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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02594124
Registration number
NCT02594124
Ethics application status
Date submitted
30/10/2015
Date registered
1/11/2015
Date last updated
22/10/2024
Titles & IDs
Public title
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
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Scientific title
An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443
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Secondary ID [1]
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2015-001870-16
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Secondary ID [2]
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ISIS 396443-CS11
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Universal Trial Number (UTN)
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Trial acronym
SHINE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Neurological
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Other neurological disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional(has expanded access)
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Description of intervention(s) / exposure
Treatment: Drugs - nusinersen
Experimental: Group 1 - Participants transitioned from ISIS 396443-CS3B (NCT02193074)
Experimental: Group 2 - Participants transitioned from ISIS 396443-CS4 (NCT02292537)
Experimental: Group 3 - Participants transitioned from ISIS 396443-CS12 (NCT02052791)
Experimental: Group 4 - Participants transitioned from ISIS 396443-CS3A (NCT01839656)
Experimental: Group 5 - Participants transitioned from 232SM202 (NCT02462759)
Treatment: Drugs: nusinersen
Administered by intrathecal (IT) injection
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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AE:unfavorable and unintended sign, symptom, or disease temporally associated with study/use of an investigational drug, whether or not it's considered related to investigational drug. SAE:AE that in view of either Investigator/Sponsor, meets any of the following criteria: results in death;is life-threatening:i.e.poses risk of death, hospitalization/it's prolongation;results in a persistent or significant incapacity or substantial disruption of normal life functions;results in congenital anomaly or birth defect in offspring;is an important event in the opinion of Investigator/Sponsor. TEAE: if it was present prior to first dose of nusinersen or first sham procedure in index study and subsequently worsened in severity/was not present prior to first dose of nusinersen or first sham procedure in index study but subsequently appeared.
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Timepoint [1]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [2]
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Number of Participants With Vital Sign Abnormalities Reported as AEs
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Assessment method [2]
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The vital sign assessments included blood pressure, temperature, pulse rate, and respiratory rate. Participants with abnormalities in these assessments recorded as AEs were reported.
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Timepoint [2]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [3]
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Number of Participants With Weight Abnormalities Reported as AEs
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Assessment method [3]
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Weight decrease was characterized by a decrease of \>=7% from baseline and weight increase was characterized by an increase of \>=7% from baseline. Participants with these abnormalities recorded as AEs were reported.
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Timepoint [3]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [4]
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Number of Participants With Neurological Abnormalities Reported as AEs
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Assessment method [4]
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Participants with abnormalities in neurological examinations recorded as AEs were reported.
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Timepoint [4]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [5]
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Number of Participants With Laboratory Abnormalities Reported as AEs
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Assessment method [5]
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Laboratory investigations included hematology, coagulation, serum chemistry and urinalysis parameters. Participants with abnormalities in these laboratory investigations recorded as AEs were reported.
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Timepoint [5]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [6]
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Number of Participants With Coagulation Parameters Reported as AEs
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Assessment method [6]
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Coagulation parameters included activated partial thromboplastin time (aPTT) and international normalized ratio (INR). Participants with abnormalities in these coagulation parameters recorded as AEs were reported.
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Timepoint [6]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [7]
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Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results
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Assessment method [7]
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Clinical significance of abnormalities in 12 lead ECG was determined based on the investigator's discretion.
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Timepoint [7]
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From Day 1 up to the end of the study (up to 2848 days)
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Primary outcome [8]
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Number of Participants Taking Any Concomitant Medication
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Assessment method [8]
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A concomitant therapy is any non-protocol-specified drug or substance (including over-the-counter medications, herbal medications, and vitamin supplements) administered between the beginning of screening and the last telephone contact or study visit.
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Timepoint [8]
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From Day 1 up to the end of the study (up to 2848 days)
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Secondary outcome [1]
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Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria
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Assessment method [1]
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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. Mean of number of new milestones achieved was calculated and reported in this outcome measure.
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Timepoint [1]
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MMDR Period: At Day 1800
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Secondary outcome [2]
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Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE)
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Assessment method [2]
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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.
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Timepoint [2]
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At Day 309
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Secondary outcome [3]
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Number of Participants Who Died or Met Permanent Ventilation
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Assessment method [3]
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Permanent ventilation was defined as tracheostomy or \>=16 hours of ventilator support per day continuously for \>21 days in the absence of an acute reversible event.
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Timepoint [3]
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MMDR Period: Up to Day 1800
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Secondary outcome [4]
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Number of Participants Not Requiring Permanent Ventilation
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Assessment method [4]
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Permanent ventilation was defined as tracheostomy or \>=16 hours of ventilator support per day continuously for \>21 days in the absence of an acute reversible event.
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Timepoint [4]
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MMDR Period: Up to Day 1800
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Secondary outcome [5]
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Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale
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Assessment method [5]
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The CHOP INTEND test is designed to evaluate the motor skills of infants with significant motor weakness, including infants with SMA. Participants who are =2 years will be continued to be assessed until a CHOP INTEND maximum score of 64 is achieved. It includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0 (worst) to 4 (best). Total scores range from 0 to 64, with higher scores indicating better movement functioning.
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Timepoint [5]
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Baseline, Day 2198
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Secondary outcome [6]
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Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score
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Assessment method [6]
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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.
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Timepoint [6]
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MMDR Period: Baseline, MMDR Day 1800
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Secondary outcome [7]
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Change From Baseline in Revised Upper Limb Module (RULM) Total Score
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Assessment method [7]
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RULM Test is used in participants with SMA to assess upper limb functional ability items and has total of 20 items with an entry item that serves as functional class identification and does not contribute to total score. Remaining 19 scorable items reflect different functional domains and graded on 3-point system with score of 0 (unable), 1 (able, with modification), and 2 (able, no difficulty). There is only 1 item that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for total score (0-37), higher scores indicating increased upper limb function. Positive change from baseline indicates improvement.
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Timepoint [7]
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MMDR Period: Baseline, MMDR Day 1800
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Secondary outcome [8]
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Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT)
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Assessment method [8]
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The 6MWT measures the distance an individual can walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes.
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Timepoint [8]
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Baseline, Day 2670
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Secondary outcome [9]
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Number of Participants Who Experienced Contracture Assesment
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Assessment method [9]
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Contracture assessment is performed to assess the motor performance in SMA. The number of participants who experienced at least one contracture at any location and severe contractures in any of the five locations (hip flexors, knee flexors, ankle planter flexors, elbow flexors, forearm flexors) are reported in this outcome measure.
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Timepoint [9]
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MMDR Period: At MMDR Day 1800
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Secondary outcome [10]
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Change From Baseline in Compound Muscular Action Potential (CMAP)
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Assessment method [10]
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CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. Peroneal amplitude (PA) and ulnar amplitude (UA) data is reported in this OM. Score \<0 indicated worse response and \>0 indicated better response than the normal matched population. Score change \<0 indicated worsening and \>0 indicated improvement as compared to baseline.
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Timepoint [10]
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MMDR Period: Baseline, MMDR Day 1800
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Secondary outcome [11]
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Change From Baseline in Body Length
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Assessment method [11]
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Participants were analyzed for change in growth parameter of body length to evaluate clinical efficacy. The body length was calculated using either WHO or Centers for Disease Control and Prevention (CDC) scales. The CDC scale allows to calculate the body length up to 20 years, while the WHO scale allows to calculate it only up to 10 years.
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Timepoint [11]
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MMDR Period: up to Day 1800
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Secondary outcome [12]
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Change From Baseline in Weight
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Assessment method [12]
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Participants were analyzed for change in growth parameter of weight to evaluate clinical efficacy. The weight was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight up to 20 years, while the WHO scale allows to calculate it only up to 10 years.
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Timepoint [12]
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MMDR Period: up to Day 1800
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Secondary outcome [13]
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Change From Baseline in Weight for Age Percentile
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Assessment method [13]
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Participants who were below the age of 36 months were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. The weight for age percentile was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight for age percentile up to 20 years, while the WHO scale allows to calculate it only up to 10 years.
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Timepoint [13]
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MMDR Period: up to Day 1800
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Secondary outcome [14]
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Percentage of CMAP Responders
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Assessment method [14]
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CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. A participant was defined as a responder if they had a peroneal amplitude =1 mV at last visit (including the amplitude =1 mV at baseline and also demonstrated as such at last visit).
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Timepoint [14]
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MMDR Period: At Day 1800
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Secondary outcome [15]
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Number of Participants Who Achieved Motor Milestones
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Assessment method [15]
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Motor milestones were measured based on 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 (SWS), standing with assistance (SWA), hands and knees crawling (HKC), and walking alone (WA).
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Timepoint [15]
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MMDR Period: up to Day 1800
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Secondary outcome [16]
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Number of Participants Who Achieved Standing Alone and Walking With Assistance
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Assessment method [16]
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Motor milestones were measured based on 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: SWS, SWA, HKC, WWA, WA and standing alone (SA). SA and WWA were assessed in this outcome measure.
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Timepoint [16]
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MMDR Period: up to Day 1800
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Secondary outcome [17]
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Total Number of Hospitalizations Due to Serious Respiratory Events
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Assessment method [17]
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Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization.
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Timepoint [17]
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Up to day 2520
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Secondary outcome [18]
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Total Number of Hospitalizations Due to Serious Adverse Events
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Assessment method [18]
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Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization.
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Timepoint [18]
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Up to day 2520
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Secondary outcome [19]
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Percent of Time in Hospitalization
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Assessment method [19]
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Timepoint [19]
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Upto day 2160
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Secondary outcome [20]
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Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit
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Assessment method [20]
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Cobb angle is a measurement of the degree of side-to-side spinal curvature used to define scoliosis.
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Timepoint [20]
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MMDR Period: Baseline, MMDR Day 1800
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Secondary outcome [21]
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Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain
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Assessment method [21]
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Items on the PedsQL generic core scale are reverse scored and transformed to a 0-100 scale. The PedsQL parent (P) and self (S) reported questionnaire was collected for participants from 2 to 25 years of age. Four dimensions were collected: Physical, Emotional, Social and School functioning and each item was scored on a 5 point ordinal scale. 0 (never) =100, 1 (almost never) = 75, 2 (sometimes)= 50, 3 (often) = 25, 4 (almost always) = 0. A total score was calculated as the sum of all the items over the number of items answered on all the scales. If more than 50% of items or more were missing, the scale score was not computed. Higher scores indicated better health related quality of life.
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Timepoint [21]
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MMDR Period: At Day 1800
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Secondary outcome [22]
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Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score
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Assessment method [22]
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The ACEND is a questionnaire that includes a total of seven domains assessing physical impact (including feeding/grooming/dressing, sitting/play, transfers, and mobility) and general caregiver impact (including time, emotion, and finance) and each domain comprises several items. The total score (TS) for each domain will be calculated on a scale of 0 to 100. Higher scores indicate a greater impact on the caregiver.
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Timepoint [22]
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MMDR Period: up to Day 1800
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Secondary outcome [23]
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Number of Participants With Disease-related Hospitalizations and AEs
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Assessment method [23]
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Timepoint [23]
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MMDR Period: up to Day 1800
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Secondary outcome [24]
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Survival Rate
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Assessment method [24]
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Survival rate was defined as the percentage of participants alive during the study and was estimated from the Kaplan Meier (KM) curve for time to death.
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Timepoint [24]
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MMDR Period: up to Day 1800
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Eligibility
Key inclusion criteria
Key
* Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines.
* Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
* Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
* Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
* Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/11/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
21/08/2023
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Sample size
Target
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Accrual to date
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Final
292
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Sydney Children's Hospital Clinical Research Centre - Sydney
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Recruitment hospital [2]
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Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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2031 - Sydney
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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Colorado
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0
0
United States of America
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Connecticut
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0
0
United States of America
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Florida
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0
United States of America
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Illinois
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0
0
United States of America
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Maryland
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0
0
United States of America
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State/province [7]
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Massachusetts
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Country [8]
0
0
United States of America
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State/province [8]
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Minnesota
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0
0
United States of America
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State/province [9]
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Missouri
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0
0
United States of America
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New York
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0
0
United States of America
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State/province [11]
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North Carolina
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0
0
United States of America
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State/province [12]
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Oregon
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0
0
United States of America
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Pennsylvania
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0
0
United States of America
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Texas
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Country [15]
0
0
United States of America
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Utah
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Country [16]
0
0
United States of America
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State/province [16]
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Washington
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0
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Belgium
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Brussels
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0
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Canada
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British Columbia
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Country [19]
0
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Canada
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State/province [19]
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Ontario
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Country [20]
0
0
Canada
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State/province [20]
0
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Quebec
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Country [21]
0
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France
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State/province [21]
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Paris 9
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Country [22]
0
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Germany
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Bayern
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Germany
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0
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Essen
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0
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Germany
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State/province [24]
0
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Freiburg
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Country [25]
0
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Hong Kong
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State/province [25]
0
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Hong Kong SAR
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Country [26]
0
0
Italy
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State/province [26]
0
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Essen
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0
0
Italy
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0
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Genova
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0
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Italy
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0
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Messina
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0
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Japan
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0
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Aichi
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0
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Japan
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0
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Hyogo
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0
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Japan
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0
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Tokyo
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0
0
Japan
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0
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Kumamoto
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0
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Japan
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0
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Miyagi
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0
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Japan
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Miyazaki
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0
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Korea, Republic of
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Korea
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0
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Spain
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Barcelona
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Spain
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Hebron
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0
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Spain
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Madrid
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Sweden
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State/province [39]
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Gothenburg
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0
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Turkey
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0
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Ankara
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0
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Turkey
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0
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Istanbul
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0
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United Kingdom
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Northumberland
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United Kingdom
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State/province [43]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Biogen
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Summary
Brief summary
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.
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Trial website
https://clinicaltrials.gov/study/NCT02594124
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Trial related presentations / publications
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Public notes
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Contacts
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Medical Director
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Biogen
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
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Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/24/NCT02594124/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/24/NCT02594124/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02594124
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