Trial · NCT02594124
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
NCT02594124, Phase 3, Completed, Biogen; results first posted 2024-10-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. Trials, record NCT02594124. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02594124
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
- NCT number
- NCT02594124
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 292 participants, actual
- Lead sponsor
- Biogen
- Conditions
- Spinal Muscular Atrophy
- Interventions
- nusinersen
- First posted
- 2015-11-02
- Last updated
- 2024-10-22
- Other registries' IDs
- eudract:2015-001870-16
- Results first posted
- 2024-10-22
Source ClinicalTrials.gov
Registered outcomes32
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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. |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 registered outcomesOutcomes32
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| 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) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. | Participants | COUNT_OF_PARTICIPANTS |
| 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 safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Weight Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. ' | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Neurological Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Coagulation Parameters Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. | Participants | COUNT_OF_PARTICIPANTS |
| 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) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Number analyzed' signifies number of participants evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Taking Any Concomitant Medication | From Day 1 up to the end of the study (up to 2848 days) | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | MMDR Period: At Day 1800 | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. | motor milestone | MEAN |
| 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 | The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. Only CS3A and CS3B arm groups were planned to be analyzed for this outcome measure (OM). | percentage of participants | NUMBER |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 outcomesResults by group1,465
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Infantile SMA Onset CS3A | AEs | 13 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Infantile SMA Onset CS3B Previous Control | AEs | 24 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Infantile SMA Onset CS3B Previous ISIS 396443 | AEs | 65 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Infantile SMA Onset 232SM202 | AEs | 12 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Later SMA Onset CS12 Type 2 | AEs | 20 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Later SMA Onset CS12 Type 3 | AEs | 25 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Later SMA Onset CS4 Previous Control | AEs | 42 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Later SMA Onset CS4 Previous ISIS 396443 | AEs | 80 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Later SMA Onset 232SM202 | AEs | 8 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Infantile SMA Onset CS3A | SAEs | 11 | Participants |
1 to 10 of 1,465Source ClinicalTrials.gov
See all 1,465 results by groupParticipant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Infantile SMA Onset CS3A | 13 | |
| Overall Study | STARTED | Infantile SMA Onset CS3B Previous Control | 24 | |
| Overall Study | STARTED | Infantile SMA Onset CS3B Previous ISIS 396443 | 65 | |
| Overall Study | STARTED | Infantile SMA Onset 232SM202 | 12 | |
| Overall Study | STARTED | Later SMA Onset CS12 Type 2 | 20 | |
| Overall Study | STARTED | Later SMA Onset CS12 Type 3 | 25 | |
| Overall Study | STARTED | Later SMA Onset CS4 Previous Control | 42 | |
| Overall Study | STARTED | Later SMA Onset CS4 Previous ISIS 396443 | 83 | |
| Overall Study | STARTED | Later SMA Onset 232SM202 | 8 | |
| Overall Study | COMPLETED | Infantile SMA Onset CS3A | 10 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events5,166
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Infantile SMA Onset CS3A | 1 | 13 | |
| other | Anaemia | Blood and lymphatic system disorders | Infantile SMA Onset CS3B Previous Control | 0 | 24 | |
| other | Anaemia | Blood and lymphatic system disorders | Infantile SMA Onset CS3B Previous ISIS 396443 | 6 | 65 | |
| other | Anaemia | Blood and lymphatic system disorders | Infantile SMA Onset 232SM202 | 1 | 12 | |
| other | Anaemia | Blood and lymphatic system disorders | Later SMA Onset 232SM202 | 1 | 8 | |
| other | Anaemia | Blood and lymphatic system disorders | Later SMA Onset CS12 Type 2 | 2 | 20 | |
| other | Anaemia | Blood and lymphatic system disorders | Later SMA Onset CS12 Type 3 | 0 | 25 | |
| other | Anaemia | Blood and lymphatic system disorders | Later SMA Onset CS4 Previous Control | 1 | 42 | |
| other | Anaemia | Blood and lymphatic system disorders | Later SMA Onset CS4 Previous ISIS 396443 | 1 | 83 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Infantile SMA Onset CS3A | 0 | 13 |
1 to 10 of 5,166Source ClinicalTrials.gov
See all 5,166 adverse eventsAdverse event totals27
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Infantile SMA Onset CS3A | 0 | 13 |
| Total, all-cause mortality | Infantile SMA Onset CS3B Previous Control | 5 | 24 |
| Total, all-cause mortality | Infantile SMA Onset CS3B Previous ISIS 396443 | 8 | 65 |
| Total, all-cause mortality | Infantile SMA Onset 232SM202 | 1 | 12 |
| Total, all-cause mortality | Later SMA Onset 232SM202 | 0 | 8 |
| Total, all-cause mortality | Later SMA Onset CS12 Type 2 | 0 | 20 |
| Total, all-cause mortality | Later SMA Onset CS12 Type 3 | 0 | 25 |
| Total, all-cause mortality | Later SMA Onset CS4 Previous Control | 1 | 42 |
| Total, all-cause mortality | Later SMA Onset CS4 Previous ISIS 396443 | 1 | 83 |
| Total, other adverse events | Infantile SMA Onset CS3A | 13 | 13 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38705943 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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