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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1263-5766 | Registry Identifier | ICTRP | |
| 2023-509442-36-00 | Registry Identifier | CTIS | |
| 2021-004156-42 | EudraCT Number |
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The study was terminated as its Part A did not meet the primary endpoint.
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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 female participants, 18 to 80 years of age with ALS followed by an open-label, long-term extension period.
Study ACT16970 consisted of 2 parts (A and B) as follows:
Part A was a 24-week, double blind, placebo-controlled part, preceded by a screening period of up to 4 weeks before Day 1.
On Day 1 of Part A, participants were randomized in a 2:1 ratio to the SAR443820 treatment arm or matching placebo arm as listed below:
Randomization was stratified by the geographic region of the study site, region of ALS onset (bulbar vs other areas), use of riluzole (yes vs no), use of edaravone (yes vs no) and use of the combination of sodium phenylbutyrate and taurursodiol (named Relyvrio in the United States of America [USA] and Albrioza in Canada) (yes vs no). Participants attended in-clinic study assessments at baseline (Day 1), Week 2, Week 4, Week 6, Week 8, Week 10, Week 12, Week 16, Week 20, Week 21, Week 22, Week 23, and Week 24. All ongoing participants at Week 24 rolled to open-label extension Part B. The Week 24 Visit was the end of Part A and the beginning of Part B.
Part B was an open-label, long-term extension period that starts from Week 24 and continues up to Week 106. The objectives of Part B were to provide extended access to SAR443820 participants in Part A and to further evaluate the safety and efficacy of long-term SAR443820 treatment. The treatment assignment of participants at randomization in Part A remained blinded to Investigators, participants, and site personnel until the end of Part B. Every participant, except those who discontinued Investigational Medicinal Product (IMP) treatment permanently in Part A, received BID oral tablets of SAR443820 in Part B.
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, with a maximum total study duration of 110 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SAR443820 | Experimental | twice daily (BID) oral SAR443820 |
|
| Placebo | Placebo Comparator | twice daily (BID) oral placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SAR443820 | Drug | Tablet oral |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Change From Baseline to Week 24 in the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Total Score | The ALSFRS-R is an instrument to evaluate the functional status of participants with ALS. It consists of 12 items across 4 sub-domains of bodily function: bulbar, fine motor, gross motor, and breathing. Each item was scored on an ordinal scale from 0 (total loss of function) to 4 (no loss of function). The total scores was the sum of the individual items score and it ranges from 0 to 48, with a higher score indicating better function. The analysis was performed using mixed-effect model with repeated measures (MMRM). Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) and Week 24 |
| Part B: Combined Assessment of the Function and Survival (CAFS) Score at Week 52 | The CAFS at Week 52 is a composite endpoint based on time to earlier occurrence of death or permanent assisted ventilation (>22 hours a day for >7 consecutive days) and change from baseline in ALSFRS-R score up to Week 52. ALSFRS-R is a rating scale where 12 functions are rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome by time to death or permanent assisted ventilation and change on ALSFRS-R if survived without permanent assisted ventilation, assigned a score which is sum of comparisons (+1 [better], 0 [tie], -1 [worse]), and summed scores were ranked, from 1 to 296 (modified ITT[mITT] population) lowest rank corresponds to participant who died first and highest rank to 1 with best ALSFRS-R outcome among those who survived. A higher rank is considered a better outcome. | Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Combined Assessment of the Function and Survival Score at Week 24 | The CAFS at Week 24 is a composite endpoint based on time to earlier occurrence of death or permanent assisted ventilation (>22 hours a day for >7 consecutive days) and change from baseline in ALSFRS-R score up to Week 24. ALSFRS-R is a rating scale where 12 functions are rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome by time to death or permanent assisted ventilation and change on ALSFRS-R if survived without permanent assisted ventilation, assigned a score which is sum of comparisons (+1 [better], 0 [tie], -1 [worse]), and summed scores were ranked, from 1 to 296 (mITT population) lowest rank corresponds to participant who died first and highest rank to 1 with best ALSFRS-R outcome among those who survived. A higher rank is considered a better outcome. |
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Inclusion Criteria:
Exclusion Criteria:
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC San Diego Health Site Number : 8400022 | La Jolla | California | 92121 | United States | ||
| USC Site Number : 8400008 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38010108 | Derived | Hincelin-Mery A, Nicolas X, Cantalloube C, Pomponio R, Lewanczyk P, Benamor M, Ofengeim D, Krupka E, Hsiao-Nakamoto J, Eastenson A, Atassi N. Safety, pharmacokinetics, and target engagement of a brain penetrant RIPK1 inhibitor, SAR443820 (DNL788), in healthy adult participants. Clin Transl Sci. 2024 Jan;17(1):e13690. doi: 10.1111/cts.13690. Epub 2023 Dec 11. |
| Label | URL |
|---|---|
| ACT16970 (Himalaya study)-Amyotrophic Lateral Sclerosis website | View source |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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The study consisted of a screening period (up to 4 weeks prior to randomization), treatment period (a 24-week double-blinded in Part A, an 80-week open-label in Part B), and a 2-week post-treatment follow up period, with a maximum total study duration of 110 weeks. The study was terminated as Part A did not meet the primary endpoint.
This study is double-blind followed by an open-label extension, 2 parts: Part (A and B) conducted at 63 investigational sites in 13 countries. A total of 397 participants were screened between 13 Apr 2022 and 17 July 2023, of which 92 were screen failures. Screen failures were due to not meeting the eligibility criteria.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part A: Placebo | Participants received placebo matching to SAR443820 tablet orally twice a day (BID) for 24 weeks in Part A. |
| FG001 | Part A: SAR443820 | Participants received SAR443820 20 milligram (mg) tablet orally BID for 24 weeks in Part A. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (Double-blind Period: 24 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 13, 2023 | Dec 23, 2024 |
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| Drug |
Tablet |
|
| Week 24 |
| Combined Assessment of the Function and Survival Score at Weeks 76 and 104 | The CAFS at Weeks 76 and 106 is a composite endpoint based on time to earlier occurrence of death or permanent assisted ventilation (>22 hours a day for >7 consecutive days) and change from baseline in ALSFRS-R score up to Weeks 76 and 104. ALSFRS-R is a rating scale where 12 functions are rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome by time to death or permanent assisted ventilation and change on ALSFRS-R if survived without permanent assisted ventilation, assigned a score which is sum of comparisons (+1 [better], 0 [tie], -1 [worse]), and summed scores were ranked, from 1 to 296 (mITT population) lowest rank corresponds to participant who died first and highest rank to 1 with best ALSFRS-R outcome among those who survived. A higher rank is considered a better outcome. | Weeks 76 and 104 |
| Part B:Change From Baseline to Weeks 52, 76, and 104 in the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Total Score | The ALSFRS-R is an instrument to evaluate the functional status of participants with ALS. It consists of 12 items across 4 sub-domains of bodily function: bulbar, fine motor, gross motor, and breathing. Each item was scored on an ordinal scale from 0 (total loss of function) to 4 (no loss of function). The total scores was the sum of the individual items score and it ranges from 0 to 48, with a higher score indicating better function. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) and Weeks 52, 76 and 104 |
| Parts A and B: Change From Baseline to Weeks 24, 52, 76, and 104 in Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 Items (ALSAQ-5) | The ALSAQ-5 is a patient-reported outcome that consists of 5 items derived from the ALSAQ-40. The 5 items closely resemble those of the 5-dimension scores of the ALSAQ-40: eating and drinking; communication; activities of daily living/independence; physical mobility; and emotional functioning. Each item was scored on a 5-point Likert scale ranging from 0 (never) to 4 (always or cannot do at all) according to the frequency of a particular problem. The total scores was the sum of the individual items score and it ranges from 0 to 20, with higher scores indicative of greater physical and emotional limitations. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) and Part A: Week 24, Part B: Weeks 52, 76 and 104 |
| Parts A and B: Change From Baseline to Weeks 24, 52, 76, and 104 in Percent Predicted Slow Vital Capacity (SVC) | SVC is the maximum volume of air that can be slowly exhaled after slow, maximal inhalation. SVC is measured in participants while they are in an upright position at least 3 trials per assessment or up to 5 trials when the highest and second highest of the first 3 measurements differ by 10% or more. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) and Part A: Week 24, Part B: Weeks 52, 76 and 104 |
| Parts A and B: Change From Baseline to Weeks 24 and 52 in Serum Neurofilament Light Chain (NfL) | Serum blood samples were collected at indicated time points to measure changes in NfL, a marker of neuronal injury. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) and Part A: Week 24 and Part B: Week 52 |
| Part A: Change From Baseline to Week 24 in Muscle Strength | The muscles measured in the study included upper limb and lower-limb muscle groups. Bilateral hand grip were measured using a grip dynamometer and all other muscles were measured using a handheld dynamometer (HHD). Nine upper and lower extremity muscles or muscle groups were examined: shoulder flexion, elbow flexion, wrist extension, first dorsal interosseous contraction, hip flexion, knee extension, and ankle dorsiflexion. Each group was measured at least twice bilaterally and the average of the 2 highest measurements were analyzed. Individual muscles are standardized into the corresponding Z-scores using data from standard sample of healthy participants. A Z-score of 0 in a muscle measurement is equal to the mean of that measurement from the standard sample. Negative numbers indicate decreased muscle strength. For analysis, individual Z-scores were averaged to produce a total megascore including all available muscle groups. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) and Week 24 |
| Part B: Time From Baseline to Occurrence of Either Death or Permanent Assisted Ventilation | The survival endpoint was defined as the time to death or permanent assisted ventilation (>22 hours a day for >7 consecutive days), whichever comes first. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) up to early termination of study, approximately 84 weeks |
| Part B: Time From Baseline to the Occurrence of Death | Time to the occurrence of death from baseline due to any reason has been reported. Baseline was defined as the Day 1 pre-dose value. | Baseline (Day 1, pre-dose) up to early termination of study, approximately 84 weeks |
| Parts B and A+B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) After SAR443820 Initiation | An adverse event (AE) was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. TEAEs were defined as the AEs that developed, worsened or became serious during the treatment-emergent period (defined as time from first administration of study treatment (Day 1) to last administration of study treatment + 14 days). Serious adverse events (SAE): Any AE that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. All TEAE occurring after SAR443820 initiation are provided for both randomized treatment arms in this endpoint. | From first dose of SAR443820 up to 14 days after last dose of SAR443820 administration in Part B, approximately 82 weeks for Parts A+B combined Arm (SAR443820/SAR443820), approximately 58 weeks for Part B Arm (Placebo/SAR443820) |
| Parts A and B: Plasma Concentration of SAR443820 | Plasma samples were collected at specified timepoints to determine plasma concentrations of SAR443820. | Part A: Day 1: 0.25- 1 hour and 1-3 hours post-dose; Weeks 2 and 8: pre-dose; Week 8: 0.25-3 hours post-dose; Part B: Week 28: pre-dose |
| Los Angeles |
| California |
| 90033 |
| United States |
| University of California Irvine Site Number : 8400012 | Orange | California | 92868 | United States |
| California Pacific Medical Center Site Number : 8400015 | San Francisco | California | 94115 | United States |
| University of Colorado Site Number : 8400025 | Aurora | Colorado | 80045 | United States |
| Georgetown University Medical Center Site Number : 8400020 | Washington D.C. | District of Columbia | 20007 | United States |
| Mayo Clinic Site Number : 8400029 | Jacksonville | Florida | 32224 | United States |
| AdventHealth Medical Group - Neurology at Winter Park Site Number : 8400006 | Winter Park | Florida | 32789 | United States |
| Northwestern Medical Group, Department of Neurology Site Number : 8400003 | Chicago | Illinois | 60611 | United States |
| Johns Hopkins University Site Number : 8400028 | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital Site Number : 8400001 | Boston | Massachusetts | 02114 | United States |
| Mount Sinai - Union Square Site Number : 8400002 | New York | New York | 10003 | United States |
| Penn State Milton S. Hershey Medical Center Site Number : 8400004 | Hershey | Pennsylvania | 17033 | United States |
| University of Pennsylvania Site Number : 8400021 | Philadelphia | Pennsylvania | 19104 | United States |
| Thomas Jefferson University Hospital Site Number : 8400014 | Philadelphia | Pennsylvania | 19107 | United States |
| University of Utah Site Number : 8400009 | Salt Lake City | Utah | 84132 | United States |
| Froedtert Hospital & Medical College of Wisconsin Site Number : 8400010 | Milwaukee | Wisconsin | 53226 | United States |
| Investigational Site Number : 0560001 | Leuven | 3000 | Belgium |
| Investigational Site Number : 1240004 | Edmonton | Alberta | T6G 2C8 | Canada |
| Investigational Site Number : 1240007 | Hamilton | Ontario | L8N 3Z5 | Canada |
| Investigational Site Number : 1240006 | London | Ontario | N6A 5A5 | Canada |
| Investigational Site Number : 1240008 | Toronto | Ontario | M4N 3M5 | Canada |
| Investigational Site Number : 1240002 | Montreal | Quebec | H3A 2B4 | Canada |
| Investigational Site Number : 1240001 | Québec | G1J 1Z4 | Canada |
| Investigational Site Number : 1560001 | Beijing | 100191 | China |
| Investigational Site Number : 1560003 | Chengdu | 610041 | China |
| Investigational Site Number : 1560005 | Guangzhou | 510515 | China |
| Investigational Site Number : 1560002 | Hangzhou | 310009 | China |
| Investigational Site Number : 1560004 | Wuhan | 430030 | China |
| Investigational Site Number : 1560006 | Xi'an | 710061 | China |
| Investigational Site Number : 2500007 | Caen | 14033 | France |
| Investigational Site Number : 2500006 | Lille | 59037 | France |
| Investigational Site Number : 2500002 | Marseille | 13385 | France |
| Investigational Site Number : 2500003 | Montpellier | 34295 | France |
| Investigational Site Number : 2500004 | Tours | 37044 | France |
| Investigational Site Number : 2500005 | Vandœuvre-lès-Nancy | 54511 | France |
| Investigational Site Number : 2760004 | Berlin | 13353 | Germany |
| Investigational Site Number : 2760003 | Dresden | 01307 | Germany |
| Investigational Site Number : 2760008 | Haag in OB | 83527 | Germany |
| Investigational Site Number : 2760005 | Hanover | 30625 | Germany |
| Investigational Site Number : 2760002 | Lübeck | 23538 | Germany |
| Investigational Site Number : 2760001 | Ulm | 89081 | Germany |
| Investigational Site Number : 2760009 | Würzburg | 97074 | Germany |
| Investigational Site Number : 3800001 | Milan | 20132 | Italy |
| Investigational Site Number : 3800004 | Milan | 20138 | Italy |
| Investigational Site Number : 3800002 | Torino | 10126 | Italy |
| Investigational Site Number : 3920003 | Nagoya | Aichi-ken | 466-8560 | Japan |
| Investigational Site Number : 3920004 | Ichikawa-shi | Chiba | 272-0827 | Japan |
| Investigational Site Number : 3920006 | Tokushima | Tokushima | 770-8503 | Japan |
| Investigational Site Number : 3920005 | Fuchu-shi | Tokyo | 183-0042 | Japan |
| Investigational Site Number : 3920001 | Ōta-ku | Tokyo | 143-8541 | Japan |
| Investigational Site Number : 3920002 | Koshi-shi | 861-1196 | Japan |
| Investigational Site Number : 5280001 | Utrecht | 3584 CX | Netherlands |
| Investigational Site Number : 6160001 | Krakow | 31-503 | Poland |
| Investigational Site Number : 6160002 | Ksawerów | 95-054 | Poland |
| Investigational Site Number : 7240005 | Barcelona | Barcelona [Barcelona] | 08035 | Spain |
| Investigational Site Number : 7240002 | L'Hospitalet de Llobregat | Barcelona [Barcelona] | 08907 | Spain |
| Investigational Site Number : 7240003 | Madrid | 28029 | Spain |
| Investigational Site Number : 7240001 | Valencia | 46026 | Spain |
| Investigational Site Number : 7520002 | Stockholm | 113 61 | Sweden |
| Investigational Site Number : 7520001 | Umeå | SE-901 85 Umea | Sweden |
| Investigational Site Number : 8260002 | Plymouth | Devon | PL6 8DH | United Kingdom |
| Investigational Site Number : 8260003 | Stoke-on-Trent | Staffordshire | ST46QG | United Kingdom |
| FG002 | Part B: Placebo/SAR443820 | Participants who had received placebo matching to SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. |
| FG003 | Part B: SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. |
| Randomized and Treated | Participants who had received at least 1 dose of the study treatment. |
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| COMPLETED |
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| NOT COMPLETED |
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| Part B (Open-label Period: 80 Weeks) |
|
|
Randomized population=all participants from screened population who had been allocated to a randomized study treatment by interactive response technology (IRT) regardless of whether study treatment was received. Participants from Part A were allowed to transition to Part B per the study design hence baseline characteristics collected at the beginning of the study applies to both Part A and Part B.
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| ID | Title | Description |
|---|---|---|
| BG000 | Part A: Placebo | Participants received placebo matching to SAR443820 tablet orally BID for 24 weeks in Part A. |
| BG001 | Part A: SAR443820 | Participants received SAR443820 20 mg tablet orally BID for 24 weeks in Part A. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Baseline Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Total Score | The ALSFRS-R is an instrument to evaluate the functional status of participants with amyotrophic lateral sclerosis (ALS). It consists of 12 items across 4 sub-domains of bodily function: bulbar, fine motor, gross motor, and breathing. Each item was scored on an ordinal scale from 0 (total loss of function) to 4 (no loss of function). The total scores was the sum of the individual items score and it ranges from 0 to 48, with a higher score indicating better function. Baseline was defined as the Day 1 pre-dose value. | Only participants with data collected at specified timepoints are reported. | Mean | Standard Deviation | score on a scale |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Secondary | Part A: Combined Assessment of the Function and Survival Score at Week 24 | The CAFS at Week 24 is a composite endpoint based on time to earlier occurrence of death or permanent assisted ventilation (>22 hours a day for >7 consecutive days) and change from baseline in ALSFRS-R score up to Week 24. ALSFRS-R is a rating scale where 12 functions are rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome by time to death or permanent assisted ventilation and change on ALSFRS-R if survived without permanent assisted ventilation, assigned a score which is sum of comparisons (+1 [better], 0 [tie], -1 [worse]), and summed scores were ranked, from 1 to 296 (mITT population) lowest rank corresponds to participant who died first and highest rank to 1 with best ALSFRS-R outcome among those who survived. A higher rank is considered a better outcome. | The mITT population consisted of all randomized participants who either died or had available baseline and at least 1 post-baseline ALSFRS-R assessment. Only participants with data collected at Week 24 is reported. The study was terminated prematurely since the Part A did not meet the primary endpoint. | Posted | Mean | Standard Deviation | score on a scale | Week 24 |
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| Secondary | Combined Assessment of the Function and Survival Score at Weeks 76 and 104 | The CAFS at Weeks 76 and 106 is a composite endpoint based on time to earlier occurrence of death or permanent assisted ventilation (>22 hours a day for >7 consecutive days) and change from baseline in ALSFRS-R score up to Weeks 76 and 104. ALSFRS-R is a rating scale where 12 functions are rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome by time to death or permanent assisted ventilation and change on ALSFRS-R if survived without permanent assisted ventilation, assigned a score which is sum of comparisons (+1 [better], 0 [tie], -1 [worse]), and summed scores were ranked, from 1 to 296 (mITT population) lowest rank corresponds to participant who died first and highest rank to 1 with best ALSFRS-R outcome among those who survived. A higher rank is considered a better outcome. | The mITT population consisted of all randomized participants who either died or had available baseline and at least 1 post-baseline ALSFRS-R assessment. Only participants with data collected at specified timepoints are reported. The study was terminated prematurely since the Part A did not meet the primary endpoint. The data for Week 104 was not collected. All participants who had either early termination or who did not enter Part B, were also included in Week 76 analysis. | Posted | Mean | Standard Deviation | score on a scale | Weeks 76 and 104 |
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| Secondary | Part B:Change From Baseline to Weeks 52, 76, and 104 in the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Total Score | The ALSFRS-R is an instrument to evaluate the functional status of participants with ALS. It consists of 12 items across 4 sub-domains of bodily function: bulbar, fine motor, gross motor, and breathing. Each item was scored on an ordinal scale from 0 (total loss of function) to 4 (no loss of function). The total scores was the sum of the individual items score and it ranges from 0 to 48, with a higher score indicating better function. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at specified timepoints are reported. The study was terminated prematurely since the Part A did not meet the primary endpoint. The data for Week 104 was not collected. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1, pre-dose) and Weeks 52, 76 and 104 |
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| Secondary | Parts A and B: Change From Baseline to Weeks 24, 52, 76, and 104 in Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 Items (ALSAQ-5) | The ALSAQ-5 is a patient-reported outcome that consists of 5 items derived from the ALSAQ-40. The 5 items closely resemble those of the 5-dimension scores of the ALSAQ-40: eating and drinking; communication; activities of daily living/independence; physical mobility; and emotional functioning. Each item was scored on a 5-point Likert scale ranging from 0 (never) to 4 (always or cannot do at all) according to the frequency of a particular problem. The total scores was the sum of the individual items score and it ranges from 0 to 20, with higher scores indicative of greater physical and emotional limitations. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at specified timepoints are reported. The study was terminated prematurely since the Part A did not meet the primary endpoint. The data for Week 104 was not collected. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1, pre-dose) and Part A: Week 24, Part B: Weeks 52, 76 and 104 |
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| Secondary | Parts A and B: Change From Baseline to Weeks 24, 52, 76, and 104 in Percent Predicted Slow Vital Capacity (SVC) | SVC is the maximum volume of air that can be slowly exhaled after slow, maximal inhalation. SVC is measured in participants while they are in an upright position at least 3 trials per assessment or up to 5 trials when the highest and second highest of the first 3 measurements differ by 10% or more. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at specified timepoints are reported. The study was terminated prematurely since the Part A did not meet the primary endpoint. The data for Week 104 was not collected. | Posted | Mean | Standard Deviation | percentage of predicted volume | Baseline (Day 1, pre-dose) and Part A: Week 24, Part B: Weeks 52, 76 and 104 |
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| Secondary | Parts A and B: Change From Baseline to Weeks 24 and 52 in Serum Neurofilament Light Chain (NfL) | Serum blood samples were collected at indicated time points to measure changes in NfL, a marker of neuronal injury. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at specified timepoints are reported. | Posted | Geometric Mean | Geometric Coefficient of Variation | picograms per milliliter (pg/mL) | Baseline (Day 1, pre-dose) and Part A: Week 24 and Part B: Week 52 |
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| Secondary | Part A: Change From Baseline to Week 24 in Muscle Strength | The muscles measured in the study included upper limb and lower-limb muscle groups. Bilateral hand grip were measured using a grip dynamometer and all other muscles were measured using a handheld dynamometer (HHD). Nine upper and lower extremity muscles or muscle groups were examined: shoulder flexion, elbow flexion, wrist extension, first dorsal interosseous contraction, hip flexion, knee extension, and ankle dorsiflexion. Each group was measured at least twice bilaterally and the average of the 2 highest measurements were analyzed. Individual muscles are standardized into the corresponding Z-scores using data from standard sample of healthy participants. A Z-score of 0 in a muscle measurement is equal to the mean of that measurement from the standard sample. Negative numbers indicate decreased muscle strength. For analysis, individual Z-scores were averaged to produce a total megascore including all available muscle groups. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at Baseline and Week 24 are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1, pre-dose) and Week 24 |
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| Secondary | Part B: Time From Baseline to Occurrence of Either Death or Permanent Assisted Ventilation | The survival endpoint was defined as the time to death or permanent assisted ventilation (>22 hours a day for >7 consecutive days), whichever comes first. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | weeks | Baseline (Day 1, pre-dose) up to early termination of study, approximately 84 weeks |
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| Secondary | Part B: Time From Baseline to the Occurrence of Death | Time to the occurrence of death from baseline due to any reason has been reported. Baseline was defined as the Day 1 pre-dose value. | The ITT population consisted of all randomized participants. Only participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | weeks | Baseline (Day 1, pre-dose) up to early termination of study, approximately 84 weeks |
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| Secondary | Parts B and A+B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) After SAR443820 Initiation | An adverse event (AE) was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. TEAEs were defined as the AEs that developed, worsened or became serious during the treatment-emergent period (defined as time from first administration of study treatment (Day 1) to last administration of study treatment + 14 days). Serious adverse events (SAE): Any AE that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. All TEAE occurring after SAR443820 initiation are provided for both randomized treatment arms in this endpoint. | The safety population consisted of all randomized participants who received at least 1 dose (including partial dose) of SAR443820. All participants from safety population and treated with SAR443820 are included in Part B arm and participants from safety population are included in Parts A+B arm. The combined safety analysis for TEAE provided the accurate summary of all TEAE in both treatment groups after the initiation of SAR443820. | Posted | Count of Participants | Participants | From first dose of SAR443820 up to 14 days after last dose of SAR443820 administration in Part B, approximately 82 weeks for Parts A+B combined Arm (SAR443820/SAR443820), approximately 58 weeks for Part B Arm (Placebo/SAR443820) |
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| Secondary | Parts A and B: Plasma Concentration of SAR443820 | Plasma samples were collected at specified timepoints to determine plasma concentrations of SAR443820. | Pharmacokinetic (PK) population consisted of all randomized participants who received at least 1 dose of SAR443820 and had at least 1 PK assessment with adequate documentation of dosing and sampling. It was prespecified (statistical analysis plan) that participants will be analyzed according to actual intervention received. In Part B, all participants received SAR443820, so data were combined. Only participants who received SAR443820 with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | nanogram (ng)/mL | Part A: Day 1: 0.25- 1 hour and 1-3 hours post-dose; Weeks 2 and 8: pre-dose; Week 8: 0.25-3 hours post-dose; Part B: Week 28: pre-dose | Samples | Samples |
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| Primary | Part A: Change From Baseline to Week 24 in the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Total Score | The ALSFRS-R is an instrument to evaluate the functional status of participants with ALS. It consists of 12 items across 4 sub-domains of bodily function: bulbar, fine motor, gross motor, and breathing. Each item was scored on an ordinal scale from 0 (total loss of function) to 4 (no loss of function). The total scores was the sum of the individual items score and it ranges from 0 to 48, with a higher score indicating better function. The analysis was performed using mixed-effect model with repeated measures (MMRM). Baseline was defined as the Day 1 pre-dose value. | The Intent-to-treat (ITT) population consisted of all randomized participants. Only participants with data collected at Baseline and Week 24 are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1, pre-dose) and Week 24 |
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| Primary | Part B: Combined Assessment of the Function and Survival (CAFS) Score at Week 52 | The CAFS at Week 52 is a composite endpoint based on time to earlier occurrence of death or permanent assisted ventilation (>22 hours a day for >7 consecutive days) and change from baseline in ALSFRS-R score up to Week 52. ALSFRS-R is a rating scale where 12 functions are rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome by time to death or permanent assisted ventilation and change on ALSFRS-R if survived without permanent assisted ventilation, assigned a score which is sum of comparisons (+1 [better], 0 [tie], -1 [worse]), and summed scores were ranked, from 1 to 296 (modified ITT[mITT] population) lowest rank corresponds to participant who died first and highest rank to 1 with best ALSFRS-R outcome among those who survived. A higher rank is considered a better outcome. | The mITT population consisted of all randomized participants who either died or had available baseline and at least 1 post-baseline ALSFRS-R assessment. All participants who had either early termination or who did not enter Part B, were also included in Week 52 analysis. | Posted | Mean | Standard Deviation | score on a scale | Week 52 |
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Adverse events were collected from first dose of study treatment (Day 1) up to 14 days after last dose of study treatment administration, up to 26 weeks for Part A, and 58 weeks for Part B. All-cause mortality (death) was assessed from signing of the informed consent form to study termination, approximately 99 weeks.
Analysis was performed on safety population.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: Placebo | Participants received placebo matching to SAR443820 tablet orally BID for 24 weeks in Part A. | 5 | 102 | 17 | 102 | 56 | 102 |
| EG001 | Part A: SAR443820 | Participants received SAR443820 20 mg tablet orally BID for 24 weeks in Part A. | 11 | 202 | 34 | 202 | 116 | 202 |
| EG002 | Part B: Placebo/SAR443820 | Participants who had received placebo matching to SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. | 9 | 79 | 17 | 79 | 29 | 79 |
| EG003 | Part B: SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. | 12 | 136 | 26 | 136 | 43 | 136 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchitis | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Covid-19 | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Covid-19 Pneumonia | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Gastrointestinal Infection | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Lower Respiratory Tract Infection | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Pneumonia Aspiration | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Respiratory Syncytial Virus Infection | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Respiratory Tract Infection | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Skin Bacterial Infection | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Suspected Covid-19 | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Urinary Tract Infection | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Urosepsis | Infections and infestations | MedDra 26.1 | Systematic Assessment |
| |
| Basal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDra 26.1 | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDra 26.1 | Systematic Assessment |
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| Amyotrophic Lateral Sclerosis | Nervous system disorders | MedDra 26.1 | Systematic Assessment |
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| Speech Disorder | Nervous system disorders | MedDra 26.1 | Systematic Assessment |
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| Subarachnoid Haemorrhage | Nervous system disorders | MedDra 26.1 | Systematic Assessment |
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| Syncope | Nervous system disorders | MedDra 26.1 | Systematic Assessment |
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| Acute Myocardial Infarction | Cardiac disorders | MedDra 26.1 | Systematic Assessment |
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| Cardiac Arrest | Cardiac disorders | MedDra 26.1 | Systematic Assessment |
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| Cardiopulmonary Failure | Cardiac disorders | MedDra 26.1 | Systematic Assessment |
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| Left Ventricular Failure | Cardiac disorders | MedDra 26.1 | Systematic Assessment |
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| Deep Vein Thrombosis | Vascular disorders | MedDra 26.1 | Systematic Assessment |
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| Acute Respiratory Failure | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Interstitial Lung Disease | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Laryngospasm | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Respiratory Distress | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Respiratory Failure | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Dysphagia | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Gastric Haemorrhage | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Haematemesis | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Pancreatitis Acute | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Salivary Hypersecretion | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Cholecystitis | Hepatobiliary disorders | MedDra 26.1 | Systematic Assessment |
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| Drug-Induced Liver Injury | Hepatobiliary disorders | MedDra 26.1 | Systematic Assessment |
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| Hepatic Function Abnormal | Hepatobiliary disorders | MedDra 26.1 | Systematic Assessment |
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| Neck Pain | Musculoskeletal and connective tissue disorders | MedDra 26.1 | Systematic Assessment |
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| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDra 26.1 | Systematic Assessment |
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| Calculus Urinary | Renal and urinary disorders | MedDra 26.1 | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | MedDra 26.1 | Systematic Assessment |
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| Ureterolithiasis | Renal and urinary disorders | MedDra 26.1 | Systematic Assessment |
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| General Physical Health Deterioration | General disorders | MedDra 26.1 | Systematic Assessment |
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| Non-Cardiac Chest Pain | General disorders | MedDra 26.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDra 26.1 | Systematic Assessment |
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| Electrocardiogram T Wave Inversion | Investigations | MedDra 26.1 | Systematic Assessment |
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| Hepatic Enzyme Increased | Investigations | MedDra 26.1 | Systematic Assessment |
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| Concussion | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Craniofacial Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Femur Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Fibula Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Head Injury | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Hip Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Humerus Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Joint Dislocation | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Scapula Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Thoracic Vertebral Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Ulna Fracture | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Covid-19 | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDra 26.1 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDra 26.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDra 26.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDra 26.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Salivary Hypersecretion | Gastrointestinal disorders | MedDra 26.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDra 26.1 | Systematic Assessment |
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| Hepatic Enzyme Increased | Investigations | MedDra 26.1 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDra 26.1 | Systematic Assessment |
|
The study was terminated prematurely since the Part A did not meet the primary endpoint.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi aventis recherche & développement | 800-633-1610 | 6# | Contact-US@sanofi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 8, 2024 | Dec 23, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| Study terminated by sponsor |
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| Withdrawal by Subject |
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| Poor compliance to protocol |
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| Adverse Event |
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| OG001 | SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A for 24 weeks, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. All death participants were automatically included in this analysis, irrespective whether they reached Week 76 or not. |
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| OG002 | Part B: Placebo/SAR443820 | Participants who had received placebo matching to SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. |
| OG003 | Part B: SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. |
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| OG003 | Part B: SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. |
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Participants who had received SAR443820 tablet in Part A, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. |
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Participants who had received placebo matching to SAR443820 tablet in Part A for 24 weeks, continued to receive first intake of SAR443820 20 mg tablet orally BID up to 80 weeks during Part B. |
| OG001 | Parts A+B: SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A for 24 weeks, continued to receive SAR443820 20 mg tablet orally BID up to 80 weeks during Part B. |
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| OG001 | SAR443820/SAR443820 | Participants who had received SAR443820 tablet in Part A for 24 weeks, continued to receive SAR443820 20 mg tablet orally BID for 80 weeks during Part B. All death participants were automatically included in this analysis, irrespective whether they reached Week 52 or not. |
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