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Can treating spinal muscular atrophy in the womb be safe and helpful? a new registry aims to find out

NCT ID NCT07805278

Knowledge-focused Sponsor: St. Jude Children's Research Hospital 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 04, 2026 · Last updated Sep 09, 2026 · Updated 2 times

Summary

This study creates a registry of pregnancies where the fetus received a genetic diagnosis of spinal muscular atrophy (SMA) and was treated with a disease-modifying therapy while in the womb, such as risdiplam. Researchers will collect information from medical records and questionnaires about maternal and fetal monitoring, pregnancy outcomes, and delivery. The goal is to fill a gap in knowledge about the safety and potential benefits of prenatal SMA treatment, and to help guide future care and research.

What this could mean

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

Active substance
risdiplam (or other SMA-directed therapy given during pregnancy)
What this could lead to
If this registry gathers enough data, it could guide best practices for treating spinal muscular atrophy before birth and improve monitoring of mothers and babies.
What could go wrong
This is a small observational registry, not a controlled trial, so it cannot prove whether prenatal treatment works. It only collects what already happened, and results may not apply to everyone.

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.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Aug 2033

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Participants who meet the eligibility criteria and consent to participate.

Ages

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

Sex

Anyone

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: * Current or past pregnancy during which the fetus received a genetic diagnosis of SMA. * Current or past treatment with SMA-directed therapy intended to treat the fetus during gestation (such as risdiplam, nusinersen, onasemnogene abeparvovec, or another product intended to treat SMA). Exclusion Criteria: * Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

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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

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact Email: •••••@•••••

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