Trial · NCT02594124
Registered outcomes32
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 32
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to the end of the study (up to 2848 days) | 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. |
| Primary | Number of Participants With Vital Sign Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | The vital sign assessments included blood pressure, temperature, pulse rate, and respiratory rate. Participants with abnormalities in these assessments recorded as AEs were reported. |
| Primary | Number of Participants With Weight Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | 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. |
| Primary | Number of Participants With Neurological Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | Participants with abnormalities in neurological examinations recorded as AEs were reported. |
| Primary | Number of Participants With Laboratory Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | Laboratory investigations included hematology, coagulation, serum chemistry and urinalysis parameters. Participants with abnormalities in these laboratory investigations recorded as AEs were reported. |
| Primary | Number of Participants With Coagulation Parameters Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | 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. |
| Primary | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | From Day 1 up to the end of the study (up to 2848 days) | Clinical significance of abnormalities in 12 lead ECG was determined based on the investigator's discretion. |
| Primary | Number of Participants Taking Any Concomitant Medication | From Day 1 up to the end of the study (up to 2848 days) | 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. |
| Secondary | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | MMDR Period: At Day 1800 | 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. |
| Secondary | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | At Day 309 | 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. |
| Secondary | Number of Participants Who Died or Met Permanent Ventilation | MMDR Period: Up to Day 1800 | 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. |
| Secondary | Number of Participants Not Requiring Permanent Ventilation | MMDR Period: Up to Day 1800 | 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. |
| Secondary | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Baseline, Day 2198 | 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. |
| Secondary | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | MMDR Period: Baseline, MMDR Day 1800 | 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. |
| Secondary | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | MMDR Period: Baseline, MMDR Day 1800 | 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. |
| Secondary | Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT) | Baseline, Day 2670 | 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. |
| Secondary | Number of Participants Who Experienced Contracture Assesment | MMDR Period: At MMDR Day 1800 | 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. |
| Secondary | Change From Baseline in Compound Muscular Action Potential (CMAP) | MMDR Period: Baseline, MMDR Day 1800 | 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. |
| Secondary | Change From Baseline in Body Length | MMDR Period: up to Day 1800 | 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. |
| Secondary | Change From Baseline in Weight | MMDR Period: up to Day 1800 | 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. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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