Muscular Atrophy, SpinalNCT07403214
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
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 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Muscular Atrophy, Spinal
- How many people
- 4,805 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Jun 2025to 30 Jun 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 OA, nusinersen, or risdiplam.
Who may be able to join
- 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.
Who cannot join
- Patients with SMA type 4.
- Patients with claims of multiple DMTs in their birth year.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data
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
- 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 |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to an average of approximately 2 years
Inpatient Visit Length of Stay PPPY During Period I
Measured: Up to approximately 1 year
Inpatient Visit Length of Stay PPPY During Period II
Measured: Up to an average of approximately 2 years
Inpatient Visit Length of Stay PPPY During Period III
Measured: 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
Measured: Up to approximately 5 years and 5 months
Time Between the First and Second DMT Initiation Among Patients Treated With Multiple DMTs
Measured: Up to an average of approximately 2 years
Time Between the Second and Third DMT Initiation Among Patients Treated With Multiple DMTs
Measured: Up to an average of approximately 2 years
Time on the First DMT During Period II Among Patients Treated With Multiple DMTs
Measured: Up to an average of approximately 2 years
Time on the First DMT During Period III Among Patients Treated With Multiple DMTs
Measured: Up to an average of approximately 2 years
Time on the Second DMT During Period III Among Patients Treated With Multiple DMTs
Measured: Up to an average of approximately 2 years
Number of Patients who Started a Second DMT
Measured: Up to approximately 5 years and 5 months
Duration of Continuous Enrollment for Patients Treated With at Least 1 DMT
Measured: Up to approximately 5 years and 5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2026
- Study start4 Jun 2025
- Primary completion30 Jun 2025
- Study completion30 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 4,805 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
- NCT07403214
- Sponsor's protocol ID
- COAV101A1US09
- Status last verified
- Feb 2026
- First submitted
- 4 Feb 2026
- Last updated
- 11 Feb 2026
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data
- ClinicalTrials.gov ID
- NCT07403214
- Acronym / sponsor ID
- COAV101A1US09
- Conditions
- Muscular Atrophy, Spinal
- Keywords
- Spinal muscular atrophy, Multiple disease modifying therapies, Real-world evidence, Treatment patterns
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4,805 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 4 Jun 2025 (actual)
- Primary completion
- 30 Jun 2025 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 11 Feb 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 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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| 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. |
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 OA, nusinersen, or risdiplam.
Key inclusion criteria (6 listed)
- 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).
Key exclusion criteria (2 listed)
- Patients with SMA type 4.
- Patients with claims of multiple DMTs in their birth year.
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 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.
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.
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.
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.
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.
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.
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.
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.
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.
Inpatient Visit Length of Stay PPPY During Period I
Time frame: Up to approximately 1 year
Inpatient Visit Length of Stay PPPY During Period II
Time frame: Up to an average of approximately 2 years
Inpatient Visit Length of Stay PPPY During Period III
Time frame: Up to an average of approximately 2 years
8 secondary outcomes
Number of Patients who Received Multiple DMTs by Order in Which DMTs were Received
Time frame: Up to approximately 5 years and 5 months
Time Between the First and Second DMT Initiation Among Patients Treated With Multiple DMTs
Time frame: Up to an average of approximately 2 years
Time Between the Second and Third DMT Initiation Among Patients Treated With Multiple DMTs
Time frame: Up to an average of approximately 2 years
Time on the First DMT During Period II Among Patients Treated With Multiple DMTs
Time frame: Up to an average of approximately 2 years
Time on the First DMT During Period III Among Patients Treated With Multiple DMTs
Time frame: Up to an average of approximately 2 years
Time on the Second DMT During Period III Among Patients Treated With Multiple DMTs
Time frame: Up to an average of approximately 2 years
Number of Patients who Started a Second DMT
Time frame: Up to approximately 5 years and 5 months
Duration of Continuous Enrollment for Patients Treated With at Least 1 DMT
Time frame: Up to approximately 5 years and 5 months
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 11 Feb 2026.
- Individual participant dataSharing planned: No
Related trials for Muscular Atrophy, Spinal
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
Spinal Muscle Atrophy
RecruitingPhase 3
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
Metastatic Castration Resistant Prostate Cancer
Recruiting
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
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 6 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 11 Feb 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 SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I) (time frame: Up to approximately 1 year). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Feb 2026.