A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies

January 23, 2026 updated by: Novartis Pharmaceuticals

Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy (SMA) Receiving Disease Modifying Therapies (DMT) - Real-world Analysis Using the Komodo Claims Data

The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam. This study was conducted using both open and closed claims 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.
4114

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 OAV, nusinersen, or risdiplam.

Description

Nusinersen and Risdiplam Cohorts:

Inclusion criteria

  • Patients with ≥1 SMA diagnosis (International Classification of Diseases, Tenth Revision, Clinical Modification [ICD-10-CM] codes: G12.0, G12.1, G12.9) at any time
  • Patients with ≥1 record of nusinersen or risdiplam in the data based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC) on or after December 23, 2016
  • Patients aged ≥ 2 at the index date
  • Patients with ≥ 1 quarter of clinical activities within 1 year prior to the index quarter (i.e., the quarter containing the index date)
  • Patients with ≥ 2 quarters of clinical activities in the first year of the follow-up

Exclusion criteria • None

Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:

Inclusion criteria

  • Patients with ≥1 SMA diagnosis (ICD-10-CM codes: G12.0, G12.1, G12.9) at any time
  • Patients with ≥1 record of OAV in the data based on relevant HCPCS codes and NDC on or after May 26, 2019
  • Patients under 2 years old at the index date
  • Patients with ≥ 2 quarters of clinical activities in their first year of follow-up

Exclusion criteria

• Treatment with nusinersen or risdiplam at any time

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.
Nusinersen Cohort
Patients with SMA aged two years or older who received nusinersen.
Risdiplam Cohort
Patients with SMA aged two years or older who received risdiplam.
Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort
Patients with SMA who were under two years of age and received OAV monotherapy.

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
Time Frame
Number of Patients by DMT During Each Year of DMT Treatment
Time Frame: Years 1, 2, 3, 4, and 5
Years 1, 2, 3, 4, and 5
Number of Patients Using Nusinersin During the Entire Treatment Follow-up Period
Time Frame: Up to 5 years
Up to 5 years
Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment
Time Frame: Years 1, 2, 3, 4, and 5
Years 1, 2, 3, 4, and 5
Annualized Drug and Drug Administration Costs for Nusinersen During the Entire Treatment Follow-up Period
Time Frame: Up to 5 years
Up to 5 years
Number of Nusinersen Injections During Each Year of Treatment
Time Frame: Years 1, 2, 3, 4, and 5
Years 1, 2, 3, 4, and 5
Number of Nusinersen Injections During the Entire Treatment Follow-up Period
Time Frame: Up to 5 years
Up to 5 years
Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period
Time Frame: Up to 4 years
Up to 4 years
Annualized Drug Costs During Each Year of Risdiplam Treatment
Time Frame: Years 1, 2, 3, and 4
Years 1, 2, 3, and 4
Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period
Time Frame: Up to 4 years
Up to 4 years
Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1
Time Frame: Year 1
Year 1
Mean Prescribed Risdiplam Dosage During Each Year of Treatment
Time Frame: Year 1, 2, 3, and 4
Year 1, 2, 3, and 4
Mean Number of Days of Supply of Risdiplam During Each Year of Treatment
Time Frame: Year 1, 2, 3, and 4
Year 1, 2, 3, and 4
Mean Daily Dosage of Risdiplam During Each Year of Treatment
Time Frame: Year 1, 2, 3, and 4
Year 1, 2, 3, and 4
Mean Prescribed Risdiplam Dosage During the Entire Treatment Follow-up Period
Time Frame: Up to 4 years
Up to 4 years
Mean Number of Days of Supply of Risdiplam During the Entire Treatment Follow-up Period
Time Frame: Up to 4 years
Up to 4 years
Mean Daily Dosage of Risdiplam During the Entire Treatment Follow-up Period
Time Frame: Up to 4 years
Up to 4 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
Measure Description
Time Frame
Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up
Time Frame: Years 1, 2, 3, and 4
All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs.
Years 1, 2, 3, and 4
Annualized All-cause Healthcare Costs During the Entire OAV Follow-up Period
Time Frame: Up to 4 years
All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs.
Up to 4 years
Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up
Time Frame: Years 1, 2, 3, and 4
SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME).
Years 1, 2, 3, and 4
Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period
Time Frame: Up to 4 years
SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME).
Up to 4 years

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.
November 6, 2024

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.
March 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.
March 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).
January 23, 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.
January 23, 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.
January 30, 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.
January 30, 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.
January 23, 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.
January 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.
  • COAV101A1US10

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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