Trial · NCT04089566
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
NCT04089566, Phase 3, Completed, Biogen; results first posted 2025-06-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy. Trials, record NCT04089566. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04089566
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
- NCT number
- NCT04089566
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 145 participants, actual
- Lead sponsor
- Biogen
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Nusinersen
- First posted
- 2019-09-13
- Last updated
- 2025-06-05
- Other registries' IDs
- eudract:2019-002663-10
- Results first posted
- 2025-06-05
Source ClinicalTrials.gov
Registered outcomes79
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part B Infantile-onset SMA: Change From Baseline in CHOP-INTEND Total Score for 50/28mg Nusinersen Versus CS3B Matched Sham Control Group | Baseline, Day 183 | The CHOP-INTEND test was designed to evaluate the motor skills of infants with significant motor weakness. 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). The change from baseline to Day 183 in the CHOP-INTEND total score was compared to CS3B study (NCT02193074) sham control group using the joint-rank methodology to account for mortality. |
| Primary | Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) | Part A: From the first dose of the study drug up to Day 389, Part C: From the first dose of the study drug up to Day 361 | An adverse event (AE) was any unfavorable \& unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with use of an investigational product, whether or not related to investigational product. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of Investigator, placed 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. AE and SAEs were regarded as treatment-emergent if it was present prior to receiving first dose of nusinersen in this current study and subsequently worsened in severity or was not present prior to receiving first dose of nusinersen and subsequently appeared. |
| Primary | Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Parts A and C: Baseline up to Day 302 | Blood chemistry parameters included protein, albumin, creatinine, blood urea nitrogen, bilirubin (total and direct), alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, glucose, calcium, phosphorus, bicarbonate, chloride, sodium, potassium, cystatin C, and 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, by the Investigator. Here, shift to low indicates 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 values postbaseline. The categories with at least one participant with shift from baseline in these parameters are reported. |
| Primary | Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters) | Parts A and C: Baseline up to Day 302 | Hematology parameters included complete blood cell count, with differential and platelet count, and absolute neutrophil count. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available, by the Investigator. Here, shift to low indicates 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 categories with at least one participant with shift from baseline in these parameters are reported. |
| Primary | Parts A and C: Number of Participants With Shifts From Baseline in Urinalysis | Parts A and C: Baseline up to Day 302 | Urinalysis included assessments of urine total protein, specific gravity, pH, protein, glucose, ketones, bilirubin, blood, red blood cells (RBC), white blood cells (WBC), 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, by the Investigator. Here, shift to low indicates 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. The categories with at least one participant with shift from baseline in these parameters are reported. |
| Primary | Parts A and C: Number of Participants With Shifts From Baseline in Cerebrospinal Fluid (CSF) Parameters | Part A: Baseline up to Day 269, Part C: Baseline up to Day 241 | CSF parameters included cell count, total protein, and glucose. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available, by the Investigator. Here, shift to low indicates 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 values postbaseline. The categories with at least one participant with shift from baseline in these parameters are reported. |
| Primary | Parts A and C: Number of Participants With Shifts From Baseline in Electrocardiograms (ECGs) | Parts A and C: Baseline up to Day 302 | The ECGs were assessed by the investigator to be normal, abnormal and abnormal AE. The number of participants with ECG shifts from normal to each of the categorical values denoting an abnormal scan (abnormal not AE, abnormal AE) was assessed. Shift from baseline to worst post-baseline values were reported. The categories with at least one participant with shift from baseline in ECG are reported. |
| Primary | Parts A and C: Number of Participants With Abnormalities in Vital Sign Parameters | Parts A and C: Baseline up to Day 302 | Vital sign assessment included temperature, pulse rate, systolic blood pressure, diastolic blood pressure, and respiratory rate. As pre-specified in protocol, the criteria for determining potentially clinically relevant abnormalities in vital signs included: temperature \< 36.0 and \> 38.0 degrees Celsius (C), pulse rate \< 60 and \> 100 beats per minute (bpm), systolic blood pressure \[\< 90, \> 140 and \> 160 millimeters of mercury (mmHg)\], diastolic blood pressure \< 50, \> 90 and \> 100 mmHg and respiratory rate \< 12 and \> 20 breaths per minute. The categories with at least one participant with clinically relevant vital sign abnormalities are reported. |
| Primary | Parts A and C: Change From Baseline in Growth Parameters (Body Height) | Parts A and C: Baseline, Day 302 | |
| Primary | Part C: Change From Baseline in Growth Parameters (Head Circumference) | Baseline, Day 302 | As pre-specified in the protocol, head circumference was measured for participants with infantile-onset SMA only. |
1 to 10 of 79Source ClinicalTrials.gov
See all 79 registered outcomesOutcomes79
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part B Infantile-onset SMA: Change From Baseline in CHOP-INTEND Total Score for 50/28mg Nusinersen Versus CS3B Matched Sham Control Group | Baseline, Day 183 | As planned, analysis was performed in the Matched sham set. Matched sham set comprised of sham control participants of the CS3B study (NCT02193074) identified by a matching algorithm and all of current study 50/28 mg participants that were included in the ITT set. | score on scale | LEAST_SQUARES_MEAN |
| PRIMARY | Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) | Part A: From the first dose of the study drug up to Day 389, Part C: From the first dose of the study drug up to Day 361 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Parts A and C: Baseline up to Day 302 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. Number analyzed 'n' indicates the number of participants evaluable for analysis of the specified parameter. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters) | Parts A and C: Baseline up to Day 302 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. 'Number analysed (n)' indicates the number of participants evaluable for the specified hematology parameter. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Number of Participants With Shifts From Baseline in Urinalysis | Parts A and C: Baseline up to Day 302 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. 'Number analysed (n)' indicates the number of participants evaluable for the specified urinalysis parameter. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Number of Participants With Shifts From Baseline in Cerebrospinal Fluid (CSF) Parameters | Part A: Baseline up to Day 269, Part C: Baseline up to Day 241 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. 'Number analysed (n)' indicates the number of participants evaluable for the specified urinalysis parameter. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Number of Participants With Shifts From Baseline in Electrocardiograms (ECGs) | Parts A and C: Baseline up to Day 302 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Number of Participants With Abnormalities in Vital Sign Parameters | Parts A and C: Baseline up to Day 302 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Parts A and C: Change From Baseline in Growth Parameters (Body Height) | Parts A and C: Baseline, Day 302 | Part A: Safety set included all participants who received at least one dose of nusinersen. Part C: ITT set included all participants who received at least one dose of nusinersen in the current study. 'Overall number of participants analyzed' signifies number of participants evaluable for this outcome measure at the specified time point. | centimeter (cm) | MEAN |
| PRIMARY | Part C: Change From Baseline in Growth Parameters (Head Circumference) | Baseline, Day 302 | ITT set included all participants who received at least one dose of nusinersen in the current study. 'Overall number of participants analyzed' signifies number of participants evaluable for this outcome measure at the specified time point. | cm | MEAN |
1 to 10 of 79Source ClinicalTrials.gov
See all 79 outcomesResults by group934
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part B Infantile-onset SMA: Change From Baseline in CHOP-INTEND Total Score for 50/28mg Nusinersen Versus CS3B Matched Sham Control Group | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 42.9 | 95% Confidence Interval 38.7 to 47.2 | score on scale | |
| Part B Infantile-onset SMA: Change From Baseline in CHOP-INTEND Total Score for 50/28mg Nusinersen Versus CS3B Matched Sham Control Group | CS3B Matched Sham Control Group | 16.9 | 95% Confidence Interval 10.1 to 23.7 | score on scale | |
| Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) | Part A: Nusinersen 28/28 mg | TEAEs | 4 | Participants | |
| Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) | Part C: Nusinersen 50/28 mg | TEAEs | 37 | Participants | |
| Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) | Part A: Nusinersen 28/28 mg | TESAEs | 1 | Participants | |
| Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) | Part C: Nusinersen 50/28 mg | TESAEs | 6 | Participants | |
| Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Part A: Nusinersen 28/28 mg | Alkaline Phosphatase Shift to High | 1 | Participants | |
| Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Part C: Nusinersen 50/28 mg | Alkaline Phosphatase Shift to High | 2 | Participants | |
| Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Part A: Nusinersen 28/28 mg | Alanine Aminotransferase Shift to High | 0 | Participants | |
| Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Part C: Nusinersen 50/28 mg | Alanine Aminotransferase Shift to High | 6 | Participants |
1 to 10 of 934Source ClinicalTrials.gov
See all 934 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Part A: 28/28 Milligrams (mg) Nusinersen | 6 | |
| Overall Study | STARTED | Part B: Infantile-Onset SMA: 12/12 mg Nusinersen | 25 | |
| Overall Study | STARTED | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 50 | |
| Overall Study | STARTED | Part B: Later-Onset SMA: 12/12 mg Nusinersen | 8 | |
| Overall Study | STARTED | Part B: Later-Onset SMA: 50/28 mg Nusinersen | 16 | |
| Overall Study | STARTED | Part C: 50/28 mg Nusinersen | 40 | |
| Overall Study | COMPLETED | Part A: 28/28 Milligrams (mg) Nusinersen | 6 | |
| Overall Study | COMPLETED | Part B: Infantile-Onset SMA: 12/12 mg Nusinersen | 13 | |
| Overall Study | COMPLETED | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 35 | |
| Overall Study | COMPLETED | Part B: Later-Onset SMA: 12/12 mg Nusinersen | 7 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events1,746
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 | Headache | Nervous system disorders | Part A: 28/28 mg Nusinersen | 2 | 6 | |
| other | Headache | Nervous system disorders | Part B: Infantile-Onset SMA: 12/12 mg Nusinersen | 0 | 25 | |
| other | Headache | Nervous system disorders | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 0 | 50 | |
| other | Headache | Nervous system disorders | Part B: Later-Onset SMA: 12/12 mg Nusinersen | 0 | 8 | |
| other | Headache | Nervous system disorders | Part B: Later-Onset SMA: 50/28 mg Nusinersen | 0 | 16 | |
| other | Headache | Nervous system disorders | Part C: 50/28 mg Nusinersen | 5 | 40 | |
| other | Paraesthesia | Nervous system disorders | Part A: 28/28 mg Nusinersen | 1 | 6 | |
| other | Paraesthesia | Nervous system disorders | Part B: Infantile-Onset SMA: 12/12 mg Nusinersen | 0 | 25 | |
| other | Paraesthesia | Nervous system disorders | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 0 | 50 | |
| other | Paraesthesia | Nervous system disorders | Part B: Later-Onset SMA: 12/12 mg Nusinersen | 0 | 8 |
1 to 10 of 1,746Source ClinicalTrials.gov
See all 1,746 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: 28/28 mg Nusinersen | 0 | 6 |
| Total, all-cause mortality | Part B: Infantile-Onset SMA: 12/12 mg Nusinersen | 6 | 25 |
| Total, all-cause mortality | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 10 | 50 |
| Total, all-cause mortality | Part B: Later-Onset SMA: 12/12 mg Nusinersen | 0 | 8 |
| Total, all-cause mortality | Part B: Later-Onset SMA: 50/28 mg Nusinersen | 0 | 16 |
| Total, all-cause mortality | Part C: 50/28 mg Nusinersen | 0 | 40 |
| Total, other adverse events | Part A: 28/28 mg Nusinersen | 4 | 6 |
| Total, other adverse events | Part B: Infantile-Onset SMA: 12/12 mg Nusinersen | 19 | 25 |
| Total, other adverse events | Part B: Infantile-Onset SMA: 50/28 mg Nusinersen | 37 | 50 |
| Total, other adverse events | Part B: Later-Onset SMA: 12/12 mg Nusinersen | 7 | 8 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
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.
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