A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

February 4, 2026 updated by: Novartis Pharmaceuticals

Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data

The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world. The DMTs included onasemnogene abeparvovec-xioi (OA), nusinersen, and risdiplam. This study was conducted using data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
4805

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New Jersey
      • East Hanover, New Jersey, United States, 07936
        • Novartis

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
DMT Cohort A
Patients with SMA who were treated with at least 1 DMT and had continuous health insurance coverage in KRD+ closed claims at the time of first DMT initiation.
DMT Cohort B
Patients with SMA who were treated with at least 1 DMT and received the first DMT at age 2 or younger.
DMT Cohort B1
A sub-group of DMT Cohort B who started the first DMT between 2016 and 2021.
DMT Cohort B2
A sub-group of DMT Cohort B who started the first DMT between 2022 and 2024.
DMT Cohort C
Patients with SMA types 1-3 who were treated with multiple DMTs.
DMT Cohort D
A subset of DMT Cohort C who received the first DMT at age two or younger.
DMT Cohort D1
A sub-group of DMT Cohort D who started the first DMT between 2016 and 2021.
DMT Cohort D2
A sub-group of DMT Cohort D who started the first DMT between 2022 and 2024.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I)
Time Frame: Up to approximately 1 year
DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time.
Up to approximately 1 year
Number of Patients by SMA-related Complication, Procedure, and DME After Receiving the First DMT and Before the Second DMT (Period II)
Time Frame: Up to an average of approximately 2 years
DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time.
Up to an average of approximately 2 years
Number of Patients by SMA-related Complication, Procedure, and DME After Receiving the Second DMT and Before the Third DMT (Period III)
Time Frame: Up to an average of approximately 2 years
DME is medical equipment that assists patients with their medical condition, can be used at home, and lasts a long time.
Up to an average of approximately 2 years
Number of Patients by All-cause Medical Encounters During Period I
Time Frame: Up to approximately 1 year
Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.
Up to approximately 1 year
Number of Patients by All-cause Medical Encounters During Period II
Time Frame: Up to an average of approximately 2 years
Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.
Up to an average of approximately 2 years
Number of Patients by All-cause Medical Encounters During Period III
Time Frame: Up to an average of approximately 2 years
Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.
Up to an average of approximately 2 years
Number of Medical Encounters per Patient per Year (PPPY) During Period I
Time Frame: Up to approximately 1 year
Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.
Up to approximately 1 year
Number of Medical Encounters PPPY During Period II
Time Frame: Up to an average of approximately 2 years
Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.
Up to an average of approximately 2 years
Number of Medical Encounters PPPY During Period III
Time Frame: Up to an average of approximately 2 years
Medical encounters included outpatient visits, inpatient admissions, emergency room visits, and specialist visits.
Up to an average of approximately 2 years
Inpatient Visit Length of Stay PPPY During Period I
Time Frame: Up to approximately 1 year
Up to approximately 1 year
Inpatient Visit Length of Stay PPPY During Period II
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years
Inpatient Visit Length of Stay PPPY During Period III
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Number of Patients who Received Multiple DMTs by Order in Which DMTs were Received
Time Frame: Up to approximately 5 years and 5 months
Up to approximately 5 years and 5 months
Time Between the First and Second DMT Initiation Among Patients Treated With Multiple DMTs
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years
Time Between the Second and Third DMT Initiation Among Patients Treated With Multiple DMTs
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years
Time on the First DMT During Period II Among Patients Treated With Multiple DMTs
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years
Time on the First DMT During Period III Among Patients Treated With Multiple DMTs
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years
Time on the Second DMT During Period III Among Patients Treated With Multiple DMTs
Time Frame: Up to an average of approximately 2 years
Up to an average of approximately 2 years
Number of Patients who Started a Second DMT
Time Frame: Up to approximately 5 years and 5 months
Up to approximately 5 years and 5 months
Duration of Continuous Enrollment for Patients Treated With at Least 1 DMT
Time Frame: Up to approximately 5 years and 5 months
Up to approximately 5 years and 5 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 4, 2025

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 30, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 30, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 4, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 4, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 11, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 11, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 4, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • COAV101A1US09

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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