Status:
TERMINATED
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Lead Sponsor:
Sanofi
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18-80 years
Phase:
PHASE2
Brief Summary
This was a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and fe...
Detailed Description
The study duration included an up to 4-week screening period, 24-week double-blind treatment period in Part A, 80-week open-label treatment period in Part B and 2-week post-treatment follow-up period,...
Eligibility Criteria
Inclusion
- Diagnosis of possible, clinically probable ALS, clinically probable laboratory supported ALS, or clinically definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria
- Time since onset of first symptom of ALS ≤2 years.
- Slow Vital Capacity (SVC) ≥60% of the predicted value.
- Had to be able to swallow the study tablets at the screening visit.
- Either not currently receiving riluzole or on a stable dose of riluzole for at least 4 weeks before the screening visit. Participants receiving riluzole were expected to remain on the same dose throughout the duration of the study.
- Either not currently receiving edaravone or on the approved standard schedule of edaravone treatment. Participants receiving edaravone had to have completed at least 1 cycle of treatment before the screening visit and were expected to continue edaravone treatment throughout the duration of the study.
- Either not currently receiving the combination of sodium phenylbutyrate and taurursodiol or on the approved standard schedule of the combination of sodium phenylbutyrate and taurursodiol treatment for at least 4 weeks before the screening visit. Participants receiving the combination of sodium phenylbutyrate and taurursodiol were expected to remain on the approved standard schedule throughout the duration of the study.
- Participants with a body weight no less than 45 kg and body mass index no less than 18 kg/m2 at the screening visit
- Female participants with childbearing potential were eligible to participate if they were not pregnant or breastfeeding and agreed to use adequate contraceptive method during study intervention period and for at least 32 days after the last dose of study drug.
- Male participants had to agree to use highly effective contraceptive method during the study period and for at least 92 days following their last dose of the study drug. Male participants were not donate sperms for the duration of study and 92 days after last dose of study drug.
Exclusion
- A history of seizure (History of febrile seizure during childhood was allowed).
- Having central IV lines, such as a peripherally inserted central catheter (PICC XE ' PICC ' \\f Abbreviation \\t 'peripherally inserted central catheter' ) or midline or portacath lines.
- With significant cognitive impairment, psychiatric disease, other neurodegenerative disorder (eg, Parkinson disease or AD), substance abuse other causes of neuromuscular weakness, or any other condition that would make the participants unsuitable for participating in the study or could interfere with assessment or completing the study in the opinion of the Investigator.
- History of recent serious infection (eg, pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with IV antibiotics, antivirals, or antifungals within 4 weeks of screening; or chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the Investigator's judgment.
- With active herpes zoster infection within 2 months prior to the screening visit.
- A documented history of attempted suicide within 6 months prior to the screening visit, present with suicidal ideation of category 4 or 5 on the Columbia Suicide Severity Rating Scale (CSSRS) , or in the Investigator's judgment are at risk for a suicide attempt.
- History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or another medically significant illness other than ALS precluding their safe participation in this study.
- Participants who were pregnant or were currently breastfeeding.
- A known history of allergy to any ingredients of SAR443820.
- Currently or previously treated with any strong or moderate CYP3A4 inhibitors or strong CYP3A4 inducers within the specified washout period before the screening visit.
- Received a live vaccine within 14 days before the screening visit.
- Participants with concurrent participation in any other interventional clinical study or who had received treatment with another investigational drug (eg sodium phenylbutyrate or taurursodiol ) within 4 weeks or 5 halflives of the investigational agent before the screening visit, whichever is longer.
- Participants who had received stem cell or gene therapy for ALS at any time in the past.
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3.0 × upper limit of normal (ULN)
- Bilirubin \>1.5 × ULN unless the participant had documented Gilbert syndrome (isolated bilirubin \>1.5 × ULN was acceptable if bilirubin was fractionated and direct bilirubin is \<35%)
- Serum albumin \<3.5 g/dL
- Estimated glomerular filtration rate \<60 mL/min/1.73 m2 (Modification of Diet in Renal Disease \[MDRD\])
- The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Key Trial Info
Start Date :
April 13 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 7 2024
Estimated Enrollment :
305 Patients enrolled
Trial Details
Trial ID
NCT05237284
Start Date
April 13 2022
End Date
March 7 2024
Last Update
March 21 2025
Active Locations (63)
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1
UC San Diego Health Site Number : 8400022
La Jolla, California, United States, 92121
2
USC Site Number : 8400008
Los Angeles, California, United States, 90033
3
University of California Irvine Site Number : 8400012
Orange, California, United States, 92868
4
California Pacific Medical Center Site Number : 8400015
San Francisco, California, United States, 94115