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New drug tofersen under watch for ALS patients in china

NCT ID NCT07223723

Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 2 times

Summary

This study checks the long-term safety of tofersen (Qalsody) in 12 Chinese adults with a specific genetic form of ALS (SOD1-ALS). Participants receive 13 doses of the drug injected into the spine over about a year. The main goal is to track any side effects and how the drug moves through the body.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

May 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Age ≥ 18 years at the time of informed consent. * Must have diagnosis of SOD1-ALS. * If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit. * If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. * All women of childbearing potential must practice effective contraception during the study. Key Exclusion Criteria: * Hypersensitivity to the active substance or any of the excipients of tofersen injection. * Current or past administration of tofersen injection, in either a commercial or a clinical study setting. * Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy. * Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, prior the Baseline Visit. * Participants who are pregnant or currently breastfeeding, and those intending to become pregnant during the study. Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis amyotrophic lateral sclerosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Amyotrophic lateral sclerosis

Contacts and locations

Locations

  • Peking University Third Hospital

    Beijing, Beijing Municipality, 100083, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.