*Himalaya
Research type
Research Study
Full title
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.
IRAS ID
1004843
Contact name
Severine Cointet
Contact email
Sponsor organisation
Sanofi-aventis recherche & développement
Eudract number
2021-004156-42
Clinicaltrials.gov Identifier
Research summary
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive loss of motor neurons in the cortex, brain stem, and spinal cord. Relentless and progressive muscle wasting and weakness are hallmarks of ALS. Most patients with ALS die from respiratory failure within 3 to 5 years after disease onset. The 2 Food and Drug Administration (FDA)-approved ALS treatments demonstrate only a moderate effect either on survival or functional decline. There is a significant unmet medical need for treating individuals with ALS.
SAR443820 is an investigational drug that is being developed to slow down the disease progression of ALS. SAR443820 is a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor. RIPK1 is protein found within cells, involved in the regulation of inflammation, cytokine release, and cell death. Both preclinical and clinical studies have shown that RIPK1 is a key mediator of programmed cell death and inflammatory pathways in ALS. SAR443820, an inhibitor of RIPK1 that can penetrate the central nervous system, has the potential to modify the course of neurodegenerative processes and slow disease progression in patients with ALS.
The purpose of this study is to see if the drug SAR443820 works to improve the symptoms of adults with ALS compared to a group receiving placebo (a dummy substance), with both groups continuing to receive their current treatment for ALS. Additional purposes of the study are to find out the pharmacokinetics (PK), and pharmacodynamics (PD) of SAR443820 and how safe and tolerable the drug is.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
22/EM/0185
Date of REC Opinion
11 Mar 2022
REC opinion
Further Information Favourable Opinion