NCT04089566 (DEVOTE) is a Phase 3 clinical trial of Nusinersen in Muscular Atrophy, Spinal, sponsored by Biogen.

Who is sponsoring NCT04089566?

NCT04089566 is sponsored by Biogen.

What drugs are being tested in NCT04089566?

Interventions in NCT04089566: Nusinersen.

What condition does NCT04089566 study?

NCT04089566 studies Muscular Atrophy, Spinal.

Is NCT04089566 still recruiting?

NCT04089566 is currently completed. Last updated 5 June 2025.

Are results published for NCT04089566?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2025-06-05 · How we verify

NCT04089566: DEVOTE

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Completed Phase 3 Results posted Last updated 5 June 2025

What this trial tests

Phase 3 trial testing Nusinersen in Muscular Atrophy, Spinal in 145 participants. Completed in 30 May 2024.

Timeline

26 March 2020

Primary endpoint
21 February 2024

30 May 2024

Quick facts

Lead sponsor	Biogen
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	double
Primary purpose	treatment
Enrollment	145
Start date	26 March 2020
Primary completion	21 February 2024
Estimated completion	30 May 2024
Sites	45 locations across Italy, Colombia, Japan, Russia, Chile, Saudi Arabia, Estonia, Taiwan

Drugs / interventions tested

Nusinersen (NUSINERSEN) — full drug profile →

Conditions studied

Muscular Atrophy, Spinal — all drugs for Muscular Atrophy, Spinal →

Sponsor

Biogen — full company profile →

Who can join

7 Days and older, any sex, with Muscular Atrophy, Spinal. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Part B Infantile-onset SMA: Change From Baseline in CHOP-INTEND Total Score for 50/28mg Nusinersen Versus CS3B Matched Sham Control Group Primary · 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

Group	Value	95% CI
Part B: Infantile-Onset SMA: 50/28 mg Nusinersen	42.9	38.7 – 47.2
CS3B Matched Sham Control Group	16.9	10.1 – 23.7

Parts A and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) Primary · 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 SA

TEAEs

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	4
Part C: Nusinersen 50/28 mg	37

TESAEs

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	6

Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) Primary · 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

Alkaline Phosphatase Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	2

Alanine Aminotransferase Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	6

Aspartate Aminotransferase Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	8

Bicarbonate Shift to Low

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	6

Bicarbonate Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	2

Bilirubin Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	1

Indirect Bilirubin Shift to Low

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	4
Part C: Nusinersen 50/28 mg	12

Indirect Bilirubin Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	1

Parts A and C: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters) Primary · 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 pa

Basophils Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	2
Part C: Nusinersen 50/28 mg	6

Basophils/Leukocytes Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	2
Part C: Nusinersen 50/28 mg	6

Eosinophils Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	3

Eosinophils/Leukocytes Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	3
Part C: Nusinersen 50/28 mg	9

Hematocrit Shift to Low

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	2

Hematocrit Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	0

Hemoglobin Shift to Low

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	3

Lymphocytes Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	1

Parts A and C: Number of Participants With Shifts From Baseline in Urinalysis Primary · 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 postbasel

Specific Gravity Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	2
Part C: Nusinersen 50/28 mg	1

pH Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	2

Protein High/positive

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	4
Part C: Nusinersen 50/28 mg	9

Glucose High/positive

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	2

Ketones High/positive

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	8

Occult Blood High/positive

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	2
Part C: Nusinersen 50/28 mg	4

RBC High/positive

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	1

WBC High/positive

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	2

Parts A and C: Number of Participants With Shifts From Baseline in Cerebrospinal Fluid (CSF) Parameters Primary · 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.

Glucose Shift to Low

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	2

Glucose Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	2

Protein Shift to Low

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	3

Protein Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	8

Erythrocytes Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	2
Part C: Nusinersen 50/28 mg	8

Leukocytes Shift to High

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	6

Parts A and C: Number of Participants With Shifts From Baseline in Electrocardiograms (ECGs) Primary · 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.

Baseline: Normal; Postbaseline: Normal

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	10

Baseline: Normal; Post-baseline: Abnormal, not AE

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	3
Part C: Nusinersen 50/28 mg	12

Baseline: Abnormal, not AE; Post-baseline: Abnormal, not AE

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	3
Part C: Nusinersen 50/28 mg	17

Baseline: Unknown; Post-baseline: Abnormal, not AE

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	1

Parts A and C: Number of Participants With Abnormalities in Vital Sign Parameters Primary · 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 particip

Temperature <36.0 C

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	4
Part C: Nusinersen 50/28 mg	13

Pulse rate <60 bpm

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	14

Pulse rate >100 bpm

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	6
Part C: Nusinersen 50/28 mg	19

Systolic blood pressure <90 mmHg

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	4
Part C: Nusinersen 50/28 mg	13

Systolic blood pressure >140 mmHg

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	3

Diastolic blood pressure <50 mmHg

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	7

Diastolic blood pressure >90 mmHg

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	0
Part C: Nusinersen 50/28 mg	1

Respiratory rate <12 breaths/min

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	1
Part C: Nusinersen 50/28 mg	7

Parts A and C: Change From Baseline in Growth Parameters (Body Height) Primary · Parts A and C: Baseline, Day 302

Group	Value	95% CI
Part A: Nusinersen 28/28 mg	7.2	± 1.70
Part C: 50/28 mg Nusinersen	0.8	± 3.12

Part C: Change From Baseline in Growth Parameters (Head Circumference) Primary · Baseline, Day 302

As pre-specified in the protocol, head circumference was measured for participants with infantile-onset SMA only.

Group	Value	95% CI
Part C: Nusinersen 50/28 mg	2.0	± NA

Part C: Change From Baseline in Growth Parameters (Chest Circumference) Primary · Baseline, Day 302

As pre-specified in protocol, chest circumference was measured for participants with infantile-onset SMA only.

Group	Value	95% CI
Part C: Nusinersen 50/28 mg	2.5	± NA

Part C: Change From Baseline in Growth Parameters (Arm Circumference) Primary · Baseline, Day 302

As pre-specified in the protocol, arm circumference was measured for participants with infantile-onset SMA. Here, negative change from baseline indicated reduction in arm circumference.

Group	Value	95% CI
Part C: Nusinersen 50/28 mg	-1.0	± NA

Adverse events — posted to ClinicalTrials.gov

Time frame: Part A: From first dose of the study up to Day 389 Part B: From first dose of the study up to Day 399 Part C: From first dose of the study up to Day 361. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Part A: 28/28 mg Nusinersen

Serious: 1/6 (17%)

Deaths: 0/6

Part B: Infantile-Onset SMA: 12/12 mg Nusinersen

Serious: 18/25 (72%)

Deaths: 6/25

Part B: Infantile-Onset SMA: 50/28 mg Nusinersen

Serious: 30/50 (60%)

Deaths: 10/50

Part B: Later-Onset SMA: 12/12 mg Nusinersen

Serious: 4/8 (50%)

Deaths: 0/8

Part B: Later-Onset SMA: 50/28 mg Nusinersen

Serious: 2/16 (13%)

Deaths: 0/16

Part C: 50/28 mg Nusinersen

Serious: 6/40 (15%)

Deaths: 0/40

Serious adverse events (69 terms)

Reaction	System	Part A: 28/28 mg Nusinersen	Part B: Infantile-Onset SM…	Part B: Infantile-Onset SM…	Part B: Later-Onset SMA: 1…	Part B: Later-Onset SMA: 5…	Part C: 50/28 mg Nusinersen
Respiratory failure	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—	—	—
Pneumonia aspiration	Infections and infestations	—	—	—	—	—	—
Cardiac arrest	Cardiac disorders	—	—	—	—	—	—
Bronchiolitis	Infections and infestations	—	—	—	—	—	—
Covid-19	Infections and infestations	—	—	—	—	—	—
Acute respiratory failure	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—	—	—
Femur fracture	Injury, poisoning and procedural complications	—	—	—	—	—	—
Sudden infant death syndrome	General disorders	—	—	—	—	—	—
Cardio-respiratory arrest	Cardiac disorders	—	—	—	—	—	—
Pneumonia viral	Infections and infestations	—	—	—	—	—	—
Respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Oxygen saturation decreased	Investigations	—	—	—	—	—	—
Atelectasis	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Tracheostomy malfunction	Injury, poisoning and procedural complications	—	—	—	—	—	—
Traumatic haemothorax	Injury, poisoning and procedural complications	—	—	—	—	—	—
Gait disturbance	General disorders	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—
Organ failure	General disorders	—	—	—	—	—	—
Abortion spontaneous	Pregnancy, puerperium and perinatal conditions	—	—	—	—	—	—
Leukocytosis	Blood and lymphatic system disorders	—	—	—	—	—	—
Cardiomyopathy	Cardiac disorders	—	—	—	—	—	—
Dysphagia	Gastrointestinal disorders	—	—	—	—	—	—
Adenoviral upper respiratory infection	Infections and infestations	—	—	—	—	—	—

Other adverse events (222 terms — click to expand)

Reaction	System	Part A: 28/28 mg Nusinersen	Part B: Infantile-Onset SM…	Part B: Infantile-Onset SM…	Part B: Later-Onset SMA: 1…	Part B: Later-Onset SMA: 5…	Part C: 50/28 mg Nusinersen
Procedural headache	Injury, poisoning and procedural complications	—	—	—	—	—	—
COVID-19	Infections and infestations	—	—	—	—	—	—
Asthenia	General disorders	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Procedural pain	Injury, poisoning and procedural complications	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—
Dysphagia	Gastrointestinal disorders	—	—	—	—	—	—
Malnutrition	Metabolism and nutrition disorders	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
Nasopharyngitis	Infections and infestations	—	—	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—	—	—
Bronchitis	Infections and infestations	—	—	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—	—	—
Procedural vomiting	Injury, poisoning and procedural complications	—	—	—	—	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Muscle contracture	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Dermatitis diaper	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Myocardial necrosis marker increased	Investigations	—	—	—	—	—	—
Respiratory failure	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—
Gastrooesophageal reflux disease	Gastrointestinal disorders	—	—	—	—	—	—
Tonsillitis	Infections and infestations	—	—	—	—	—	—
Gastroenteritis	Infections and infestations	—	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—	—
Influenza	Infections and infestations	—	—	—	—	—	—
Otitis media	Infections and infestations	—	—	—	—	—	—
Bronchiolitis	Infections and infestations	—	—	—	—	—	—
Pneumonia klebsiella	Infections and infestations	—	—	—	—	—	—
Stoma site infection	Infections and infestations	—	—	—	—	—	—
Viral infection	Infections and infestations	—	—	—	—	—	—
Viral upper respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Klebsiella infection	Infections and infestations	—	—	—	—	—	—

Most-reported serious reactions: Respiratory failure, Pneumonia, Pneumonia aspiration, Cardiac arrest, Bronchiolitis, Covid-19, Acute respiratory failure, Fall.

Data from ClinicalTrials.gov NCT04089566 adverse events section.

Sponsor's own description

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C). The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Gene-based therapies for neurodegenerative diseases.
Sun J, Roy S. · · 2021 · cited 126× · PMID 33526943 · DOI 10.1038/s41593-020-00778-1
Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine.
Chaytow H, Faller KME, Huang YT, Gillingwater TH. · · 2021 · cited 103× · PMID 34337562 · DOI 10.1016/j.xcrm.2021.100346
Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8
Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy.
Aslesh T, Yokota T, Yokota T. · · 2022 · cited 46× · PMID 35159227 · DOI 10.3390/cells11030417
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders.
Jablonka S, Hennlein L, Sendtner M. · · 2022 · cited 37× · PMID 34983696 · DOI 10.1186/s42466-021-00162-9
New therapies for spinal muscular atrophy: where we stand and what is next.
Antonaci L, Pera MC, Mercuri E. · · 2023 · cited 30× · PMID 37067602 · DOI 10.1007/s00431-023-04883-8
Scientific rationale for a higher dose of nusinersen.
Finkel RS, Ryan MM, Pascual Pascual SI, Day JW, et al · · 2022 · cited 26× · PMID 35567345 · DOI 10.1002/acn3.51562
Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape.
Ponomarev AS, Chulpanova DS, Yanygina LM, Solovyeva VV, et al · · 2023 · cited 22× · PMID 37762045 · DOI 10.3390/ijms241813743

Verify or expand the search:

Other trials of Nusinersen

Trials testing the same drug.

NCT06555419 — A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pe · Phase 1 · recruiting
NCT04576494 — Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) · NA · completed
NCT05067790 — A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spina · Phase 3 · active not recruiting
NCT05187260 — Antisense Oligonucleotide for Spinal Muscular Atrophy · unknown
NCT04729907 — A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants · Phase 3 · active not recruiting

Other recruiting trials for Muscular Atrophy, Spinal

Currently open trials in the same condition.

NCT07221669 — A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Ba · Phase 3 · recruiting
NCT06555419 — A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pe · Phase 1 · recruiting
NCT05861999 — A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atr · Phase 4 · recruiting
NCT05861986 — A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Particip · Phase 4 · recruiting
NCT05808764 — A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy · Phase 2 · recruiting

Other Biogen trials

Trials by the same sponsor.

NCT07483632 — A Study to Learn About the Safety of Diroximel Fumarate (DRF) and Dimethyl Fumarate (DMF) and Their Effects on Relapses · Phase 3 · not yet recruiting
NCT06628687 — A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it Duri · recruiting
NCT07444450 — A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy · Phase 3 · not yet recruiting
NCT07444489 — A Study to Learn More About the Long-Term Safety and Effects of Felzartamab Infusions in Adults With Kidney Transplants · Phase 3 · not yet recruiting
NCT07444476 — A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years Wit · Phase 3 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04089566 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Biogen
Last refreshed: 5 June 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04089566.

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