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Compound · clinical trials

Nusinersen clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — the 3 trials in our USA and UK registers that name Nusinersen as an intervention. Synced 25 Sep 2026
3Current trials
2Recruiting now
2Lead sponsors
2Country registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 โ€” Top 14 finalist
Compiled from ClinicalTrials.gov registry records ยท Last verified ยท Editorial standards
At a glance

Nusinersen is named as an intervention in 3 current clinical trials in our USA and UK registers. 2 are recruiting patients now. The most common phase is Phase 2. The sponsor with the most trials is Biogen (2). The most-studied condition is Spinal Muscular Atrophy.

Nusinersen also has an approved-drug profile on this site (FDA label data): Spinraza (Nusinersen) →

Trials by status

  • Recruiting267%
  • Active, closed133%

Trials by phase

  • Phase 2133%
  • Phase 1133%
  • Phase 3133%
01

Clinical trials of Nusinersen

3 trials
StudyPhaseStatusLead sponsorRegisters
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
NCT07047144 · ClinicalTrials.gov ↗ · Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2
Phase 2 Recruiting Scholar Rock, Inc. UK, USA
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRxโ„ข System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
NCT06555419 · ClinicalTrials.gov ↗ · Muscular Atrophy, Spinal
Phase 1 Recruiting Biogen UK, USA
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
NCT05067790 · ClinicalTrials.gov ↗ · Spinal Muscular Atrophy
Phase 3 Active, closed Biogen USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.

02

Nusinersen across our data

Ireland · EU · US

The same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.

Licensed in Ireland (HPRA)

3 single-substance products containing Nusinersen on the HPRA medicines list.

All 3 Irish products →

Supply shortages

No entry for Nusinersen in the EMA, FDA or German BfArM shortage lists we track.

03

Conditions studied with Nusinersen

5 conditions
Spinal Muscular Atrophy2SMA1Spinal Muscular Atrophy Type 21Spinal Muscular Atrophy Type 31Muscular Atrophy, Spinal1
04

Sponsors

lead sponsor per trial
Lead sponsorTrials
Biogen2
Scholar Rock, Inc.1
05

By country register

RegisterTrialsRecruiting
Clinical trials in USA32
Clinical trials in UK22

A trial with sites in more than one country appears in each of those registers.

06

Related compounds

07

Questions about Nusinersen trials

How many clinical trials are testing Nusinersen?

3 current trials in our USA and UK registers name Nusinersen as a drug or biological intervention, as of the registry sync on 25 September 2026. Completed and withdrawn studies are not counted.

Are Nusinersen trials recruiting patients?

Yes. 2 of the 3 trials are recruiting: 2 with sites in USA and 2 with sites in UK. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Who sponsors Nusinersen clinical trials?

Biogen (2) and Scholar Rock, Inc (1). The number in brackets is how many trials each organisation leads.

Is Nusinersen licensed in Ireland?

Yes. The HPRA medicines list includes 3 single-substance products containing Nusinersen, for example Spinraza 12 mg solution for injection (Biogen Netherlands B.V.). A licence does not mean a product is currently marketed or reimbursed; see each product's record.

Is there a Nusinersen shortage?

Not in the lists we track: Nusinersen has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.

Where does this information come from?

Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Nusinersen works or whether it is effective, and it is not medical advice.

How this page is built

Everything on this page is copied from public ClinicalTrials.gov registry records โ€” it is not written from memory, and nothing here describes how Nusinersen works or whether it is effective. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Nusinersen (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 25 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician โ€” our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us โ€” we correct promptly and log it.