A Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of SAR443820 in adult participants with amyotrophic lateral sclerosis, followed by an open-label extension
This is a study for patients diagnosed with amyotrophic lateral sclerosis (ALS). The purpose of the study is to evaluate twice daily (BID) oral doses of the investigational drug SAR443820 compared to placebo (which has no active medication) in addition to the standard of care, (e.g. riluzole or edaravone) for ALS. The study will evaluate the effectiveness (how well it works) and the possible risks of taking SAR443820. The study will last approximately 2 years. The study will be divided into 4 periods, including a screening period (up to 4 weeks), two treatment periods (Part A and Part B), and an end-of study follow-up period.
There will be nine visits of which seven will be in-person for Part A and nine in-person visits for Part B. Blood and urine will be collected 9 times in Part A including (1 optional) DNA sampling, and 10 times on Part B for a total of nineteen times. There will be early discontinuation visit on-site involving blood draw if for some reason the study had to be terminated early. Participants will perform several tests, including ECG and answer questionnaires.
Amount can vary depending on length of participation. Participants gets paid 75$ /on site study visit and 25$ for phone call completed for your participation
Participants must have been diagnosed with possible, clinically probable ALS, clinically probable laboratory-supported ALS, or clinically definite ALS
Participants must have an ALS disease duration (from first symptom onset to the screening visit) ≤2 years
Participants must have an ALSFRS-R prestudy slope ≥0.5 points/month prior to the screening visit.
Participants must be able to swallow the study tablets at the screening visit
Participants with central IV lines, such as a peripherally inserted central catheter (PICC) or midline or port-a-cath lines.
Participants with a significant cognitive impairment, psychiatric disease, other neurodegenerative disorder that would make the participants unsuitable for participating in the study
Participants with a history of recent serious infection within 4 weeks of the screening visit or chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the Investigator’s judgment.
Participants with an active herpes zoster infection within 2 months prior to the screening visit.