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A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Information (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for approximately 24 months from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study if they agree to continue participating in the follow-up assessments.
An optional sub-study was planned to assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. This substudy was withdrawn upon implementation of protocol version 4.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Risdiplam | Experimental | Participants who are taking risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI are enrolled in this study. A Remote Virtual Site option is available in the study allowing enrollment of participants with SMA who are prescribed risdiplam treatment even if their physician is not a participating investigator. A central investigator will be responsible for data collection and data entry for participants who do not have a participating investigator. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Risdiplam | Drug | Risdiplam taken by participants as prescribed by their physician. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Pre-existing conditions which worsen during a study are also considered as AE. | From enrollment up to the duration of the study or until withdrawal of consent, loss to follow-up, or death (up to approximately 24 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale | The CGI-C is used to score a clinician's impression of a participant's change in global health. The CGI-C is a single item measure of change in global health, using seven response options: "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", and "very much worse". Participants considered as "improved" include responses of "very much improved, "much improved" and "minimally improved". |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | United States | ||
| Phoenix Children's Hospital |
For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
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| From enrollment up to the duration of the study or until withdrawal of consent, loss to follow-up, or death (up to approximately 24 months) |
| Phoenix |
| Arizona |
| 85016 |
| United States |
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72103 | United States |
| Loma Linda University Health Care | Loma Linda | California | 92354 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90010 | United States |
| Valley Children's Hospital | Madera | California | 93636 | United States |
| University California - Irvine | Orange | California | 92868 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| University of Colorado | Aurora | Colorado | 80045 | United States |
| Medstar Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | United States |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| Nemour's Children's Hospital, Florida | Orlando | Florida | 32827 | United States |
| All Children's Research Institute, Inc. | St. Petersburg | Florida | 33701 | United States |
| Advent Health Orlando | Winter Park | Florida | 32789 | United States |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | United States |
| University of Kentucky Medical Center | Lexington | Kentucky | 40536 | United States |
| Norton Children's Hospital | Louisville | Kentucky | 40202 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| University of Michigan Pediatric Rehabilitation Center | Ann Arbor | Michigan | 48109 | United States |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan | 49503 | United States |
| Gillette Children's Specialty Healthcare | Minnetonka | Minnesota | 55343 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Washington University;Wash Uni. Sch. Of Med | St Louis | Missouri | 63110 | United States |
| NYU Hospital for Joint Diseases | New York | New York | 10003 | United States |
| Columbia University Med Center | New York | New York | 10032 | United States |
| Cincinnati Childrens Hospital | Cincinnati | Ohio | 45229 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Childrens Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| University of Texas at Austin Health sciences, Dell Medical School | Austin | Texas | 78723 | United States |
| Neurology & Neuromuscular Care Center | Denton | Texas | 76208 | United States |
| Central Texas Neurology Consultants | Round Rock | Texas | 78681 | United States |
| Methodist Children's Hospital of South Texas | San Antonio | Texas | 78229 | United States |
| University Of Utah | Salt Lake City | Utah | 84108 | United States |
| University of Virginia Children?s Hospital | Charlottesville | Virginia | 22903 | United States |
| MultiCare Health System Institute for Research and Innovation | Tacoma | Washington | 98405 | United States |
| UBC (Remote Coordinating Center, no physical facility) | Morgantown | West Virginia | 26508 | United States |
| Childrens Hospital of Wisconsin | Milwaukee | Wisconsin | 53201 | United States |
| Instituto de Rehabilitación del Caribe | Santurce | 00912 | Puerto Rico |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
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| ID | Term |
|---|---|
| C000629884 | Risdiplam |
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