Updated Review
Published: September 15, 2022
As of 9/15/22 we found an online report of a 43-patient, randomized, double-blind, placebo-controlled trial of RT001 (https://www.biospace.com/article/releases/biojiva-reports-results-of-pilot-phase-2-clinical-trial-of-rt001-in-patients-with-amyotrophic-lateral-sclerosis-als-/). This showed encouraging trends toward slowing ALSFRS-R and ALSAQ40 progression. Based upon this, we change our Trials grade from U to D. This and a published report of a small expanded access program (https://pubmed.ncbi.nlm.nih.gov/35765222/) suggested reasonably good tolerability and safety. No SAEs were attributed to RT001. As far as AEs, 25% of participants had GI side effects. We thus change our Risks grade from U to C.
Updated Review
Published: March 22, 2021
As of 3/22/21, we found a new clinical trial enrolling patients with ALS. We look forward to seeing the results of this trial. We found no other publicly available information to warrant any grade changes.
Key Information
Click on any letter grade below for more info:
Mechanism Grade: B
Preclinical Trials Grade: F
Cases Grade: D
Trials Grade: D
Risks Grade: C
Published: Jan 2019
RT001 has a novel mechanism for reducing oxidative stress that could theoretically work better than more traditional antioxidants. In the small trial of patients with Friedreich’s ataxia, it seems to be safe and well-tolerated at lower dosages but can cause nausea and diarrhea at higher doses. At the time of this writing, there is very little efficacy or safety data in PALS. An expanded access program is underway which allows PALS at certain clinics to try this compound free of charge. Data resulting from this expanded access program will help the planning of a possible future clinical trial.
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