An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Public ClinicalTrials.gov record NCT04256265. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
Study identification
- NCT ID
- NCT04256265
- Recruitment status
- Approved For Marketing
- Study type
- Expanded access
- Phase
- Not listed
- Enrollment
- Not listed
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Months and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- Oct 5, 2020
United States locations
- U.S. sites
- 29
- U.S. states
- 20
- U.S. cities
- 28
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Arkansas Children's Hospital; Pediatrics | Little Rock | Arkansas | 72202 | — |
| Children's Hospital Los Angeles | Los Angeles | California | 90010 | — |
| Stanford University | Palo Alto | California | 94304 | — |
| University of Colorado in Denver-Anschutz Medical Campus | Aurora | Colorado | 80045 | — |
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Comprehensive NeuroBehavioral Institute | Plantation | Florida | 33317 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | — |
| Ann and Robert H. Lurie Children Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Southern Illinois University, School of Medicine | Springfield | Illinois | 62702 | — |
| Indiana Hemophilia & Thrombosis center | Indianapolis | Indiana | 46260 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| University of Louisville | Louisville | Kentucky | 40202 | — |
| Massachusetts General Hospital; Neurology | Boston | Massachusetts | 02114 | — |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan | 49503 | — |
| University of Mississippi Medical Center; Neurology | Jackson | Michigan | 39216 | — |
| Gillette Spcl Children's Clin; Pediatric Endocrinology | Saint Paul | Minnesota | 55101 | — |
| St. Louis Children Hospital | St Louis | Missouri | 63110 | — |
| Goryeb Children's Hospital | Morristown | New Jersey | 07960 | — |
| Northwell Hospital | New Hyde Park | New York | 11042 | — |
| NYU Langone | New York | New York | 10003 | — |
| University of Rochester Medical Center | Rochester | New York | 14642 | — |
| Wake Forest Baptist Medical Center | Winston-Salem | North Carolina | 27157 | — |
| Akron Childrens Hospital | Akron | Ohio | 44308 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| University of Virginia Children's Hospital; Developmental | Charlottesville | Virginia | 22903 | — |
| University of Wisconsin American Family; Childrens Hospital | Madison | Wisconsin | 53792 | — |
| Childrens Hospital of Wisconsin | Milwaukee | Wisconsin | 53201 | — |
| Medical College of Wisconsin, Inc. | Milwaukee | Wisconsin | 53226-3596 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04256265, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 5, 2020 · Synced Oct 24, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04256265 live on ClinicalTrials.gov.