ACT16753
What it found
The trial was stopped early because the treatment was not working.
Reason given: The study was terminated as it did not meet its primary and key secondary endpoints
What was tested
Treatment tested
- Oditrasertib
Placebo or sham
- Placebo
- Matched placebo for product
Results by group
Part A: Change From Baseline to Week 48 in Serum Neurofilament Light Chain (sNfL) Levels
Baseline (up to Day 1, pre-dose) and Week 48
| Group | Value | Interval | Number analysed |
|---|---|---|---|
| Part A: Placebo | 9.622 picograms per milliliter (pg/mL) | 5.316 (Standard Deviation) | 80 |
| Part A: SAR443820 | 10.319 picograms per milliliter (pg/mL) | 7.402 (Standard Deviation) | 85 |
| Part A: Placebo | 0.958 picograms per milliliter (pg/mL) | 6.983 (Standard Deviation) | 80 |
| Part A: SAR443820 | -0.343 picograms per milliliter (pg/mL) | 7.761 (Standard Deviation) | 85 |
Part A: Placebo
Part A: SAR443820
Part A: Placebo
Part A: SAR443820
p = 0.8042; LS Geometric Mean Ratio to Baseline 0.986 (95% CI 0.881–1.103)
Part B: Change From Baseline to Week 72 in Serum Neurofilament Light Chain Levels
Baseline (up to Day 1, pre-dose) and Week 72
| Group | Value | Interval | Number analysed |
|---|---|---|---|
| Part B: Placebo/SAR443820 | 9.622 pg/mL | 5.316 (Standard Deviation) | 80 |
| Part B: SAR443820/SAR443820 | 10.319 pg/mL | 7.402 (Standard Deviation) | 85 |
| Part B: Placebo/SAR443820 | -0.320 pg/mL | 3.433 (Standard Deviation) | 80 |
| Part B: SAR443820/SAR443820 | -0.270 pg/mL | 5.247 (Standard Deviation) | 85 |
Part B: Placebo/SAR443820
Part B: SAR443820/SAR443820
Part B: Placebo/SAR443820
Part B: SAR443820/SAR443820
Values as posted on ClinicalTrials.gov. Outcomes measured differently across trials are not directly comparable.
Safety
| Group | Participants at risk | Serious adverse events | Deaths |
|---|---|---|---|
| Part A: Placebo | 86 | 1 (1.2%) | 0 |
| Part A: SAR443820 | 87 | 2 (2.3%) | 0 |
| Part B: Placebo/SAR443820 | 77 | 5 (6.5%) | 0 |
| Part B: SAR443820/SAR443820 | 68 | 0 (0.0%) | 0 |
As posted on ClinicalTrials.gov. Serious adverse events include any serious health event during the trial, whether or not the treatment caused it.
What it measured
Main measures
Part A: Change From Baseline to Week 48 in Serum Neurofilament Light Chain (sNfL) Levels
Baseline (up to Day 1, pre-dose) and Week 48
MRI lesionsBlood or spinal fluid markersPart B: Change From Baseline to Week 72 in Serum Neurofilament Light Chain Levels
Baseline (up to Day 1, pre-dose) and Week 72
MRI lesionsBlood or spinal fluid markers1)Part A: Week 48 sNfL levels relative to baseline
Other measures (58)
Part A: Number of New Gadolinium-Enhancing T1 Hyperintense Lesions as Detected by Magnetic Resonance Imaging (MRI)
Post-baseline (Baseline: Day 1, pre-dose) and up to Week 48
MRI lesionsPart B: Number of New Gadolinium-Enhancing T1 Hyperintense Lesions as Detected by MRI
From Week 48 to approximately up to Week 72
MRI lesionsPart A: Number of New, Enlarging T2 Hyperintense Lesions as Detected by MRI
Post-baseline (Baseline: Day 1, pre-dose) and up to Week 48
MRI lesionsPart B: Number of New, Enlarging T2 Hyperintense Lesions as Detected by MRI
From Week 48 to approximately up to Week 72
MRI lesionsPart A: Time to Onset of 12 Weeks Confirmed Disability Progression (CDP) From Baseline as Assessed by the Expanded Disability Status Scale (EDSS) Score
Baseline (Day 1, pre-dose) and Week 48
Disability progressionBladder, bowel and sexual functionPart B: Time to Onset of 24 Weeks Confirmed Disability Progression From Baseline as Assessed by the Expanded Disability Status Scale Score
Baseline (Day 1, pre-dose) and approximately up to Week 72
Disability progressionBladder, bowel and sexual functionPart A: Time to Onset of Sustained 20% Increase in 9-Hole Peg Test (9-HPT) Confirmed Over at Least 12 Weeks
Baseline (Day 1, pre-dose) and Week 48
Disability progressionArm and hand functionPart B: Time to Onset of Sustained 20% Increase in 9-Hole Peg Test Confirmed Over at Least 24 Weeks
Baseline (Day 1, pre-dose) and approximately up to Week 72
Disability progressionArm and hand functionPart A: Time to Onset of Sustained 20% Increase in Timed 25-Foot Walk Test (T25-FW) Confirmed Over at Least 12 Weeks
Baseline (Day 1, pre-dose) and Week 48
Disability progressionWalking and mobilityPart B: Time to Onset of Sustained 20% Increase in Timed 25-Foot Walk Test Confirmed Over at Least 24 Weeks
Baseline (Day 1, pre-dose) and approximately up to Week 72
Disability progressionWalking and mobility
Who could take part
Relapsing and progressive · 18 to 60
Inclusion Criteria: * Male or female, 18 to 60 years (inclusive) of age, at the time of signing the informed consent. * Participants with diagnosis of RRMS, SPMS (relapsing or non-relapsing) or primary progressive subtype according to the 2017 revision of the McDonald diagnostic criteria (SPMS diagnostic criteria according to initial relapsing remitting disease course followed by progression with or without occasional relapses, minor remissions, and plateaus; progression denotes the continuous worsening of neurological impairment over at least 6 months). * Participants with Expanded Disability Status Scale (EDSS) score of 2 to 6 inclusive at screening. * Participants who were either untreated or in the opinion of the Investigator were stable on an allowed disease-modifying therapy (DMT) (interferons, glatiramer acetate, fumarates, or teriflunomide) for at least the past 3 months, AND not anticipated to require a change in multiple sclerosis (MS) treatment for the duration of Part A and Part B (through Week 96); in Part B changes in dose of allowed DMTs or transition to other allowed DMTs was permitted). * Participants with body weight at least 45 kg and body mass index (BMI) at least 18.0 kg/m\^2. * Contraceptive use by men and women was consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Exclusion Criteria: * Participants with immunodeficiency syndromes or other autoimmune diseases requiring immunosuppressive therapy * Participants with a history of seizures or epilepsy (history of febrile seizure during childhood was allowed). * Participants with known clinical relapse (acute or subacute episodes of new or increasing neurological dysfunction followed by full or partial recovery, in absence of fever or infection) within 8 weeks of screening. * Participants with neurological disease history other than MS, eg, head trauma within 3 months, cerebrovascular disease, and vascular dementia. * Participants with a history of recent serious infection (eg, pneumonia, septicemia) within 4 weeks of screening; an infection requiring hospitalization or intravenous antibiotics, antivirals, or antifungals within 4 weeks of screening; or chronic bacterial infections (such as tuberculosis) deemed unacceptable, as per Investigator's judgment. * Participants who had significant cognitive impairment, psychiatric disease, other neurodegenerative disorders (eg, Parkinson disease or Alzheimer disease), substance abuse, or any other conditions that made the participants unsuitable for participating in the study or interfered with assessment or completing the study in the opinion of the Investigator. * Participants with a documented history of attempted suicide over the 24 weeks prior to the Screening Visit, presents with suicidal ideation of category 4 or 5 on the Columbia Suicide Severity Rating Scale (CSSRS), or if in the Investigator's judgment, the participant was at risk for a suicide attempt. * Participants with a history of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or another medically significant illness other than MS precluding their safe participation in this study. * Participants who received a live vaccine within 14 days before the Screening Visit. * Participants with a known history of allergy to any ingredients of SAR443820 (mannitol, lactose monohydrate, sodium starch glycolate, colloidal silicon dioxide, magnesium stearate, hypromellose, titanium dioxide, polyethylene glycol, and microcrystalline cellulose). * Participants who used any medications that are moderate or strong inhibitors or strong inducers of cytochrome P450 3A4 (CYP3A4). * Participants who used of any of the following medications/treatments: fampridine/dalfampridine, ofatumumab, fingolimod, cladribine, siponimod, ponesimod, ozanimod, alemtuzumab, mitoxantrone, ocrelizumab, natalizumab, or similar approved compounds but with different trade names and any unapproved treatments or therapies for MS; any DMTs newly approved after January 2023 that are marketed at any time during the course of the double-blind study period. These medications were not allowed within 5 half-lives before the Screening Visit and for the duration of Part A and Part B. * Participants who had prior/concurrent clinical study enrollment, ie, the participant had taken other investigational drugs within 4 weeks or 5 half-lives, whichever was longer, before the first Screening Visit; concurrent or recent participation in non-interventional studies may be permitted. Participants with abnormal laboratory test(s) at the Screening Visit: * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) more than 3.0 x upper limit of normal (ULN) * Bilirubin more than 1.5 x ULN; unless the participant has documented Gilbert syndrome (isolated bilirubin more than 1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin less than 35%) * Serum albumin less than 3.5 g/dL * Estimated Glomerular filtration rate less than 60 mL/min/1.73 m\^2 (Modification of Diet in Renal Disease [MDRD]) * Other abnormal laboratory values or electrocardiogram (ECG) changes that are deemed clinically significant as per Investigator's judgment * The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial
As registered. Criteria can change, so ask the trial team or your neurologist.
About this trial
In the sponsor's words
This was a Phase 2, randomized, double-blind, placebo-controlled 2 parallel-arm study to assess the effect on serum neurofilament light chain (sNfL), safety and tolerability of oral SAR443820 compared to placebo in male and female participants aged 18 to 60 years with relapsing-remitting multiple sclerosis (RRMS), secondary progressive multiple sclerosis (SPMS) (relapsing or non-relapsing), or primary progressive multiple sclerosis (PPMS) followed by an open-label long-term extension period. The total study duration was approximately 100 weeks and included the following: 4-week screening period 48-week double-blind treatment period (Part A) 48-week open-label long-term extension period (Part B) The study was terminated prior to completion (of Week 96) as primary endpoint was not met. Therefore final duration was less than 96 weeks.
Papers
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Related trials
- Rolipram to Treat Multiple Sclerosis
Phase 2 · Completed
No result found
History
Started
Dec 2022
Main results due
Nov 2024
Finished
Nov 2024
Results posted
Oct 2025
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