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 extensio

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis; Lou Gehrig's Disease; 10029317

• Registration Number: NL-OMON53447
• Lead Sponsor: Sanofi BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 6

Inclusion Criteria

• Male or female, 18-80 years of age (inclusive)
• 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.
• 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
are 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 must have completed at
least 1 cycle of treatment before the screening visit and are expected to
continue edaravone treatment throughout the duration of the study.
• Weight : Participants with a body weight no less than 45 kg and body mass
index no less than 18 kg/m2.
• Female participants with childbearing potential are eligible to participate
if they are not pregnant or breastfeeding and agree to use adequate
contraceptive method during study intervention period and for at least 32 days
after the last dose of study drug.
• Male participants must 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 must not donate sperms for the duration of
study and 92 days after last dose of study drug.

Exclusion Criteria

• A history of seizure (History of febrile seizure during childhood is allowed).
• Having central IV lines, such as a peripherally inserted central catheter
(PICC) or midline or port-a-cath lines.
• With significant cognitive impairment, psychiatric disease, other
neurodegenerative disorder (eg, Parkinson disease or AD), substance abuse, 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 (C-SSRS), 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 are pregnant or are currently breastfeeding.
• A known history of allergy to any ingredients of SAR443820.
Prior/concomitant therapy :
• Currently or previously treated with any strong or moderate CYP3A4 inhibitors
or strong CYP3A4 inducers listed in Appendix 10 of the protocol 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 have received treatment with another investigational drug
within 4 weeks or 5 half-lives of the investigational agent before the
screening visit, whichever is longer.
• Participants who have 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 has documented Gilbert syndrome
(isolated bilirubin >1.5 × ULN is acceptable if bilirubin is 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])

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Part A: Change from baseline in the ALSFRS-R total score from screening to Week<br /><br>24<br /><br><br /><br>Part B: Combined assessment of the function and survival (CAFS) score at Week<br /><br>52</p><br>