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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-002098-12 | EudraCT Number |
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The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nusinersen | Experimental | Nusinersen administered as an intrathecal injection |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nusinersen | Drug | Solution for intrathecal injection |
|
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| Measure | Description | Time Frame |
|---|---|---|
| Time to Death or Respiratory Intervention | The time was the age of the participant at the first occurrence of either a respiratory intervention or death. Respiratory intervention was defined as invasive or noninvasive ventilation for ≥6 hours/day continuously for 7 or more days OR tracheostomy. | Screening up to Day 2891 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants Developing Clinically Manifested Spinal Muscular Atrophy (SMA) | A participant was considered having clinically manifested SMA if any of the following occurred:
|
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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 |
|---|---|---|---|---|---|---|
| David Geffen School of Medicine | Los Angeles | California | 90095 | United States | ||
| University of California Davis Health System |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37409780 | Derived | Crawford TO, Swoboda KJ, De Vivo DC, Bertini E, Hwu WL, Finkel RS, Kirschner J, Kuntz NL, Nazario AN, Parsons JA, Pechmann A, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Zhu C, Raynaud S, Lago TR, Paradis AD, Foster R, Chin R, Berger Z; NURTURE Study Group. Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study. Muscle Nerve. 2023 Aug;68(2):157-170. doi: 10.1002/mus.27853. Epub 2023 Jul 6. | |
| 27939059 |
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A total of 25 participants diagnosed with Spinal Muscular Atrophy (SMA) were enrolled in the study.
Participants took part in the multiple investigative sites from 18 May 2015 to 17 Dec 2024.
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| ID | Title | Description |
|---|---|---|
| FG000 | ISIS 396443 2 SMN2 Copies | Participants with 2 survival motor neuron 2 (SMN2) copies received 12 milligrams (mg) nusinersen, intrathecally (IT) on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. |
| FG001 |
| 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 | Oct 17, 2021 | Aug 25, 2025 |
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| At 13 and 24 months of age |
| Percentage of Participants Alive | Up to 8 years of age |
| Percentage of Participants Who Attained Motor Milestones Assessed as Part of the Hammersmith Infant Neurological Examination (HINE) | HINE is evaluated in infants between 2-24 months of age. It's a simple, standardized instrument including 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of HINE (HINE-2) was assessed, which evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform task and score of 2, 3, or 4 indicating full milestone development. Total score is calculated by summing item scores to give maximum possible score of 26. Higher score indicates good neurological function. | Day 700 |
| Percentage of Participants Who Attained Motor Milestones as Assessed by World Health Organization (WHO) Criteria | The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support, standing with assistance, hands and knees crawling, walking with assistance, standing alone and walking alone. | Baseline up to Day 2891 |
| Change From Baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | The CHOP-INTEND test was designed to evaluate the motor skills of infants with significant motor weakness. Participants who were ≥2 years were continued to be assessed until a CHOP INTEND maximum score of 64 was achieved. It included 16 items (capturing neck, trunk, and proximal and distal limb strength), nine of which were scored 0, 1, 2, 3, or 4, five were scored as 0, 2, or 4, one was scored as 0, 1, 2, or 4, and one as 0, 2, 3, or 4 with higher scores indicating greater muscle strength and function. Total score was calculated as the sum of scores for each item. Total score ranged from 0 (worst possible score) and 64 (best possible score). CHOP-INTEND assessments were discontinued once participants achieved a maximum score of 64, so the number of participants with available data points decreased over time. | Baseline, Day 2891 |
| Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) | 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 and ranged from 0 to 66. Higher scores indicate increased motor function. Baseline was defined as the time of first HFMSE score after Day 700. | Baseline, Day 2160 |
| Change From Baseline in Weight for Age | The World Health Organization (WHO) child growth standards for participants aged up to 10 years was used to determine the percentiles. WHO Anthro software was used to calculate the percentiles for the given weights of each child. Negative change from baseline indicates low weight for age percentile. | Baseline, Day 2891 |
| Change From Baseline in Weight for Length | The WHO child growth standards for participants aged up to 10 years was used to determine the percentiles. WHO Anthro software was used to calculate the percentiles for the given weights of each child. | Baseline, Day 1849 |
| Change From Baseline in Head Circumference | Baseline, Day 2891 |
| Change From Baseline in Chest Circumference | Baseline, Day 2891 |
| Change From Baseline in Head to Chest Circumference Ratio | Negative change from baseline indicates reduction in head-to-chest circumference ratio. | Baseline, Day 2891 |
| Change From Baseline in Arm Circumference | Baseline, Day 2891 |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE was any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. | From the signing of the informed consent form (ICF) up to the end of the study (up to Day 2891) |
| Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters) | Hematology parameters included hemoglobin, hematocrit, erythrocytes, platelets, leukocytes, neutrophils, eosinophils, basophils, lymphocytes, and monocytes count. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicates values that were normal, low or unknown at baseline and shifted to high postbaseline values. | Baseline up to Day 2891 |
| Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Blood chemistry parameters included bilirubin (direct and indirect), alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, creatinine, sodium, potassium, chloride, protein, albumin, calcium, phosphate, glucose, cystatin C, creatine kinase. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicates values that were normal, low or unknown at baseline and shifted to high postbaseline values. | Baseline up to Day 2891 |
| Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Urinalysis Parameters) | Urinalysis included assessments of specific gravity, pH, protein, glucose, ketones, bilirubin, occult blood, erythrocytes, leukocytes, epithelial cells, bacteria, casts and crystals. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicates values that were normal, low or unknown at baseline and shifted to high postbaseline values. | Baseline up to Day 2891 |
| Number of Participants With Shifts From Baseline in Coagulation Parameters [Activated Partial Thromboplastin Time (aPTT)] | aPTT was evaluated to assess safety. Shift to high measured change in normal, low and unknown values at baseline to high values postbaseline. | Baseline up to Day 2891 |
| Number of Participants With Shifts From Baseline in Coagulation Parameters [Prothrombin Time (PT)] | PT was evaluated to assess safety. Shift to high measured change in normal, low and unknown values at baseline to high values postbaseline. | Baseline up to Day 2891 |
| Number of Participants With Shifts From Baseline in Coagulation Parameters [International Normalized Ratio (INR)] | INR was evaluated to assess safety. Shift to high measured change in normal, low and unknown values at baseline to high values postbaseline. | Baseline up to Day 2891 |
| Percentage of Participants With Clinically Significant Shifts in Electrocardiograms (ECG) Abnormalities | Clinical significance of abnormalities in ECG was determined based on the investigator's discretion. Shift to abnormal indicated values that were normal or unknown at baseline and shifted to abnormal values post-baseline. | Baseline up to Day 2891 |
| Change From Baseline in Vital Signs (Temperature) | Negative change from baseline indicates reduction in temperature. | Baseline, Day 2891 |
| Change From Baseline in Vital Signs (Blood Pressure) | Baseline, Day 2891 |
| Change From Baseline in Vital Signs (Heart Rate) | Negative change from baseline indicates reduction in heart rate. | Baseline, Day 2891 |
| Change From Baseline in Vital Signs (Respiratory Rate) | Negative change from baseline indicates reduction in respiratory rate. | Baseline, Day 2891 |
| Number of Participants With Neurological Examination Abnormalities Reported as AEs | Participants with abnormalities in neurological examinations recorded as AEs were reported. | From the signing of the ICF up to the end of the study (up to Day 2891) |
| Cerebrospinal Fluid (CSF) Concentration of Nusinersen | Predose on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, 2801 |
| Plasma Concentration of Nusinersen | Predose on Days 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, 2801 and 4-hour post-dose on Day 1 |
| Sacramento |
| California |
| 95817 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Nemours Children's Hospital, Orlando | Orlando | Florida | 32827 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611-2605 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21205 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Columbia University | New York | New York | 10032 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| University of Utah | Salt Lake City | Utah | 84112 | United States |
| Seattle Children's Research Institute | Seattle | Washington | 98101 | United States |
| Queensland Children's Hospital | South Brisbane | Queensland | 4101 | Australia |
| Royal Children's Hospital | Parkville | Victoria | 3052 | Australia |
| Universitaetsklinikum Freiburg | Freiburg im Breisgau | Baden-Wurttemberg | 79106 | Germany |
| Ospedale Pediatrico Bambino Gesù | Rome | Lazio | 165 | Italy |
| Fondazione Serena Onlus - Centro Clinico Nemo | Milan | 20162 | Italy |
| Hamad General Hospital | Doha | 3050 | Qatar |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | 807 | Taiwan |
| National Taiwan University Hospital | Taipei | 100 | Taiwan |
| Hacettepe University Medical Faculty | Ankara | 6230 | Turkey (Türkiye) |
| Yeditepe University Medical School Hospital | Istanbul | 31755 | Turkey (Türkiye) |
| Derived |
| Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7. |
| ISIS 396443 3 SMN2 Copies |
Participants with 3 SMN2 copies received 12 mg nusinersen, IT on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. |
| COMPLETED |
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| NOT COMPLETED |
|
|
The Intent-to-treat (ITT) set included all participants who received at least 1 dose of ISIS 396443.
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| ID | Title | Description |
|---|---|---|
| BG000 | ISIS 396443 2 SMN2 Copies | Participants with 2 SMN2 copies received 12 mg nusinersen, IT on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. |
| BG001 | ISIS 396443 3 SMN2 Copies | Participants with 3 SMN2 copies received 12 mg nusinersen, IT on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | days |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| 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 | Time to Death or Respiratory Intervention | The time was the age of the participant at the first occurrence of either a respiratory intervention or death. Respiratory intervention was defined as invasive or noninvasive ventilation for ≥6 hours/day continuously for 7 or more days OR tracheostomy. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis. | Posted | Median | 95% Confidence Interval | months | Screening up to Day 2891 |
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| Secondary | Proportion of Participants Developing Clinically Manifested Spinal Muscular Atrophy (SMA) | A participant was considered having clinically manifested SMA if any of the following occurred:
| The ITT set included all participants who received at least 1 dose of ISIS 396443. | Posted | Number | 95% Confidence Interval | proportion of participants | At 13 and 24 months of age |
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| Secondary | Percentage of Participants Alive | The ITT set included all participants who received at least 1 dose of ISIS 396443. | Posted | Number | percentage of participants | Up to 8 years of age |
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| Secondary | Percentage of Participants Who Attained Motor Milestones Assessed as Part of the Hammersmith Infant Neurological Examination (HINE) | HINE is evaluated in infants between 2-24 months of age. It's a simple, standardized instrument including 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of HINE (HINE-2) was assessed, which evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform task and score of 2, 3, or 4 indicating full milestone development. Total score is calculated by summing item scores to give maximum possible score of 26. Higher score indicates good neurological function. | The ITT set included all participants who received at least 1 dose of ISIS 396443. | Posted | Number | percentage of participants | Day 700 |
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| Secondary | Percentage of Participants Who Attained Motor Milestones as Assessed by World Health Organization (WHO) Criteria | The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support, standing with assistance, hands and knees crawling, walking with assistance, standing alone and walking alone. | The ITT set included all participants who received at least 1 dose of ISIS 396443. | Posted | Number | percentage of participants | Baseline up to Day 2891 |
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| Secondary | Change From Baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | The CHOP-INTEND test was designed to evaluate the motor skills of infants with significant motor weakness. Participants who were ≥2 years were continued to be assessed until a CHOP INTEND maximum score of 64 was achieved. It included 16 items (capturing neck, trunk, and proximal and distal limb strength), nine of which were scored 0, 1, 2, 3, or 4, five were scored as 0, 2, or 4, one was scored as 0, 1, 2, or 4, and one as 0, 2, 3, or 4 with higher scores indicating greater muscle strength and function. Total score was calculated as the sum of scores for each item. Total score ranged from 0 (worst possible score) and 64 (best possible score). CHOP-INTEND assessments were discontinued once participants achieved a maximum score of 64, so the number of participants with available data points decreased over time. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) | 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 and ranged from 0 to 66. Higher scores indicate increased motor function. Baseline was defined as the time of first HFMSE score after Day 700. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 2160 |
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| Secondary | Change From Baseline in Weight for Age | The World Health Organization (WHO) child growth standards for participants aged up to 10 years was used to determine the percentiles. WHO Anthro software was used to calculate the percentiles for the given weights of each child. Negative change from baseline indicates low weight for age percentile. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | percentile | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Weight for Length | The WHO child growth standards for participants aged up to 10 years was used to determine the percentiles. WHO Anthro software was used to calculate the percentiles for the given weights of each child. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis. 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | percentile | Baseline, Day 1849 |
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| Secondary | Change From Baseline in Head Circumference | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | centimeter (cm) | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Chest Circumference | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | cm | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Head to Chest Circumference Ratio | Negative change from baseline indicates reduction in head-to-chest circumference ratio. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | ratio | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Arm Circumference | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | cm | Baseline, Day 2891 |
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| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE was any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. | The ITT set included all participants who received at least 1 dose of ISIS 396443. | Posted | Count of Participants | Participants | From the signing of the informed consent form (ICF) up to the end of the study (up to Day 2891) |
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| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters) | Hematology parameters included hemoglobin, hematocrit, erythrocytes, platelets, leukocytes, neutrophils, eosinophils, basophils, lymphocytes, and monocytes count. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicates values that were normal, low or unknown at baseline and shifted to high postbaseline values. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis of the specified hematology parameter. | Posted | Count of Participants | Participants | Baseline up to Day 2891 |
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| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Blood chemistry parameters included bilirubin (direct and indirect), alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, creatinine, sodium, potassium, chloride, protein, albumin, calcium, phosphate, glucose, cystatin C, creatine kinase. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicates values that were normal, low or unknown at baseline and shifted to high postbaseline values. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis of the specified blood chemistry parameter. | Posted | Count of Participants | Participants | Baseline up to Day 2891 |
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| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Urinalysis Parameters) | Urinalysis included assessments of specific gravity, pH, protein, glucose, ketones, bilirubin, occult blood, erythrocytes, leukocytes, epithelial cells, bacteria, casts and crystals. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicates values that were normal, low or unknown at baseline and shifted to high postbaseline values. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis of the specified urinalysis parameter. | Posted | Count of Participants | Participants | Baseline up to Day 2891 |
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| Secondary | Number of Participants With Shifts From Baseline in Coagulation Parameters [Activated Partial Thromboplastin Time (aPTT)] | aPTT was evaluated to assess safety. Shift to high measured change in normal, low and unknown values at baseline to high values postbaseline. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis. | Posted | Count of Participants | Participants | Baseline up to Day 2891 |
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| Secondary | Number of Participants With Shifts From Baseline in Coagulation Parameters [Prothrombin Time (PT)] | PT was evaluated to assess safety. Shift to high measured change in normal, low and unknown values at baseline to high values postbaseline. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis. | Posted | Count of Participants | Participants | Baseline up to Day 2891 |
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| Secondary | Number of Participants With Shifts From Baseline in Coagulation Parameters [International Normalized Ratio (INR)] | INR was evaluated to assess safety. Shift to high measured change in normal, low and unknown values at baseline to high values postbaseline. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis. | Posted | Count of Participants | Participants | Baseline up to Day 2891 |
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| Secondary | Percentage of Participants With Clinically Significant Shifts in Electrocardiograms (ECG) Abnormalities | Clinical significance of abnormalities in ECG was determined based on the investigator's discretion. Shift to abnormal indicated values that were normal or unknown at baseline and shifted to abnormal values post-baseline. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Overall number of participants analyzed signifies the number of participants with post-baseline ECG abnormalities. | Posted | Number | percentage of participants | Baseline up to Day 2891 |
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| Secondary | Change From Baseline in Vital Signs (Temperature) | Negative change from baseline indicates reduction in temperature. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | degree Celsius | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Vital Signs (Blood Pressure) | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | millimeters of mercury (mmHg) | Baseline, Day 2891 |
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| Secondary | Change From Baseline in Vital Signs (Heart Rate) | Negative change from baseline indicates reduction in heart rate. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | beats per minute (beats/min) | Baseline, Day 2891 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Vital Signs (Respiratory Rate) | Negative change from baseline indicates reduction in respiratory rate. | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Mean | Standard Deviation | breaths per minute (breaths/min) | Baseline, Day 2891 |
|
| |||||||||||||||||||||||||||||
| Secondary | Number of Participants With Neurological Examination Abnormalities Reported as AEs | Participants with abnormalities in neurological examinations recorded as AEs were reported. | The ITT set included all participants who received at least 1 dose of ISIS 396443. | Posted | Count of Participants | Participants | From the signing of the ICF up to the end of the study (up to Day 2891) |
|
| ||||||||||||||||||||||||||||||
| Secondary | Cerebrospinal Fluid (CSF) Concentration of Nusinersen | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanograms per milliliter (ng/mL) | Predose on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, 2801 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Plasma Concentration of Nusinersen | The ITT set included all participants who received at least 1 dose of ISIS 396443. Here 'number analyzed' signifies the number of participants available for analysis at a specified time point. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/ml | Predose on Days 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, 2801 and 4-hour post-dose on Day 1 |
|
|
From the signing of the ICF up to the end of the study (up to Day 2891)
The ITT set included all participants who received at least 1 dose of ISIS 396443.
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 | ISIS 396443 2 SMN2 Copies | Participants with 2 SMN2 copies received 12 mg nusinersen, IT on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. | 0 | 15 | 10 | 15 | 15 | 15 |
| EG001 | ISIS 396443 3 SMN2 Copies | Participants with 3 SMN2 copies received 12 mg nusinersen, IT on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. | 0 | 10 | 4 | 10 | 10 | 10 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tachycardia | Cardiac disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia mycoplasmal | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia pneumococcal | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia respiratory syncytial viral | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory syncytial virus bronchiolitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Influenza a virus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Respirovirus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Feeding disorder | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tendon disorder | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hyperreflexia | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Medical device change | Surgical and medical procedures | MedDRA 27.1 | Systematic Assessment |
| |
| Tonsillectomy | Surgical and medical procedures | MedDRA 27.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypochromic anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lymphocytosis | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Neutrophilia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Cryptorchism | Congenital, familial and genetic disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Developmental hip dysplasia | Congenital, familial and genetic disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Epilepsy congenital | Congenital, familial and genetic disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypermobility syndrome | Congenital, familial and genetic disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Micrognathia | Congenital, familial and genetic disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Deafness bilateral | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Excessive cerumen production | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hyperacusis | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Motion sickness | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tympanic membrane perforation | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Anisocoria | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Astigmatism | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dacryostenosis acquired | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Eczema eyelids | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Eye discharge | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Heterophoria | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypermetropia | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Myopia | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Strabismus | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Breath odour | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Infantile colic | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lip pain | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lip swelling | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Malocclusion | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tooth loss | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Application site erythema | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Infusion site bruising | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Infusion site extravasation | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Medical device site rash | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Allergy to arthropod bite | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dust allergy | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Immunodeficiency | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Multiple allergies | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Atypical pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Bacterial labyrinthitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchiolitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchitis viral | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Covid-19 | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Dysentery | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Ear infection bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Ear infection staphylococcal | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Labyrinthitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Lice infestation | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Metapneumovirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Otitis media bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia moraxella | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory tract infection bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Stoma site infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Streptococcal infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Tinea infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Tinea pedis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Urinary tract infection bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral pharyngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral rash | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral rhinitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Vulvovaginal candidiasis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Accident | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Foreign body in respiratory tract | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Musculoskeletal procedural complication | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Nail injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Nasal injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Post procedural complication | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Post procedural discomfort | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Post procedural swelling | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Procedural vomiting | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Radial head dislocation | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Stoma site hypergranulation | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Stoma site pain | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Subcutaneous haematoma | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Urethral injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Venomous sting | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Adenovirus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Audiogram abnormal | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Blood calcium increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Blood glucose decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Bone density decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Breath sounds abnormal | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Candida test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Carnitine decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Crystal urine present | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Cystatin c increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Enterovirus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Eosinophil count increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Human rhinovirus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Lymphocyte count increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Nerve conduction studies abnormal | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Neutrophil count increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Platelet count increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Protein urine present | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Respirovirus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Sars-cov-2 test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Serratia test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Streptococcus test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Walking distance test abnormal | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Acidosis | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Feeding disorder | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Feeding intolerance | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Food intolerance | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lipid metabolism disorder | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Vitamin d deficiency | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Weight gain poor | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Acquired plagiocephaly | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Barrel chest | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Clubbing | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Coccydynia | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Foot deformity | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Growth failure | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Joint contracture | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Joint range of motion decreased | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Kyphosis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ligament laxity | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lordosis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Muscle atrophy | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tendon disorder | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Toe walking | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Torticollis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Trismus | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Winged scapula | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Seborrhoeic keratosis | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.1 | Systematic Assessment |
| |
| Areflexia | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Clonus | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Extensor plantar response | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Facial paresis | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Febrile convulsion | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Gross motor delay | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hyperreflexia | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypotonia | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Motor developmental delay | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Muscle contractions involuntary | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Myoclonus | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pleocytosis | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Speech disorder | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Speech disorder developmental | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Unresponsive to stimuli | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Umbilical granuloma | Pregnancy, puerperium and perinatal conditions | MedDRA 27.1 | Systematic Assessment |
| |
| Autism spectrum disorder | Psychiatric disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Automatism | Psychiatric disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Disturbance in social behaviour | Psychiatric disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Procedural anxiety | Psychiatric disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bladder hypertrophy | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Leukocyturia | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Vulvovaginal rash | Reproductive system and breast disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchial hyperreactivity | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchitis chronic | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Catarrh | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Cyanosis central | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Increased upper airway secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Lower respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Nasal septum deviation | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pharyngeal erythema | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pulmonary artery dilatation | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory acidosis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory muscle weakness | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Sputum retention | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Stridor | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Eczema nummular | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hidradenitis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ichthyosis acquired | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Miliaria | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Photosensitivity reaction | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Seborrhoeic dermatitis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Skin hypopigmentation | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Superficial inflammatory dermatosis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Aortic dilatation | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pallor | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
|
Our agreement is subject to confidentiality but generally the PI can publish, for non-commercial 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 | Dec 11, 2024 | Aug 25, 2025 | SAP_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 |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| White |
|
| Other |
|
| Not Reported Due to Confidentiality Regulations |
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Participants with 3 SMN2 copies received 12 mg nusinersen, IT on Days 1, 15, 29, 64, 183, 302, 421, 540, 659, 778, 897, 1016, 1135, 1254, 1373, 1492, 1611, 1730, 1849, 1968, 2087, 2206, 2325, 2444, 2563, 2682, and 2801. |
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