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SMA drug risdiplam under Long-Term watch: will it hold up?

NCT ID NCT05232929

Disease control Sponsor: Genentech, Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Sep 10, 2026 · Updated 5 times

Summary

This study follows 403 adults and children with spinal muscular atrophy who are already taking risdiplam (Evrysdi) as prescribed by their doctor. Researchers will track side effects and how well the drug works over about two years. The goal is to see if risdiplam remains safe and effective for long-term use.

What this could mean

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

Active substance
risdiplam (Evrysdi)
What this could lead to
If successful, this study will confirm that risdiplam remains safe and effective for long-term use in managing spinal muscular atrophy.
What could go wrong
This is an observational follow-up, not a test of a new treatment. It may not prove long-term benefit beyond what is already known.

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

403 people

The number who actually took part.

Started

Mar 2022

Expected to finish

Oct 2026

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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.

Inclusion Criteria: * Provide informed consent or assent when appropriate, as determined by the participant's age and individual site and local standards * Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the risdiplam (Evrysdi) USPI, after U.S. FDA approval (07 August 2020) Exclusion Criteria: * Hypersensitivity to risdiplam * Participated in a registrational trial for risdiplam (i.e., Firefish \[NCT02913482\], Sunfish \[NCT02908685\], Jewelfish \[NCT03032172\], and Rainbowfish \[NCT03779334\])

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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.

Spinal muscular atrophy

Contacts and locations

Locations

  • Advent Health Orlando

    Winter Park, Florida, 32789, United States

  • All Children's Research Institute, Inc.

    St. Petersburg, Florida, 33701, United States

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • Central Texas Neurology Consultants

    Round Rock, Texas, 78681, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90010, United States

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Childrens Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hospital of Wisconsin

    Milwaukee, Wisconsin, 53201, United States

  • Cincinnati Childrens Hospital

    Cincinnati, Ohio, 45229, United States

  • Columbia University Med Center

    New York, New York, 10032, United States

  • Gillette Children's Specialty Healthcare

    Minnetonka, Minnesota, 55343, United States

  • Helen DeVos Children's Hospital at Spectrum Health

    Grand Rapids, Michigan, 49503, United States

  • Instituto de Rehabilitación del Caribe

    Santurce, 00912, Puerto Rico

  • Loma Linda University Health Care

    Loma Linda, California, 92354, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Medstar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007, United States

  • Methodist Children's Hospital of South Texas

    San Antonio, Texas, 78229, United States

  • MultiCare Health System Institute for Research and Innovation

    Tacoma, Washington, 98405, United States

  • NYU Hospital for Joint Diseases

    New York, New York, 10003, United States

  • Nemour's Children's Hospital, Florida

    Orlando, Florida, 32827, United States

  • Neurology & Neuromuscular Care Center

    Denton, Texas, 76208, United States

  • Norton Children's Hospital

    Louisville, Kentucky, 40202, United States

  • Penn State Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • UBC (Remote Coordinating Center, no physical facility)

    Morgantown, West Virginia, 26508, United States

  • University California - Irvine

    Orange, California, 92868, United States

  • University Of Utah

    Salt Lake City, Utah, 84108, United States

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72103, United States

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of Colorado

    Aurora, Colorado, 80045, United States

  • University of Kentucky Medical Center

    Lexington, Kentucky, 40536, United States

  • University of Michigan Pediatric Rehabilitation Center

    Ann Arbor, Michigan, 48109, United States

  • University of Texas at Austin Health sciences, Dell Medical School

    Austin, Texas, 78723, United States

  • University of Virginia Children?s Hospital

    Charlottesville, Virginia, 22903, United States

  • Valley Children's Hospital

    Madera, California, 93636, United States

  • Washington University;Wash Uni. Sch. Of Med

    St Louis, Missouri, 63110, United States

More trials for these conditions

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