Trial · NCT01703988

An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

NCT01703988, Phase 1, Phase 2, Completed, Biogen; results first posted 2017-03-13

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy. Trials, record NCT01703988. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01703988

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
NCT number: NCT01703988
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 34 participants, actual
Lead sponsor: Biogen
Conditions: Spinal Muscular Atrophy
Interventions: Nusinersen
First posted: 2012-10-11
Last updated: 2021-04-13
Other registries' IDs: eudract:2017-000327-27
Results first posted: 2017-03-13

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Participants were followed for the duration of the study; mean (SD) duration of treatment was 82.9 (15.4) days	An AE is any unfavorable and unintended sign, symptom, or disease temporally associated with the study or use of the investigational drug product, whether or not the AE is considered related to the investigational drug product. An SAE is any AE that, in the view of either the Investigator or Sponsor, meets any of the following criteria: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; results in congenital anomaly or birth defect; and is an important medical event in the judgment of the investigator. Drug-related is an event related or possibly related to study drug. Severity of AEs was assessed as mild, moderate, or severe.
Secondary	Plasma Pharmacokinetics: Maximal Observed Plasma Drug Concentration (Cmax)	Day 1 and Day 85
Secondary	Plasma Pharmacokinetics: Time to Reach Cmax in Plasma	Day 1 and Day 85
Secondary	Plasma Pharmacokinetics: Plasma Pharmacokinetics: Area Under the Plasma Concentration Time Curve From the Time of the IT Dose to 6 Hours After Dosing (AUC0-6hr)	Day 1 and Day 85
Secondary	Cerebrospinal Fluid (CSF) Pharmacokinetics: Predose CSF Drug Concentrations	Day 1, Day 29, and Day 85
Secondary	Urine Pharmacokinetics: Renal Clearance, Cohort 4	Day 1 and Day 85	Renal clearance of nusinersen for participants was assessed in the 12 mg reporting group only, per protocol.

Source ClinicalTrials.gov

Outcomes6

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 Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Participants were followed for the duration of the study; mean (SD) duration of treatment was 82.9 (15.4) days	Safety Population, defined as all enrolled participants who received at least 1 dose of study drug.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Plasma Pharmacokinetics: Maximal Observed Plasma Drug Concentration (Cmax)	Day 1 and Day 85	Pharmacokinetic (PK) Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point.	ng/mL	MEAN
SECONDARY	Plasma Pharmacokinetics: Time to Reach Cmax in Plasma	Day 1 and Day 85	PK Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point.	hours	MEDIAN
SECONDARY	Plasma Pharmacokinetics: Plasma Pharmacokinetics: Area Under the Plasma Concentration Time Curve From the Time of the IT Dose to 6 Hours After Dosing (AUC0-6hr)	Day 1 and Day 85	PK Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point.	ng*hr/mL	MEAN
SECONDARY	Cerebrospinal Fluid (CSF) Pharmacokinetics: Predose CSF Drug Concentrations	Day 1, Day 29, and Day 85	PK Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point. (This study includes participants previously enrolled in Study ISIS 396443 - CS1 (NCT01494701).	ng/mL	MEAN
SECONDARY	Urine Pharmacokinetics: Renal Clearance, Cohort 4	Day 1 and Day 85	Pharmacokinetic (PK) Population, defined as all enrolled participants who have evaluable PK data	mL/hr	MEAN

Source ClinicalTrials.gov

Results by group65

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 3 mg	Any AE	8	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 6 mg	Any AE	6	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 9 mg	Any AE	9	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 12 mg	Any AE	9	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 3 mg	Any drug-related AE	0	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 6 mg	Any drug-related AE	0	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 9 mg	Any drug-related AE	0	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 12 mg	Any drug-related AE	0	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 3 mg	Any SAE	2	Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs	Nusinersen 6 mg	Any SAE	0	Participants

1 to 10 of 65Source ClinicalTrials.gov

See all 65 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Nusinersen 3 mg	8
Overall Study	STARTED	Nusinersen 6 mg	8
Overall Study	STARTED	Nusinersen 9 mg	9
Overall Study	STARTED	Nusinersen 12 mg	9
Overall Study	COMPLETED	Nusinersen 3 mg	8
Overall Study	COMPLETED	Nusinersen 6 mg	8
Overall Study	COMPLETED	Nusinersen 9 mg	9
Overall Study	COMPLETED	Nusinersen 12 mg	8
Overall Study	NOT COMPLETED	Nusinersen 3 mg	0
Overall Study	NOT COMPLETED	Nusinersen 6 mg	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events368

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
other	Palpitations	Cardiac disorders	Nusinersen 3 mg	1	8
other	Palpitations	Cardiac disorders	Nusinersen 6 mg	0	8
other	Palpitations	Cardiac disorders	Nusinersen 9 mg	0	9
other	Palpitations	Cardiac disorders	Nusinersen 12 mg	0	9
other	Ear pain	Ear and labyrinth disorders	Nusinersen 3 mg	1	8
other	Ear pain	Ear and labyrinth disorders	Nusinersen 6 mg	0	8
other	Ear pain	Ear and labyrinth disorders	Nusinersen 9 mg	0	9
other	Ear pain	Ear and labyrinth disorders	Nusinersen 12 mg	0	9
other	Conjunctivitis	Eye disorders	Nusinersen 3 mg	0	8
other	Conjunctivitis	Eye disorders	Nusinersen 6 mg	0	8

1 to 10 of 368Source ClinicalTrials.gov

See all 368 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Nusinersen 3 mg
Total, all-cause mortality	Nusinersen 6 mg
Total, all-cause mortality	Nusinersen 9 mg
Total, all-cause mortality	Nusinersen 12 mg
Total, other adverse events	Nusinersen 3 mg	8	8
Total, other adverse events	Nusinersen 6 mg	6	8
Total, other adverse events	Nusinersen 9 mg	9	9
Total, other adverse events	Nusinersen 12 mg	9	9
Total, serious adverse events	Nusinersen 3 mg	2	8
Total, serious adverse events	Nusinersen 6 mg	0	8

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
31420846	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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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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