Clinical Trials

Numerous clinical trials evaluate nusinersen across Early Phase 1 through Phase 4 post-marketing studies, focusing primarily on spinal muscular atrophy variants (infantile-onset, types II and 3, and adult), related neuromuscular manifestations, and ultrasound-assisted administration. Sponsored by pharmaceutical companies such as Biogen and academic medical centers, these trials encompass various recruitment statuses, including recruiting, active not recruiting, completed, terminated, and withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07047144 RECRUITING
Spinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type 3; Neuromuscular Manifestations; Anti-myostatin
Scholar Rock, Inc.
 2025-09-15 PHASE2
NCT05866419 RECRUITING
Spinal Muscular Atrophy; Spine Deformity; Scoliosis
Alcyone Therapeutics, Inc
 2023-11-27
NCT06555419 RECRUITING
Muscular Atrophy, Spinal
Biogen
 2025-01-16 PHASE1
NCT05067790 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy
Biogen
 2022-01-21 PHASE3
NCT04729907 COMPLETED
Muscular Atrophy, Spinal
Biogen
 2021-04-19 PHASE3
NCT05522361 UNKNOWN
Spinal Muscular Atrophy
Clinic for Special Children
 2022-11-06 PHASE4
NCT04488133 COMPLETED
Muscular Atrophy, Spinal
Biogen
 2021-01-04 PHASE4
NCT05156320 COMPLETED
Spinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA; Neuromuscular Diseases; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Manifestations; Anti-myostatin
Scholar Rock, Inc.
 2022-04-14 PHASE3
NCT02386553 COMPLETED
Spinal Muscular Atrophy
Biogen
 2015-05-18 PHASE2
NCT05386680 COMPLETED
Spinal Muscular Atrophy
Novartis Pharmaceuticals
 2023-01-12 PHASE3
NCT04089566 COMPLETED
Muscular Atrophy, Spinal
Biogen
 2020-03-26 PHASE3
NCT02594124 COMPLETED
Spinal Muscular Atrophy
Biogen
 2015-11-04 PHASE3
NCT04576494 COMPLETED
Spinal Muscular Atrophy
CHU de Reims
 2022-01-24
NCT04674618 COMPLETED
Muscular Atrophy, Spinal; Ultrasound
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
 2020-12-14
NCT04050852 WITHDRAWN
Spinal Muscular Atrophy
NYU Langone Health
 2019-07-17 EARLY_PHASE1
NCT02462759 TERMINATED
Spinal Muscular Atrophy
Biogen
 2015-08-19 PHASE2
NCT01839656 COMPLETED
Spinal Muscular Atrophy
Biogen
 2013-05-08 PHASE2
NCT02292537 COMPLETED
Spinal Muscular Atrophy
Biogen
 2014-11-24 PHASE3
NCT02052791 COMPLETED
Spinal Muscular Atrophy
Biogen
 2014-01-31 PHASE1
NCT02193074 TERMINATED
Spinal Muscular Atrophy
Biogen
 2014-08-19 PHASE3
NCT01703988 COMPLETED
Spinal Muscular Atrophy
Biogen
 2012-10-31 PHASE1; PHASE2
NCT01780246 COMPLETED
Spinal Muscular Atrophy
Biogen
 2013-01-31 PHASE1
NCT01494701 COMPLETED
Spinal Muscular Atrophy
Biogen
 2011-11-30 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-05-01)

Check the Nusinersen product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Nusinersen is an antisense oligonucleotide that specifically binds to the intronic splice silencer N1 (ISS-N1) site in intron 7 of SMN2 pre-mRNA, blocking the interaction of splicing repressors hnRNP A1/A2 and promoting exon 7 inclusion. This targeted splicing modification restores the production of functional, full-length survival motor neuron protein, thereby mitigating motor neuron degeneration in patients with spinal muscular atrophy.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.