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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-003225-41 | EudraCT Number |
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| Name | Class |
|---|---|
| Ionis Pharmaceuticals, Inc. | INDUSTRY |
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The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BIIB067 | Experimental | Participants who have completed Parts A, B, or C of study 233AS101 will be placed in this arm. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tofersen | Drug | Participants will receive a loading dose regimen followed by maintenance dosing. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (TESAEs) | An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, life-threatening event, requires inpatient hospitalization, significant disability/incapacity or congenital anomaly. TEAEs were defined as any AEs or SAE with an onset date and time that was on or after the first dose of study drug, or any pre-existing condition that worsened in severity after the first dose of study drug. | From first dose of the study drug in the current study up to end of follow-up period (up to Week 364) |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Concentration of BIIB067 | Week 4 | |
| Concentration of BIIB067 in Cerebrospinal Fluid (CSF) | Week 4 | |
| 233AS101 and 233AS102 Integrated Summary of Efficacy (ISE): Total CSF Superoxide Dismutase 1 (SOD1) Protein Ratio to Baseline |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Phoenix | Arizona | 85013 | United States | ||
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41661214 | Derived | Miller TM, Cudkowicz ME, Shaw PJ, Genge A, Sobue G, Bucelli RC, Chio A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Salachas F, Bruneteau G, Al-Chalabi A, Amorin M, Nestorov I, Graham D, Lin L, Sun P, McNeill M, Malek S, Inra J, Garafalo S, Fradette S; VALOR and OLE Working Group. Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis. JAMA Neurol. 2026 Feb 1;83(2):115-125. doi: 10.1001/jamaneurol.2025.4946. | |
| 36129998 |
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A total of 139 participants were randomized in the study, of which 95 participants rolled over from Part C and 44 participants rolled over from Parts A and B of the parent study 233AS101 (NCT02623699).
Participants were enrolled and took part at the investigative sites in Belgium, Canada, France, Germany, Italy, Japan, Denmark, the United Kingdom, and the United States from 08 Mar 2017 to 12 Aug 2024.
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| ID | Title | Description |
|---|---|---|
| FG000 | 233AS101: Part C (Prior Placebo) | Participants who were randomized to placebo in Part C of the parent study 233AS101 received 3 loading doses of BIIB067, 100 milligrams (mg), once every 2 weeks (Q2W), on Days 1, 15, and 29 by intrathecal (IT) bolus injection in this study followed by up to 90 maintenance doses of BIIB067, every 4 weeks (Q4W), until the last enrolled participant had their Week 152 maintenance dose visit. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Aug 4, 2021 | Aug 12, 2025 |
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This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. |
| Baseline, Weeks 52, 104 and 148 |
| 233AS101 and 233AS102 ISE: Neurofilament Light Chain (NfL) Plasma Concentration Ratio to Baseline | This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Baseline, Weeks 52, 104 and 148 |
| 233AS101 and 233AS102 ISE: Change From Baseline in Total Amyotropic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score | The ALSFRS-R measures 4 functional domains, including respiratory, bulbar function, gross motor skills, and fine motor skills. There are 12 questions, each scored from 0 (no function) to 4 (full function). The ALSFRS-R total score was calculated as the sum of the 4 functional domain scores, ranging from 0 to 48, where higher scores representing better function. Negative change from baseline indicates disease progression. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Baseline, Weeks 52, 104 and 148 |
| 233AS101 and 233AS102 ISE: Change From Baseline in Percent Predicted Slow Vital Capacity (SVC) | Vital capacity was measured by means of an SVC test, administered in the upright position. Upright SVC was determined by performing 3 to 5 measures, in accordance with criteria established by the American Thoracic Society and the European Respiratory Society. The percent predicted SVC was calculated as [observed SVC divided by predicted SVC]*100%. The predicted SVC was adjusted by sex, age, height, which was programmed into and performed by the equipment used. Negative change from baseline indicated worsening of respiratory capacity. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Baseline, Weeks 52, 104 and 148 |
| 233AS101 and 233AS102 ISE: Change From Baseline in Handheld Dynamometry (HHD) Overall Megascore | Quantitative muscle strength was evaluated using the HHD Megascore, which tests isometric strength of multiple muscles using standard participant positioning. Approximately 8 muscle groups were examined (per each side) in both upper and lower extremities. The muscle strength values were normalized to Z scores as (post-baseline measurements - mean)/SD and averaged to provide HHD overall megascore. The overall megascore was created by averaging all eight bilateral measurement Z scores, if no more than 10 (≤ 10) measures are missing. A negative change from baseline indicated decreased muscle strength. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Baseline, Weeks 52, 104 and 148 |
| 233AS101 and 233AS102 ISE: Change From Baseline in Individual Muscle Strength Assessed by HHD | Individual muscle strength was evaluated using handheld dynamometer which tests the isometric strength of multiple muscles using standard participant positioning. Eight muscle groups were examined (per each side) in both upper and lower extremities. Negative change from baseline=decreased muscle strength. The analyses was based on observed data. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on data collected from both 233AS101 & 233AS102 studies. This is reported as a part of final integrated analyses. | Baseline, Weeks 52, 104 and 148 |
| 233AS101 and 233AS102 ISE: Time to Death or Permanent Ventilation | Permanent ventilation was defined as ≥ 22 hours of mechanical ventilation [invasive or noninvasive] per day for ≥ 21 consecutive days. An event of permanent ventilation was based on an adjudicated event (i.e., adjudicated by the Endpoint Adjudication Committee (EAC) as having met the permanent ventilation criteria defined in the protocol). Time to death or permanent ventilation was defined as the time to the earliest occurrence of death or permanent ventilation. The start date for calculating time to death or permanent ventilation in days was date of first dose. Participants without an event were censored at the last known alive dates. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on data collected from both 233AS101 & 233AS102 studies. This is reported as a part of final integrated analyses. Time to permanent ventilation or death was summarized using the Kaplan-Meier product limit method. | From the baseline of the study 233AS101 up to the end of the follow-up period of the current study (up to Week 364) |
| 233AS101 and 233AS102 ISE: Time to Death | Time to death was defined as the time from first dose received in 233AS101 to death. Participants who do not meet the endpoint definition were censored at the participant's last known alive date. Only events that were adjudicated by the EAC are included. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on data collected from both 233AS101 & 233AS102 studies. This is reported as a part of final integrated analyses. Time to death was summarized using the Kaplan-Meier product limit method. | From the baseline of the study 233AS101 up to the end of the follow-up period of the current study (up to Week 364) |
| La Jolla |
| California |
| 92093-0949 |
| United States |
| Research Site | San Francisco | California | 94118 | United States |
| Research Site | Jacksonville | Florida | 32224 | United States |
| Research Site | Miami | Florida | 33136 | United States |
| Research Site | Orlando | Florida | 32806 | United States |
| Research Site | Atlanta | Georgia | 30322 | United States |
| Research Site | Chicago | Illinois | 60611 | United States |
| Research Site | Baltimore | Maryland | 21287 | United States |
| Research Site | Boston | Massachusetts | 02114 | United States |
| Research Site | Rochester | Minnesota | 55905 | United States |
| Research Site | St Louis | Missouri | 63110 | United States |
| Research Site | Lincoln | Nebraska | 68510 | United States |
| Research Site | Cleveland | Ohio | 44106 | United States |
| Research Site | Portland | Oregon | 97213 | United States |
| Research Site | Knoxville | Tennessee | 37920 | United States |
| Research Site | Leuven | 3000 | Belgium |
| Research Site | Calgary | Alberta | T2N 4Z6 | Canada |
| Research Site | Edmonton | Alberta | T6G 2B7 | Canada |
| Research Site | Toronto | Ontario | M4N 3M5 | Canada |
| Research Site | Montreal | Quebec | H3A2B4 | Canada |
| Bispebjerg Hospital | Copenhagen | 2400 | Denmark |
| Research Site | Clermont-Ferrand | Puy De Dome | 63003 | France |
| Research Site | Ulm | Baden-Wurttemberg | 89081 | Germany |
| Research Site | Torino | 10126 | Italy |
| Research Site | Bunkyō City | Japan |
| Research Site | Kagoshima | Japan |
| Research Site | Shinjuku-ku | Japan |
| Research Site | Suita-shi | Japan |
| Research Site | Sheffield | South Yorkshire | S102HQ | United Kingdom |
| Derived |
| Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chio A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. |
| FG001 | 233AS101: Part C (Prior BIIB067 100 mg) | Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
| FG002 | 233AS101: Part A and B (All Doses) | Participants who were randomized to BIIB067 or placebo in Part A (at doses 10 mg, 20 mg, 40 mg and 60 mg) or Part B (at doses 20 mg, 40 mg, 60 mg and 100 mg) of the parent study 233AS101 received 3 loading doses of BIIB067, 20 mg, 40 mg, 60 mg, or 100 mg, eventually escalated to 100 mg, 2 weeks apart, and up to 90 maintenance doses, Q4W, by IT bolus injection until the last enrolled participant had their Week 152 maintenance dose visit in this study. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
The safety population included all participants who were enrolled and received at least one dose of study treatment in 233AS102.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 233AS101: Part C (Prior Placebo) | Participants who were randomized to placebo in Part C of the parent study 233AS101 received 3 loading doses of BIIB067, 100mg, Q2W, on Days 1, 15, and 29 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067, Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
| BG001 | 233AS101: Part C (Prior BIIB067 100 mg) | Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
| BG002 | 233AS101: Part A and B (All Doses) | Participants who were randomized to BIIB067 or placebo in Part A (at doses 10 mg, 20 mg, 40 mg and 60 mg) or Part B (at doses 20 mg, 40 mg, 60 mg and 100 mg) of the parent study 233AS101 received 3 loading doses of BIIB067, 20 mg, 40 mg, 60 mg, or 100 mg, eventually escalated to 100 mg, 2 weeks apart, and up to 90 maintenance doses, Q4W, by IT bolus injection until the last enrolled participant had their Week 152 maintenance dose visit in this study. |
| BG003 | 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 |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (TESAEs) | An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, life-threatening event, requires inpatient hospitalization, significant disability/incapacity or congenital anomaly. TEAEs were defined as any AEs or SAE with an onset date and time that was on or after the first dose of study drug, or any pre-existing condition that worsened in severity after the first dose of study drug. | The safety population included all participants who were enrolled and received at least one dose of study treatment in 233AS102. | Posted | Count of Participants | Participants | From first dose of the study drug in the current study up to end of follow-up period (up to Week 364) |
|
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| Secondary | Plasma Concentration of BIIB067 | As planned, plasma concentration of BIIB067 was summarized for 233AS101 Part C participants only. Pharmacokinetic (PK) population included all participants who received at least 1 dose of study treatment & had at least 1 post-dosing PK concentration measurement in current study. 'Overall number of participants analyzed' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Mean | Standard Error | nanograms per milliliter (ng/mL) | Week 4 |
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| Secondary | Concentration of BIIB067 in Cerebrospinal Fluid (CSF) | As planned, concentration of BIIB067 in CSF was summarized for 233AS101 Part C participants only. PK population included all participants who received at least 1 dose of study treatment & had at least 1 post-dosing PK concentration measurement in current study. 'Overall number of participants analyzed' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Mean | Standard Error | ng/mL | Week 4 |
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| Secondary | 233AS101 and 233AS102 Integrated Summary of Efficacy (ISE): Total CSF Superoxide Dismutase 1 (SOD1) Protein Ratio to Baseline | This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. Number analyzed 'n' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Geometric Least Squares Mean | 95% Confidence Interval | ratio | Baseline, Weeks 52, 104 and 148 |
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| Secondary | 233AS101 and 233AS102 ISE: Neurofilament Light Chain (NfL) Plasma Concentration Ratio to Baseline | This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. Number analyzed 'n' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Geometric Least Squares Mean | 95% Confidence Interval | ratio | Baseline, Weeks 52, 104 and 148 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 233AS101 and 233AS102 ISE: Change From Baseline in Total Amyotropic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score | The ALSFRS-R measures 4 functional domains, including respiratory, bulbar function, gross motor skills, and fine motor skills. There are 12 questions, each scored from 0 (no function) to 4 (full function). The ALSFRS-R total score was calculated as the sum of the 4 functional domain scores, ranging from 0 to 48, where higher scores representing better function. Negative change from baseline indicates disease progression. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. Number analyzed 'n' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Weeks 52, 104 and 148 |
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| Secondary | 233AS101 and 233AS102 ISE: Change From Baseline in Percent Predicted Slow Vital Capacity (SVC) | Vital capacity was measured by means of an SVC test, administered in the upright position. Upright SVC was determined by performing 3 to 5 measures, in accordance with criteria established by the American Thoracic Society and the European Respiratory Society. The percent predicted SVC was calculated as [observed SVC divided by predicted SVC]*100%. The predicted SVC was adjusted by sex, age, height, which was programmed into and performed by the equipment used. Negative change from baseline indicated worsening of respiratory capacity. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. Number analyzed 'n' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Least Squares Mean | Standard Error | percentage of predicted volume | Baseline, Weeks 52, 104 and 148 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 233AS101 and 233AS102 ISE: Change From Baseline in Handheld Dynamometry (HHD) Overall Megascore | Quantitative muscle strength was evaluated using the HHD Megascore, which tests isometric strength of multiple muscles using standard participant positioning. Approximately 8 muscle groups were examined (per each side) in both upper and lower extremities. The muscle strength values were normalized to Z scores as (post-baseline measurements - mean)/SD and averaged to provide HHD overall megascore. The overall megascore was created by averaging all eight bilateral measurement Z scores, if no more than 10 (≤ 10) measures are missing. A negative change from baseline indicated decreased muscle strength. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on the data collected from both 233AS101 and 233AS102 studies. This is reported as a part of the final integrated analyses. Baseline is defined as the Day 1 of 233AS101 Part C. Data has been reported for Weeks 52, 104 and 148 from the 233AS101 Part C baseline. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. Number analyzed 'n' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Weeks 52, 104 and 148 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | 233AS101 and 233AS102 ISE: Change From Baseline in Individual Muscle Strength Assessed by HHD | Individual muscle strength was evaluated using handheld dynamometer which tests the isometric strength of multiple muscles using standard participant positioning. Eight muscle groups were examined (per each side) in both upper and lower extremities. Negative change from baseline=decreased muscle strength. The analyses was based on observed data. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on data collected from both 233AS101 & 233AS102 studies. This is reported as a part of final integrated analyses. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. Number analyzed 'n' indicates the number of participants evaluable for this outcome measure at specified time point. | Posted | Mean | Standard Deviation | kilogram (kg) | Baseline, Weeks 52, 104 and 148 |
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| Secondary | 233AS101 and 233AS102 ISE: Time to Death or Permanent Ventilation | Permanent ventilation was defined as ≥ 22 hours of mechanical ventilation [invasive or noninvasive] per day for ≥ 21 consecutive days. An event of permanent ventilation was based on an adjudicated event (i.e., adjudicated by the Endpoint Adjudication Committee (EAC) as having met the permanent ventilation criteria defined in the protocol). Time to death or permanent ventilation was defined as the time to the earliest occurrence of death or permanent ventilation. The start date for calculating time to death or permanent ventilation in days was date of first dose. Participants without an event were censored at the last known alive dates. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on data collected from both 233AS101 & 233AS102 studies. This is reported as a part of final integrated analyses. Time to permanent ventilation or death was summarized using the Kaplan-Meier product limit method. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. | Posted | Median | 95% Confidence Interval | weeks | From the baseline of the study 233AS101 up to the end of the follow-up period of the current study (up to Week 364) |
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| Secondary | 233AS101 and 233AS102 ISE: Time to Death | Time to death was defined as the time from first dose received in 233AS101 to death. Participants who do not meet the endpoint definition were censored at the participant's last known alive date. Only events that were adjudicated by the EAC are included. This outcome measure was not a standalone analysis for 233AS102. Analysis was performed on data collected from both 233AS101 & 233AS102 studies. This is reported as a part of final integrated analyses. Time to death was summarized using the Kaplan-Meier product limit method. | Integrated analysis was performed on overall ITT population which included all Part C participants of 233AS101 and participants who rolled over from 233AS101 Part C into 233AS102. 'Overall number of participants analyzed' exceeds the total number of participants who started study 233AS102 as it indicates participants who were randomized in the Part C 233AS101 study. | Posted | Median | 95% Confidence Interval | weeks | From the baseline of the study 233AS101 up to the end of the follow-up period of the current study (up to Week 364) |
|
From first dose of the study drug in the current study up to end of follow-up period (up to Week 364)
The safety population included all participants who were enrolled and received at least one dose of study treatment in 233AS102.
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 | 233AS101: Part C (Prior Placebo) | Participants who were randomized to placebo in Part C of the parent study 233AS101 received 3 loading doses of BIIB067, 100 mg, Q2W, on Days 1, 15, and 29 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067, Q4W, until the last participant enrolled had their Week 152 maintenance dose visit. | 7 | 32 | 16 | 32 | 31 | 32 |
| EG001 | 233AS101: Part C (Prior BIIB067 100 mg) | Participants who were randomized to BIIB067, 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067, Q4W, until the last participant enrolled had their Week 152 maintenance dose visit. | 14 | 63 | 33 | 63 | 63 | 63 |
| EG002 | 233AS101: Parts A and B (All Doses) | Participants who were randomized to BIIB067 in Part A (at doses 10 mg, 20 mg, 40 mg and 60 mg) or Part B (at doses 20 mg, 40, 60 mg and 100 mg) of the parent study 233AS101 received 3 loading doses of BIIB067, 100 mg, on Days 1, 15, and 29 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067, Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. | 5 | 44 | 22 | 44 | 43 | 44 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arrhythmia | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cardiac failure acute | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Myopericarditis | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Papilloedema | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastric perforation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastric ulcer perforation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Large intestine perforation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Death | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Covid-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Covid-19 pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| H1n1 influenza | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Meningitis aseptic | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Myelitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pharyngeal abscess | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pulmonary sepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Streptococcal bacteraemia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Skull fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Stoma site pain | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Breast cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Uterine cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Brain hypoxia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Intracranial pressure increased | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Neuromyelitis optica spectrum disorder | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Neurosarcoidosis | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Radiculopathy | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Vocal cord paralysis | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Calculus urinary | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Chronic respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oesophagobronchial fistula | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonitis aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Respiratory arrest | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Euthanasia | Surgical and medical procedures | MedDRA 27.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vertigo | Ear and labyrinth disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Diplopia | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Papilloedema | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Covid-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Suspected covid-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Immunisation reaction | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Neurological procedural complication | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural complication | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural pruritus | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural swelling | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Procedural nausea | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Traumatic lumbar puncture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Blood urine present | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Csf glucose increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Csf protein increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Csf white blood cell count increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Burning sensation | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Intracranial pressure increased | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Muscle contractions involuntary | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Muscle spasticity | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pleocytosis | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Radicular pain | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Sensory disturbance | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Laryngospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
|
Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| US Biogen Clinical Trial Center | Biogen | 866-633-4636 | clinicaltrials@biogen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 6, 2024 | Aug 12, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| C000709090 | tofersen |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Black or African American |
|
| Native Hawaiian or Other Pacific Islander |
|
| White |
|
| Not Reported |
|
| Other |
|
| Title | Measurements |
|---|---|
|
| Units | Counts |
|---|
| Participants |
|
|
| Units | Counts |
|---|
| Participants |
|
|
Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|
Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067, Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|
| OG001 | 233AS101 and 233AS102 (Part C): Early-start BIIB067 100 mg | Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|
| OG001 | 233AS101 and 233AS102 (Part C): Early-start BIIB067 100 mg | Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|
Participants who were randomized to placebo in Part C of the parent study 233AS101 received 3 loading doses of BIIB067, 100 mg, Q2W, on Days 1, 15, and 29 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067, Q4W, until the last enrolled participant had their Week 152 maintenance dose visit.
| OG001 | 233AS101 and 233AS102 (Part C): Early-start BIIB067 100 mg | Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|
| OG001 |
| 233AS101 and 233AS102 (Part C): Early-start BIIB067 100 mg |
Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
| OG001 | 233AS101 and 233AS102 (Part C): Early-start BIIB067 100 mg | Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|
Participants who were randomized to BIIB067 100 mg in Part C of the parent study 233AS101 received 2 loading doses of BIIB067, 100 mg, on Days 1 and 29, and one dose of BIIB067-matched placebo on Day 15 by IT bolus injection in this study followed by up to 90 maintenance doses of BIIB067,Q4W, until the last enrolled participant had their Week 152 maintenance dose visit. |
|
|
|