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New study tracks Risdiplam's Real-World impact on spinal muscle atrophy

NCT ID NCT07531719

Knowledge-focused 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Sep 04, 2026 · Updated 6 times

Summary

This study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.

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 successful, this study could provide real-world evidence on how well risdiplam improves motor function in young children with spinal muscle atrophy.
What could go wrong
This is an observational study, not a controlled trial, so results may be less definitive. It is also small (30 participants) and not yet recruiting.

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 30 people

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

Started

Jul 2026

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.

Who is studied

The participants must have a confirmed diagnosis of 5q-autosomal recessive SMA type I or type II with at least two survival motor neuron 2 (SMN2) copies for which Risdiplam is prescribed as per the SmPC.

Ages

Up to 5 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: * Maximum age to be 6 months for type I participants and 5 years for type II participants at the time of diagnosis. * Participants must start/have started treatment with Risdiplam within 6 months of diagnosis. * Participant to be receiving adequate nutrition and hydration at index date, in the opinion of the Investigator. * Participant to be adequately recovered from any acute illness at the time index date. Exclusion Criteria: * Participants not receiving treatment for disease with Risdiplam according to standard of care and in line with the current summary of product characteristics (SPC)/local labeling. * Participants not receiving the Roche studied medicinal product, but a biosimilar. * Concomitant or previous administration in any investigational drug or device study. * Concomitant or previous administration of approved treatment for SMA other than Risdiplam. * Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system diseases at index date. * Participants requiring invasive ventilation or tracheostomy at index date. * Participants requiring awake non-invasive ventilation due to respiratory insufficiency or with awake hypoxemia with or without ventilator support or with history of respiratory failure or severe pneumonia, and have not fully recovered their pulmonary function at the time of index date. * Severe contractures of joints of lower limbs and upper limbs at the time of index date.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy spinal muscular atrophy, type 1 spinal muscular atrophy, type II

As listed by the trial registrant

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

Spinal muscle atrophy

How to take part

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

  1. The places running it

    2 sites. 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

  • Air Force Specialized Hospital

    RECRUITING

    Cairo, DUMMY_VALUE, Egypt

  • Neurology Department, Ain Shams University Hospitals

    RECRUITING

    Cairo, 11566, Egypt

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