Trial · NCT02462759

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

NCT02462759, Phase 2, Terminated, Biogen; results first posted 2020-01-27

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). Trials, record NCT02462759. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02462759

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Trial

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

Title: A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
NCT number: NCT02462759
Status: Terminated
Phases: Phase 2
Enrollment: 21 participants, actual
Lead sponsor: Biogen
Conditions: Spinal Muscular Atrophy
Interventions: Nusinersen, Sham Procedure
First posted: 2015-06-04
Last updated: 2021-02-17
Other registries' IDs: eudract:2014-003657-33
Results first posted: 2020-01-27
Limitations and caveats: Despite the early termination of both Parts of the study, the data from this study is of quality and reliable.

Source ClinicalTrials.gov

Registered outcomes21

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) and Serious Adverse Events (SAEs)	Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)	An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A SAE is any untoward medical occurrence that at any dose results in death, life-threatening event, requires inpatient hospitalization, significant disability/incapacity or congenital anomaly.
Primary	Number of Participants With Change From Baseline in Clinical Laboratory Parameters	Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)	Clinically significant changes in laboratory parameters were evaluated for assessing the safety of ISIS 396443.
Primary	Number of Participants With Change From Baseline in Electrocardiograms (ECGs)	Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596	Clinically significant changes in ECG measurements were evaluated for assessing the safety of ISIS 396443.
Primary	Number of Participants With Change From Baseline in Vital Signs	Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596	Clinically significant changes in vital signs were evaluated for assessing the safety of ISIS 396443. Vital signs that were assessed included resting systolic and diastolic blood pressure, pulse rate, respiratory rate, temperature, pulse oximetry, and transcutaneous carbon dioxide.
Primary	Change From Baseline in Head Circumference	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Participants were analyzed for change in growth parameter of head circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the head circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.
Primary	Change From Baseline in Chest Circumference	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Participants were analyzed for change in growth parameter of chest circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the chest circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days\>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.
Primary	Change From Baseline in Arm Circumference	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Participants were analyzed for change in growth parameter of arm circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the arm circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.
Primary	Change From Baseline in Weight for Age	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Participants were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the weight for age percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.
Primary	Change From Baseline in Weight	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Participants were analyzed for change in growth parameter of weight to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the weight percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.
Primary	Change From Baseline in Head to Chest Circumference (HCC) Ratio	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Participants were analyzed for change in growth parameter of HCC to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the HCC circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

1 to 10 of 21Source ClinicalTrials.gov

See all 21 registered outcomes

Outcomes21

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) and Serious Adverse Events (SAEs)	Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)	The safety population included all participants who were randomized and received at least 1 dose of study treatment or sham procedure.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Change From Baseline in Clinical Laboratory Parameters	Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)	The safety population included all participants who were randomized and received at least 1 dose of study treatment or sham procedure.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Change From Baseline in Electrocardiograms (ECGs)	Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596	The safety population included all participants who were randomized and received at least 1 dose of study treatment or sham procedure.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Change From Baseline in Vital Signs	Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596	The safety population included all participants who were randomized and received at least 1 dose of study treatment or sham procedure.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Change From Baseline in Head Circumference	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Safety population: participants who were randomized and received at least 1 dose of study treatment or sham procedure. Number analyzed indicates participants who were evaluated for the specified time points. Due the early termination of Part 2, no participants in the ISIS 396443 Part 2(participants on sham in Part 1) were on study beyond Day 659.	centimeter (cm)	MEAN
PRIMARY	Change From Baseline in Chest Circumference	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Safety population: participants who were randomized and received at least 1 dose of study treatment or sham procedure. Number analyzed indicates participants who were evaluated for the specified time points. Due the early termination of Part 2, no participants in the ISIS 396443 Part 2(participants on sham in Part 1) were on study beyond Day 659.	cm	MEAN
PRIMARY	Change From Baseline in Arm Circumference	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Safety population: participants who were randomized and received at least 1 dose of study treatment or sham procedure. Number analyzed indicates participants who were evaluated for the specified time points. Due the early termination of Part 2, no participants in the ISIS 396443 Part 2(participants on sham in Part 1) were on study beyond Day 659.	cm	MEAN
PRIMARY	Change From Baseline in Weight for Age	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Safety population: participants who were randomized and received at least 1 dose of study treatment or sham procedure. Number analyzed indicates participants who were evaluated for the specified time points. Due the early termination of Part 2, no participants in the ISIS 396443 Part 2(participants on sham in Part 1) were on study beyond Day 659.	kilogram (kg)	MEAN
PRIMARY	Change From Baseline in Weight	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Safety population: participants who were randomized and received at least 1 dose of study treatment or sham procedure. Number analyzed indicates participants who were evaluated for the specified time points. Due the early termination of Part 2, no participants in the ISIS 396443 Part 2(participants on sham in Part 1) were on study beyond Day 659.	kg	MEAN
PRIMARY	Change From Baseline in Head to Chest Circumference (HCC) Ratio	Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138	Safety population: participants who were randomized and received at least 1 dose of study treatment or sham procedure. Number analyzed indicates participants who were evaluated for the specified time points. Due the early termination of Part 2, no participants in the ISIS 396443 Part 2(participants on sham in Part 1) were on study beyond Day 659.	ratio	MEAN

1 to 10 of 21Source ClinicalTrials.gov

See all 21 outcomes

Results by group251

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) and Serious Adverse Events (SAEs)	Sham Procedure in Part 1	AEs	6	Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	ISIS 396443 Part 2(Participants on Sham in Part 1)	AEs	6	Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	ISIS 396443 Part 1 & 2	AEs	14	Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	Sham Procedure in Part 1	SAEs	3	Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	ISIS 396443 Part 2(Participants on Sham in Part 1)	SAEs	4	Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	ISIS 396443 Part 1 & 2	SAEs	9	Participants
Number of Participants With Change From Baseline in Clinical Laboratory Parameters	Sham Procedure in Part 1		0	Participants
Number of Participants With Change From Baseline in Clinical Laboratory Parameters	ISIS 396443 Part 2(Participants on Sham in Part 1)		0	Participants
Number of Participants With Change From Baseline in Clinical Laboratory Parameters	ISIS 396443 Part 1 & 2		0	Participants
Number of Participants With Change From Baseline in Electrocardiograms (ECGs)	Sham Procedure in Part 1		0	Participants

1 to 10 of 251Source ClinicalTrials.gov

See all 251 results by group

Participant flow18

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

Period	Milestone	Group	Participants
Part 1: Double Blind	STARTED	Sham Procedure (Part 1)	7
Part 1: Double Blind	STARTED	ISIS 396443 (Part 1)	14
Part 1: Double Blind	STARTED	ISIS 396443 (Part 2)	0
Part 1: Double Blind	COMPLETED	Sham Procedure (Part 1)	6
Part 1: Double Blind	COMPLETED	ISIS 396443 (Part 1)	14
Part 1: Double Blind	COMPLETED	ISIS 396443 (Part 2)	0
Part 1: Double Blind	NOT COMPLETED	Sham Procedure (Part 1)	1
Part 1: Double Blind	NOT COMPLETED	ISIS 396443 (Part 1)	0
Part 1: Double Blind	NOT COMPLETED	ISIS 396443 (Part 2)	0
Part 2: Open-Label Phase	STARTED	Sham Procedure (Part 1)	0

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events675

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	Anaemia	Blood and lymphatic system disorders	Sham Procedure in Part 1	0	7
other	Anaemia	Blood and lymphatic system disorders	ISIS 396443 Part 1 & 2	2	14
other	Anaemia	Blood and lymphatic system disorders	ISIS 396443 Part 2(Participants on Sham in Part 1)	0	6
other	Leukocytosis	Blood and lymphatic system disorders	Sham Procedure in Part 1	0	7
other	Leukocytosis	Blood and lymphatic system disorders	ISIS 396443 Part 1 & 2	2	14
other	Leukocytosis	Blood and lymphatic system disorders	ISIS 396443 Part 2(Participants on Sham in Part 1)	0	6
other	Thrombocytosis	Blood and lymphatic system disorders	Sham Procedure in Part 1	0	7
other	Thrombocytosis	Blood and lymphatic system disorders	ISIS 396443 Part 1 & 2	1	14
other	Thrombocytosis	Blood and lymphatic system disorders	ISIS 396443 Part 2(Participants on Sham in Part 1)	0	6
other	Bradycardia	Cardiac disorders	Sham Procedure in Part 1	0	7

1 to 10 of 675Source ClinicalTrials.gov

See all 675 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sham Procedure in Part 1	1	7
Total, all-cause mortality	ISIS 396443 Part 1 & 2	0	14
Total, all-cause mortality	ISIS 396443 Part 2(Participants on Sham in Part 1)	0	6
Total, other adverse events	Sham Procedure in Part 1	6	7
Total, other adverse events	ISIS 396443 Part 1 & 2	14	14
Total, other adverse events	ISIS 396443 Part 2(Participants on Sham in Part 1)	6	6
Total, serious adverse events	Sham Procedure in Part 1	3	7
Total, serious adverse events	ISIS 396443 Part 1 & 2	9	14
Total, serious adverse events	ISIS 396443 Part 2(Participants on Sham in Part 1)	4	6

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33501671	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 comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
ISIS 396443 Part 1 & 2 vs ISIS 396443 Part 2(Participants on Sham in Part 1): Number of Participants With Plasma Antibodies to ISIS 396443

Source Trial comparisons

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.

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Read this trial from software.

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