Muscular Atrophy, SpinalNCT07378943
A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
An observational study of Muscular Atrophy, Spinal, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Muscular Atrophy, Spinal
- How many people
- 4,114 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Nov 2024to 30 Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OAV, nusinersen, or risdiplam.
Who may be able to join
- 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
- Patients with ≥1 SMA diagnosis (ICD-10-CM codes: G12.0, G12.1, G12.9) at any time
Show all 9Show fewer
- 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
Who cannot join
- Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:
- Treatment with nusinersen or risdiplam at any time
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
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.
- Conditions
- Muscular Atrophy, Spinal
- Keywords
- Spinal muscular atrophy, Disease modifying therapies, Real-world evidence, Dosing patterns, Treatment costs, Healthcare costs
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients by DMT During Each Year of DMT Treatment
Measured: Years 1, 2, 3, 4, and 5
Number of Patients Using Nusinersin During the Entire Treatment Follow-up Period
Measured: Up to 5 years
Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment
Measured: Years 1, 2, 3, 4, and 5
Annualized Drug and Drug Administration Costs for Nusinersen During the Entire Treatment Follow-up Period
Measured: Up to 5 years
Number of Nusinersen Injections During Each Year of Treatment
Measured: Years 1, 2, 3, 4, and 5
Number of Nusinersen Injections During the Entire Treatment Follow-up Period
Measured: Up to 5 years
Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period
Measured: Up to 4 years
Annualized Drug Costs During Each Year of Risdiplam Treatment
Measured: Years 1, 2, 3, and 4
Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period
Measured: Up to 4 years
Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1
Measured: Year 1
Mean Prescribed Risdiplam Dosage During Each Year of Treatment
Measured: Year 1, 2, 3, and 4
Mean Number of Days of Supply of Risdiplam During Each Year of Treatment
Measured: Year 1, 2, 3, and 4
Mean Daily Dosage of Risdiplam During Each Year of Treatment
Measured: Year 1, 2, 3, and 4
Mean Prescribed Risdiplam Dosage During the Entire Treatment Follow-up Period
Measured: Up to 4 years
Mean Number of Days of Supply of Risdiplam During the Entire Treatment Follow-up Period
Measured: Up to 4 years
Mean Daily Dosage of Risdiplam During the Entire Treatment Follow-up Period
Measured: Up to 4 years
Secondary outcomes (4)
Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up
All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs.
Measured: Years 1, 2, 3, and 4
Annualized All-cause Healthcare Costs During the Entire OAV Follow-up Period
All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs.
Measured: Up to 4 years
Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up
SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME).
Measured: Years 1, 2, 3, and 4
Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period
SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME).
Measured: Up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2026
- Study start6 Nov 2024
- Primary completion30 Mar 2025
- Study completion30 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 4,114 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07378943
- Sponsor's protocol ID
- COAV101A1US10
- Status last verified
- Jan 2026
- First submitted
- 23 Jan 2026
- Last updated
- 30 Jan 2026
- Results posted
- No
- Individual data sharing
- No
Publications
Toro Jimenez W, Yang M, Song W, Lemus Wirtz E, Ye D. Longitudinal Dosing Patterns and Treatment Costs Among Patients with Spinal Muscular Atrophy Initiating Nusinersen and Risdiplam. Adv Ther. 2026 Aug 6. doi: 10.1007/s12325-026-03687-0. Online ahead of print.
PubMed 42563034
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07378943
- Acronym / sponsor ID
- COAV101A1US10
- Conditions
- Muscular Atrophy, Spinal
- Keywords
- Spinal muscular atrophy, Disease modifying therapies, Real-world evidence, Dosing patterns, Treatment costs, Healthcare costs
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4,114 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 6 Nov 2024 (actual)
- Primary completion
- 30 Mar 2025 (actual)
- Study completion
- 30 Mar 2025 (actual)
- First posted
- 30 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OAV, nusinersen, or risdiplam.
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (2 listed)
- Treatment with nusinersen or risdiplam at any time
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of Patients by DMT During Each Year of DMT Treatment
Time frame: 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
Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment
Time frame: 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
Number of Nusinersen Injections During Each Year of Treatment
Time frame: Years 1, 2, 3, 4, and 5
Number of Nusinersen Injections During the Entire Treatment Follow-up Period
Time frame: Up to 5 years
Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period
Time frame: Up to 4 years
Annualized Drug Costs During Each Year of Risdiplam Treatment
Time frame: Years 1, 2, 3, and 4
Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period
Time frame: Up to 4 years
Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1
Time frame: Year 1
Mean Prescribed Risdiplam Dosage During Each Year of Treatment
Time frame: 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
Mean Daily Dosage of Risdiplam During Each Year of Treatment
Time frame: Year 1, 2, 3, and 4
Mean Prescribed Risdiplam Dosage During the Entire Treatment Follow-up Period
Time frame: 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
Mean Daily Dosage of Risdiplam During the Entire Treatment Follow-up Period
Time frame: Up to 4 years
4 secondary outcomes
Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up
Time frame: Years 1, 2, 3, and 4
Annualized All-cause Healthcare Costs During the Entire OAV Follow-up Period
Time frame: Up to 4 years
Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up
Time frame: Years 1, 2, 3, and 4
Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period
Time frame: Up to 4 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2026.
- Derived from this study: PubMed 42563034Toro Jimenez W, Yang M, Song W, Lemus Wirtz E, Ye D. Longitudinal Dosing Patterns and Treatment Costs Among Patients with Spinal Muscular Atrophy Initiating Nusinersen and Risdiplam. Adv Ther. 2026 Aug 6. doi: 10.1007/s12325-026-03687-0. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Atrophy, Spinal, run by Novartis Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients by DMT During Each Year of DMT Treatment (time frame: Years 1, 2, 3, 4, and 5). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07378943, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2026.