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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-001947-18 | EudraCT Number |
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The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc.
In August 2016, sponsorship of the trial was transferred to Biogen.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nusinersen | Experimental | Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274. |
|
| Sham procedure | Sham Comparator | Sham comparator on Days 1, 29, 85 and 274. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nusinersen | Drug | Administered by intrathecal (IT) lumbar puncture (LP) injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. | Baseline and Month 15 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. |
Not provided
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 |
|---|---|---|---|---|---|---|
| UCLA Clinical and Translational Research Center | Los Angeles | California | 90095 | United States | ||
| Lucile Packard Children's Hospital at Stanford |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31420846 | Derived | Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019 Sep;33(9):919-932. doi: 10.1007/s40263-019-00656-w. | |
| 29443664 | Derived |
| Label | URL |
|---|---|
| Cure SMA | View source |
Not provided
After parental informed consent was obtained and prior to any treatment, participants entered a Screening Period of up to 21 days to determine their eligibility for the study. Of the 179 participants screened, 53 were screening failures.
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| ID | Title | Description |
|---|---|---|
| FG000 | Sham Procedure | Sham comparator on Days 1, 29, 85 and 274. |
| FG001 | Nusinersen | Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 12, 2016 | Dec 8, 2017 |
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| Sham procedure | Procedure | Small needle prick on the lower back at the location where the IT injection is normally made |
|
| Baseline and Month 15 |
| Proportion of Participants That Achieved Any New Motor Milestone at Month 15 | New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. | Month 15 |
| Number of New Motor Milestones Achieved Per Participant | New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. | Month 15 |
| Change From Baseline in Revised Upper Limb Module (RULM) Test | The RULM Test is used in patients with SMA to assess upper limb functional ability items that are reflective of activities of daily living (i.e., raise a can to mouth as if drinking, take a coin and place it in a box, remove the lid of a container). The RULM test has a total of 20 items with an entry item that serves as functional class identification and does not contribute to the total score. The remaining 19 scorable items reflect different functional domains and are graded on a 3-point system with a score of 0 (unable), 1 (able, with modification), and a maximum of 2 (able, no difficulty). There is only 1 item (item I) that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for a total score range of 0-37, with higher scores increased great upper limb function. A positive change from Baseline indicates improvement. | Baseline and Month 15 |
| Proportion of Participants That Achieved Standing Alone | If the participant was unable to achieve standing alone at Baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder. | Month 15 |
| Proportion of Participants That Achieved Walking With Assistance | If the participant was unable to achieve walking with assistance at baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder. | Month 15 |
| Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death; an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity. Any other medically important event that, in the opinion of the investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above. | Baseline through Month 15 |
| Number of Participants With Clinically Significant Vital Sign Abnormalities | Vital signs assessed for clinical significance include resting blood pressure, pulse, respiratory rate, and temperature. | Baseline through Month 15 |
| Number of Participants With Clinically Significant Weight Abnormalities | Weight changes assessed from Baseline to Month 15. | Baseline through Month 15 |
| Number of Participants With Clinically Significant Neurological Examination Abnormalities | Neurological changes assessed for clinical significance include assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes. | Baseline through Month 15 |
| Number of Participants With Clinically Significant Physical Examination Abnormalities | Physical examination changes were assessed for clinical significance. | Baseline through Month 15 |
| Number of Participants With Clinically Significant Laboratory Parameter Abnormalities | Laboratory parameter changes assessed for clinical significance include serum chemistry, hematology, coagulation and urinalysis. | Baseline through Month 15 |
| Number of Participants With Abnormal, Clinically Relevant Post-Baseline Worsening in Electrocardiogram (ECG) in Results | The number of participants with abnormal, clinically relevant worsening, defined as participants with an ECG interpreted as abnormal and clinically relevant, with a comparison with Baseline value is reported. | Baseline through Month 15 |
| Number of Participants Taking Any Concomitant Medication Related to Dosing Procedure or Sham Procedure | Concomitant medications include prescription and over-the-counter medications administered to participants on or after the first day of study treatment. | Baseline through Month 15 |
| Palo Alto |
| California |
| 94304 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Nemours Children's Hospital | Orlando | Florida | 32827 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia - Neurology | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Medical Center | Dallas | Texas | 75235 | United States |
| Children's Hospital - London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| McGill University Health Centre-Glen Site-CIM | Montreal | Quebec | H4A 3JI | Canada |
| Armand Trousseau Hospital, I-Motion, Clinical Trials Platform | Paris | France |
| Universitatsklinikum Essen | Essen | Germany |
| University Hospital Freiberg, Center for Paediatrics and Adolescent Medicine, Department of Neuropaediatrics and Muscular Disease | Freiburg im Breisgau | Germany |
| The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine | Hong Kong | Hong Kong SAR | Hong Kong |
| AOU Policlinico G. Martino Dipartimento di Neuroscienze e Centro Clinico Nemo Sud | Messina | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli-Universita Cattolica de Sacro Cuore-UOC Neuropsichiatre Infantile | Rome | Italy |
| Hyogo College of Medicine | Nishinomya-shi | Hyōgo | Japan |
| Tokyo Women's Medical University | Shinjuku-ku | Tokyo | Japan |
| Seoul National University Children's Hospital | Seoul | Korea | South Korea |
| Hospital Sant Joan de Deu | Barcelona | Spain |
| University of Gothenburg, The Queen Silvia Children's Hospital | Gothenburg | Sweden |
| Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504. |
| Muscular Dystrophy Association | View source |
| National Organization for Rare Diseases | View source |
| Completed Treatment |
|
| COMPLETED |
|
| NOT COMPLETED |
|
Intent-to-Treat (ITT) Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Sham Procedure | Sham comparator on Days 1, 29, 85 and 274. |
| BG001 | Nusinersen | Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274. |
| 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 |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Hammersmith Functional Motor Scale - Expanded (HFMSE) Score | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. | Mean | Standard Deviation | scores 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. | ITT Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Missing postbaseline HFMSE data were imputed using the multiple imputation method. | Posted | Least Squares Mean | 95% Confidence Interval | scores on a scale | Baseline and Month 15 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. | ITT Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Missing postbaseline HFMSE data were imputed using multiple imputation. | Posted | Number | 95% Confidence Interval | Proportion of participants | Baseline and Month 15 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Proportion of Participants That Achieved Any New Motor Milestone at Month 15 | New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. | Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data. | Posted | Number | 95% Confidence Interval | Proportion of participants | Month 15 |
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of New Motor Milestones Achieved Per Participant | New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. | Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data. | Posted | Mean | Standard Deviation | milestones achieved | Month 15 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Revised Upper Limb Module (RULM) Test | The RULM Test is used in patients with SMA to assess upper limb functional ability items that are reflective of activities of daily living (i.e., raise a can to mouth as if drinking, take a coin and place it in a box, remove the lid of a container). The RULM test has a total of 20 items with an entry item that serves as functional class identification and does not contribute to the total score. The remaining 19 scorable items reflect different functional domains and are graded on a 3-point system with a score of 0 (unable), 1 (able, with modification), and a maximum of 2 (able, no difficulty). There is only 1 item (item I) that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for a total score range of 0-37, with higher scores increased great upper limb function. A positive change from Baseline indicates improvement. | ITT Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Missing postbaseline data were imputed using multiple imputation. | Posted | Least Squares Mean | 95% Confidence Interval | scores on a scale | Baseline and Month 15 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Proportion of Participants That Achieved Standing Alone | If the participant was unable to achieve standing alone at Baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder. | Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data. | Posted | Number | 95% Confidence Interval | Proportion of participants | Month 15 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Proportion of Participants That Achieved Walking With Assistance | If the participant was unable to achieve walking with assistance at baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder. | Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data. | Posted | Number | 95% Confidence Interval | Proportion of participants | Month 15 |
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| Secondary | Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death; an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity. Any other medically important event that, in the opinion of the investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. All participants with AEs were reported in this outcome measure, whereas in Adverse Event section there was at 5% reporting threshold to be met. | Posted | Number | participants | Baseline through Month 15 |
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| Secondary | Number of Participants With Clinically Significant Vital Sign Abnormalities | Vital signs assessed for clinical significance include resting blood pressure, pulse, respiratory rate, and temperature. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Any new or worsening vital sign findings were reported as AEs and are presented in the AE/SAE section of the results. | Posted | Number | participants | Baseline through Month 15 |
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| Secondary | Number of Participants With Clinically Significant Weight Abnormalities | Weight changes assessed from Baseline to Month 15. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Any new or worsening weight abnormality findings were reported as AEs and are presented in the AE/SAE section of the results. | Posted | Number | participants | Baseline through Month 15 |
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| Secondary | Number of Participants With Clinically Significant Neurological Examination Abnormalities | Neurological changes assessed for clinical significance include assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Neurological examination clinical significance was not collected. | Posted | Baseline through Month 15 |
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| Secondary | Number of Participants With Clinically Significant Physical Examination Abnormalities | Physical examination changes were assessed for clinical significance. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Physical examination clinical significance was not collected. | Posted | Baseline through Month 15 |
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| Secondary | Number of Participants With Clinically Significant Laboratory Parameter Abnormalities | Laboratory parameter changes assessed for clinical significance include serum chemistry, hematology, coagulation and urinalysis. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Any new or worsening clinical laboratory parameter findings were reported as AEs and are presented in the AE/SAE section of the results. | Posted | Number | participants | Baseline through Month 15 |
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| Secondary | Number of Participants With Abnormal, Clinically Relevant Post-Baseline Worsening in Electrocardiogram (ECG) in Results | The number of participants with abnormal, clinically relevant worsening, defined as participants with an ECG interpreted as abnormal and clinically relevant, with a comparison with Baseline value is reported. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. | Posted | Number | participants | Baseline through Month 15 |
|
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| Secondary | Number of Participants Taking Any Concomitant Medication Related to Dosing Procedure or Sham Procedure | Concomitant medications include prescription and over-the-counter medications administered to participants on or after the first day of study treatment. | Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. | Posted | Number | participants | Baseline through Month 15 |
|
|
From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
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 | Sham Procedure | Sham comparator on Days 1, 29, 85 and 274. | 0 | 42 | 12 | 42 | 42 | 42 |
| EG001 | Nusinersen | Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274. | 0 | 84 | 14 | 84 | 75 | 84 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal distension | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia adenoviral | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia parainfluenzae viral | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory syncytial virus bronchitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Joint contracture | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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After the interim analysis of the study, the decision was made in October 2016 to terminate the study early and participants were invited for end-of-study visits.
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 | |
|---|---|---|---|---|
| Biogen Study Medical Director | Biogen | 866-633-4636 | clinicaltrials@biogen.com |
| SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Jun 30, 2016 | Jan 23, 2018 | Prot_001.pdf |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000590926 | nusinersen |
Not provided
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Asian |
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| Black |
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| White |
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| Multiple |
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| Units | Counts |
|---|---|
| Participants |
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