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Completed

NCT Number: NCT07403214

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

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.

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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.

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.

Treatment and study plan

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. Inpatient Visit Length of Stay PPPY During Period I

    Time frame: Up to approximately 1 year

  11. Inpatient Visit Length of Stay PPPY During Period II

    Time frame: Up to an average of approximately 2 years

  12. Inpatient Visit Length of Stay PPPY During Period III

    Time frame: Up to an average of approximately 2 years

Secondary outcomes

  1. Number of Patients who Received Multiple DMTs by Order in Which DMTs were Received

    Time frame: Up to approximately 5 years and 5 months

  2. Time Between the First and Second DMT Initiation Among Patients Treated With Multiple DMTs

    Time frame: Up to an average of approximately 2 years

  3. Time Between the Second and Third DMT Initiation Among Patients Treated With Multiple DMTs

    Time frame: Up to an average of approximately 2 years

  4. Time on the First DMT During Period II Among Patients Treated With Multiple DMTs

    Time frame: Up to an average of approximately 2 years

  5. Time on the First DMT During Period III Among Patients Treated With Multiple DMTs

    Time frame: Up to an average of approximately 2 years

  6. Time on the Second DMT During Period III Among Patients Treated With Multiple DMTs

    Time frame: Up to an average of approximately 2 years

  7. Number of Patients who Started a Second DMT

    Time frame: Up to approximately 5 years and 5 months

  8. Duration of Continuous Enrollment for Patients Treated With at Least 1 DMT

    Time frame: Up to approximately 5 years and 5 months

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2025
Study completion
2025
First posted
Feb 11, 2026
Registry last updated
Feb 11, 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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