care (SOC) treatment of approved products (i.e., riluzole and edaravone). Additionally, subjects will be allowed to receive treatment with off-label sodium phenylbutyrate and taurursodiol, which are accepted for ALS treatment. Subjects will be evaluated every 2 months for safety, tolerability (adverse events, safety laboratory, vital signs, ECG, withdrawal rates and reasons) and efficacy (e.g. biomarkers, clinical outcomes (ALSFRS-R and SVC, quality of life and survival). All subjects who complete the 6 months dosing will be switched to the active arm for a 12-month open label extension (OLE).

Timeline

Start

2022-05-31

Primary completion

2023-11-02

Completion

2024-11-04

Publications

• Cudkowicz M, Drory VE, Chio A, Lunetta C, Shoesmith C, van Eijk RPA, Salomon-Zimri S, Shtossel D, Kerem N, Shapira G, Shomron N, Russek-Blum N, Tracik F, Rosenfeld J, Shefner J. Safety and Efficacy of PrimeC in Amyotrophic Lateral Sclerosis: The PARADIGM Randomized Clinical Trial. JAMA Neurol. 2026 May 1;83(5):471-480. doi: 10.1001/jamaneurol.2026.0230.

Drugs

Indications