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

NCT07403214 | Completed

• 1 other identifier: COAV101A1US09
• Study Type: observational
• Target: 4,805
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Muscular Atrophy, Spinal

Keywords

Spinal muscular atrophy; Multiple disease modifying therapies; Real-world evidence; Treatment patterns

Outcome Measures

Primary Outcomes (12)

• Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I)

  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)

  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)

  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

  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

  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

  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

  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

  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

  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

  Up to approximately 1 year

• Inpatient Visit Length of Stay PPPY During Period II

  Up to an average of approximately 2 years

• Inpatient Visit Length of Stay PPPY During Period III

  Up to an average of approximately 2 years

Secondary Outcomes (8)

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

  Up to approximately 5 years and 5 months

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

  Up to an average of approximately 2 years

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

  Up to an average of approximately 2 years

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

  Up to an average of approximately 2 years

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

  Up to an average of approximately 2 years

Study Arms (8)

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Patients with SMA type 4.
• Patients with claims of multiple DMTs in their birth year.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2026
• First Posted: February 11, 2026
• Study Start: June 4, 2025
• Primary Completion: June 30, 2025
• Study Completion: June 30, 2025
• Last Updated: February 11, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)