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Completed

NCT Number: NCT07378943

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

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.

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Key information

Conditions

Muscular Atrophy, Spinal Central Nervous System Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis

East Hanover, New Jersey, 07936, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Number of Patients by DMT During Each Year of DMT Treatment

    Time frame: Years 1, 2, 3, 4, and 5

  2. Number of Patients Using Nusinersin During the Entire Treatment Follow-up Period

    Time frame: Up to 5 years

  3. Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment

    Time frame: Years 1, 2, 3, 4, and 5

  4. Annualized Drug and Drug Administration Costs for Nusinersen During the Entire Treatment Follow-up Period

    Time frame: Up to 5 years

  5. Number of Nusinersen Injections During Each Year of Treatment

    Time frame: Years 1, 2, 3, 4, and 5

  6. Number of Nusinersen Injections During the Entire Treatment Follow-up Period

    Time frame: Up to 5 years

  7. Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period

    Time frame: Up to 4 years

  8. Annualized Drug Costs During Each Year of Risdiplam Treatment

    Time frame: Years 1, 2, 3, and 4

  9. Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period

    Time frame: Up to 4 years

  10. Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1

    Time frame: Year 1

  11. Mean Prescribed Risdiplam Dosage During Each Year of Treatment

    Time frame: Year 1, 2, 3, and 4

  12. Mean Number of Days of Supply of Risdiplam During Each Year of Treatment

    Time frame: Year 1, 2, 3, and 4

  13. Mean Daily Dosage of Risdiplam During Each Year of Treatment

    Time frame: Year 1, 2, 3, and 4

  14. Mean Prescribed Risdiplam Dosage During the Entire Treatment Follow-up Period

    Time frame: Up to 4 years

  15. Mean Number of Days of Supply of Risdiplam During the Entire Treatment Follow-up Period

    Time frame: Up to 4 years

  16. Mean Daily Dosage of Risdiplam During the Entire Treatment Follow-up Period

    Time frame: Up to 4 years

Secondary outcomes

  1. 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.

  2. 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.

  3. 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).

  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).

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Jan 30, 2026
Registry last updated
Jan 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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