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Can a nationwide registry unlock the secrets of adult spinal muscular atrophy?

NCT ID NCT05618379

Knowledge-focused 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 04, 2026 · Last updated Aug 05, 2026 · Updated 1 time

Summary

This study is a nationwide registry in China that will follow adults with a genetic form of spinal muscular atrophy (SMA). The goal is to describe how the disease naturally progresses over time and how disease-modifying treatments are used in real-world settings. By collecting data from 200 participants, researchers hope to gain a clearer picture of the condition in adults, which may help improve future care and treatment strategies.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
This registry could help doctors better understand how spinal muscular atrophy progresses in adults and how treatments are used in real life, potentially leading to improved care and support strategies.
What could go wrong
As an observational registry, it does not test a new treatment, so it won't directly lead to a cure. The findings may also be specific to the Chinese population and may not apply to other regions.

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.

Participants

About 200 people

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

Started

Jan 2023

Expected to finish

Jun 2028

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.

Who is studied

Adult participants with 5q-SMA will be enrolled to obtain information on both the natural history of the disease and effectiveness and safety of DMTs.

Ages

18 years and older

Sex

Anyone

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: -Genetically confirmed 5q-SMA. Key Exclusion Criteria: -Other types of SMA (non 5q-SMA). NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

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

Muscular atrophy, spinal

Contacts and locations

Locations

  • Huashan Hospital, Fudan University

    Shanghai, Shanghai Municipality, 200040, China

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100730, China

  • Peking University Shenzhen Hospital

    Shenzhen, Guangdong, 518000, China

  • Qilu Hospital of Shandong University

    Jinan, Shandong, 250012, China

  • The First Affiliated Hospital of Fujian Medical University

    Fuzhou, Fujian, 350005, China

  • The First Affiliated Hospital of Nanchang University

    Nanchang, Jiangxi, 330006, China

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu, 215006, China

  • The First Affiliated Hospital, Sun Yat-sen University

    Guangzhou, Guangdong, 510080, China

  • The First People's Hospital of Yunnan Province

    Kunming, Yun'nan, 650032, China

  • The Second Hospital of Hebei Medical University

    Shijiazhuang, Hebei, 050000, China

  • West China Hospital, Sichuan University

    Chengdu, Sichuan, 610041, China

  • Xiangya Hospital of Central South University

    Changsha, Hunan, 410008, China

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