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Can a second treatment help kids with SMA who stalled after gene therapy?

NCT ID NCT05861999

Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 11, 2026 · Updated 6 times

Summary

This study tests whether adding risdiplam, a daily oral medicine, can help children under 2 with spinal muscular atrophy (SMA) who stopped getting better or started declining after receiving gene therapy. The study will enroll 28 children and measure changes in motor skills over 72 weeks. The goal is to see if risdiplam can improve movement and function beyond what gene therapy alone achieved.

What this could mean

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

Active substance
Risdiplam (a liquid medicine taken by mouth)
What this could lead to
If it works, this could offer a way to help children with SMA who have stopped improving after gene therapy regain some motor function.
What could go wrong
This is a small, early-phase study with only 28 children, so results may not apply to everyone. Risdiplam may cause side effects, and it is not known if it will reverse the plateau or decline.

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 28 people

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

Started

Aug 2024

Expected to finish

Mar 2029

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

3 to 24 months

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: * \<2 years of age at the time of informed consent * Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene * Confirmed presence of two SMN2 gene copies as documented through laboratory testing * Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically * Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment * If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration. * In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function/ability (respiratory, motor function, other) per appropriate expectation. Exclusion Criteria: * Previous or current enrolment in investigational study prior to initiation of study treatment * Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information * Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide * Concomitant or previous use of an anti-myostatin agent * Participants requiring invasive ventilation or tracheostomy * Presence of feeding tube and an OrSAT score of 0 * Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening * Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    18 sites in 6 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie

    RECRUITING

    Berlin, 13353, Germany

  • Children'S Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Healthcare of Atlanta Center for Advanced Pediatrics

    RECRUITING

    Atlanta, Georgia, 30329-2309, United States

  • Children's Hospital of Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of the King's Daughter

    WITHDRAWN

    Norfolk, Virginia, 23510, United States

  • Great Ormond Street Hospital For Children

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Helen DeVos Children's Hospital at Spectrum Health

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Instytut Pomnik Centrum Zdrowia Dziecka

    RECRUITING

    Warsaw, 04-730, Poland

  • Schneider Children's Medical Center of Israel

    RECRUITING

    Petah Tikva, 4920235, Israel

  • Sidra Medicine

    RECRUITING

    Al Rayyan, Qatar

  • Soroka Medical Center

    RECRUITING

    Beersheba, 8410101, Israel

  • Sourasky MC, Dana-Dwek Children's Hospital

    RECRUITING

    Tel Aviv, 6423906, Israel

  • Stanford Univ Medical Center

    RECRUITING

    Palo Alto, California, 94304, United States

  • UKGM Standort Gießen

    RECRUITING

    Giessen, 35392, Germany

  • University of Arkansas for Medical Sciences

    RECRUITING

    Little Rock, Arkansas, 72103, United States

  • University of Florida Pediatrics

    RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gda?sk, 80-952, Poland

  • Valley Children's Hospital

    RECRUITING

    Madera, California, 93636, United States

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