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New daily pill shows promise for spinal muscular atrophy

NCT ID NCT03032172

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the safety and drug levels of risdiplam, a daily oral medication, in 174 adults and children with spinal muscular atrophy (SMA). Participants had previously received other SMA treatments. The main goals were to check for side effects and measure how the drug moves through the body. Results will help guide future use of risdiplam for SMA.

What this could mean

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

Active substance
risdiplam
What this could lead to
If it works, this could provide a daily oral treatment option for spinal muscular atrophy, potentially improving motor function and survival.
What could go wrong
This is an early-phase, open-label study without a comparison group, so results may not confirm effectiveness. Side effects and long-term risks are still being evaluated.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

174 people

The number who actually took part.

Started

Mar 2017

Finished

Feb 2025

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

6 months to 60 years

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: * Confirmed diagnosis of 5q-autosomal recessive SMA * Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previous treatment with any of the following: 1.) Nusinersen (defined as having received \>= 4 doses of nusinersen, provided that the last dose was received \>= 90 days prior to screening) or 2.) Olesoxime (provided that the last dose was received \<= 12 months and \>= 90 days prior to screening) or 3.) AVXS-101 (provided that the time of treatment was \>= 12 months prior to screening) * Adequately recovered from any acute illness at the time of screening and considered well enough to participate in the opinion of the Investigator * For women of childbearing potential: negative blood pregnancy test at screening, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating eggs for at least 28 days after the final dose of study drug * For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures and agreement to refrain from donating sperm * For participants aged 2 years or younger at screening: 1.) Parent or caregiver of participant is willing to consider nasogastric, naso-jejunal or gastrostomy tube placement, as recommended by the Investigator, during the study to maintain safe hydration, nutrition and treatment delivery; 2.) Parent or caregiver of participant is willing to consider the use of non-invasive ventilation, as recommended by the Investigator during the study Exclusion Criteria: * Inability to meet study requirements * Concomitant participation in any investigational drug or device study * Previous participation in any investigational drug or device study within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer with the exception of studies of olesoxime, AVXS-101, or nusinersen * Any history of gene or cell therapy, with the exception of AVXS-101 * Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator * Inadequate venous or capillary blood access for the study procedures, in the opinion of the Investigator * For patients aged \< 2 years, hospitalization for a pulmonary event within 2 months prior to screening and pulmonary function not fully recovered at the time of screening * Lactating women * Suspicion of regular consumption of drugs of abuse * For adults and adolescents only, positive urine test for drugs of abuse or alcohol at screening or Day -1 visit * Presence of clinically significant electrocardiogram (ECG) abnormalities before study drug administration from average of triplicate measurement or cardiovascular disease * History of malignancy if not considered cured * For participants aged \> 6 years, significant risk for suicidal behavior, in the opinion of the Investigator as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS) * Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration * Recently initiated treatment for spinal muscular atrophy (within \<6 weeks prior to enrollment) with oral salbutamol or another beta 2-adrenergic agonist taken orally * Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine, is not allowed * Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to risdiplam or to the constituents of its formulation * Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the participant in this study * Recent history (less than one year) of ophthalmological diseases * Any prior use of an inhibitor or inducer of FMO1 or FMO3 taken within 2 weeks (or within 5 elimination half-lives, whichever is longer) prior to dosing

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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

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS, United Kingdom

  • Boston Childrens Hospital

    Boston, Massachusetts, 02115, United States

  • CHRU de Montpellier, Hopital Gui de Chauliac

    Montpellier, 34295, France

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Lombardy, 20122, Italy

  • Hopital Femme Mere Enfant

    Bron, 69677, France

  • Hopital Roger Salengro

    Lille, 59037, France

  • Hopital des Enfants

    Toulouse, 31059, France

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

  • IRCCS Istituto Giannina Gaslini

    Genoa, Liguria, 16147, Italy

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, Lazio, 00165, Italy

  • Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie

    Warsaw, 02-097, Poland

  • Nemours Children's Hospital

    Orlando, Florida, 32827, United States

  • Policlinico Agostino Gemelli

    Rome, Lazio, 00168, Italy

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • The Newcastle upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • UCL Institute of Child Health & Great Ormond Street Hospital for Children

    London, WC1N 1EH, United Kingdom

  • UMC Utrecht

    Utrecht, 3584 CX, Netherlands

  • UOSD Malattie Neurodegenerative

    Messina, Sicily, 98125, Italy

  • UZ Gent

    Ghent, 9000, Belgium

  • UZ Leuven Gasthuisberg

    Leuven, 3000, Belgium

  • Universitäts-Kinderspital (UKBB) Neuropädiatrie

    Basel, 4005, Switzerland

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

More trials for these conditions

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