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Massive data dive reveals how SMA drugs perform outside the lab

NCT ID NCT07403214

Knowledge-focused Sponsor: Novartis Pharmaceuticals 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 analyzed health records of nearly 5,000 people with spinal muscular atrophy (SMA) to see how well three approved treatments work in everyday medical practice. Researchers looked at complications, medical equipment use, and hospital visits before and after starting each therapy. The goal was to understand real-world treatment patterns and outcomes, not to test a new drug.

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

4,805 people

The number who actually took part.

Started

Jun 2025

Finished

Jun 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.

Who is studied

SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OA, nusinersen, or risdiplam.

Ages

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

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 * Patients with ≥1 SMA diagnosis at any time (International Classification of Disease, Tenth Revision, Clinical Modification \[ICD-10-CM\] codes: G12.0, G12.1, G12.9). * Patients with ≥1 record of OA, nusinersen, or risdiplam based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC). * Patients who received at least 2 of the 3 DMTs, with the first DMT initiated on or after 26 May 2019, when at least 2 DMTs were available. The initiation of the first DMT was defined as the index date and the first DMT was defined as the index DMT. * Patients with SMA types 1, 2, or 3. * Patients with ≥1 quarter of clinical activities within 1 year prior to the index quarter (for patients with SMA type 2 and 3 only). * Patients with ≥1 quarter of clinical activities any time after the index quarter, unless death occurred. Exclusion criteria * Patients with SMA type 4. * Patients with claims of multiple DMTs in their birth year.

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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

  • Novartis

    East Hanover, New Jersey, 07936, United States

More trials for these conditions

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