A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT05337553. 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
A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.
Study identification
- NCT ID
- NCT05337553
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 269 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 21 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 6, 2022 (Actual)
- Primary completion
- Sep 25, 2024 (Actual)
- Completion
- Dec 2027 (Estimated)
- Last update posted
- Jun 1, 2026
2022 – 2027
United States locations
- U.S. sites
- 30
- U.S. states
- 21
- U.S. cities
- 30
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's | Phoenix | Arizona | 85016 | — |
| UCSD & Rady Children's | La Jolla | California | 92037 | — |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | — |
| UCSF Benioff Children's Hospital, Medical Center | San Francisco | California | 94158 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| UF Health, Shands Hospital | Gainesville | Florida | 32610 | — |
| Rare Disease Research | Atlanta | Georgia | 30329 | — |
| Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Indiana University -Riley Research | Indianapolis | Indiana | 46202 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Fairway | Kansas | 66205 | — |
| Boston Children's Hospital - Harvard | Boston | Massachusetts | 02115 | — |
| BSHS Office of Research | Grand Rapids | Michigan | 49503 | — |
| Washington University in St. Louis | St Louis | Missouri | 63110 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Stony Brook University Hospital | Stony Brook | New York | 11794 | — |
| Duke University Medicine | Durham | North Carolina | 27705 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Penn State College of Medicine | Hershey | Pennsylvania | 17033 | — |
| CHOP Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| UT Pediatric Neurosciences/Dell Children's Medical Center | Austin | Texas | 78723 | — |
| Neurology Rare Disease Center | Denton | Texas | 75208 | — |
| Cook Children's Hospital | Fort Worth | Texas | 76104 | — |
| University of Virginia Children's Hospital | Charlottesville | Virginia | 22903 | — |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | — |
| MultiCare Institute of Research and Innovation | Tacoma | Washington | 98405 | — |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 23 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05337553, 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 Jun 1, 2026 · Synced Oct 27, 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 NCT05337553 live on ClinicalTrials.gov.