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New hope for SMA babies: risdiplam trial launches in newborns

NCT ID NCT05808764

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study is testing the drug risdiplam in newborn babies with spinal muscular atrophy (SMA), a serious muscle-weakening disease. The goal is to see how the drug moves through the body and if it is safe for infants under 20 days old. Ten babies will take part, and researchers will monitor side effects and drug levels in the blood.

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

11 people

The number who actually took part.

Started

Apr 2024

Finished

May 2026

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

Up to 19 days

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: * Male or female newborn infant aged \<20 days at first dose * Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing. * Gestational age equal to or greater than 37 weeks * Receiving adequate nutrition and hydration at the time of screening * Adequately recovered from any acute illness at baseline and considered well enough to participate in the study * Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator. Exclusion Criteria: * Presence of clinical symptoms or signs consistent with SMA Type 0 * In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures * Systolic blood pressure or diastolic blood pressure or heart rate abnormalities * Presence of clinically relevant electrocardiogram (ECG) abnormalities * The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing * Concurrent or previous administration of nusinersen or onasemnogene abeparvovec * Clinically significant abnormalities in laboratory test

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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

  • Ann and Robert H. Lurie Children Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • CHR Citadelle

    Liège, 4000, Belgium

  • Children'S Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Clinic for Special Children.

    Gordonville, Pennsylvania, 17529, United States

  • Fondazione Policlinico Univeristario A. Gemelli

    ROMA, Emilia-Romagna, 00168, Italy

  • Fondazione Serena Onlus - CENTRO CLINICO NEMO

    Milano, Emilia-Romagna, 20162, Italy

  • Hopital Universitaire des Enfants Reine Fabiola

    Brussels, 1020, Belgium

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    Warsaw, 04-730, Poland

  • OUS (Oslo University Hospital), Rikshospitalet

    Oslo, 0372, Norway

  • UMC Utrecht

    Utrecht, 3508, Netherlands

  • Universitatsklinikum Essen

    Essen, 45147, Germany

  • University Of Michigan

    Ann Arbor, Michigan, 48109, United States

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-952, Poland

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