NCT05337553Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
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
Source: ClinicalTrials.gov · Last updated 1 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 269 (actual)
- First submitted
- 14 Apr 2022
- Start date
- 6 Jul 2022
- Primary completion date
- 25 Sept 2024
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT05337553
- EU CT number
- 2024-511852-42-00
- EudraCT number
- 2022-000193-25
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 269
- From 6 Jul 2022 to the primary completion date, 25 Sept 2024
- 53 sites in 9 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Cannot have previously taken anti-myostatin therapies
Other illness
- Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)
- History of Spinal Fusion within 6 months of Screening. MAGEC rod nonsurgical adjustments are allowed during the study
Other
- Must weigh at least 15kg
- Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
Who the study is looking for
- Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
- Ambulant or Non-Ambulant
- Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen and/or risdiplam and/or a history of onasemnogene abeparvovec
Age in the registry: 4 years to 21 years · Sex: All · Does not accept healthy volunteers
Sites (53)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Antwerp University Hospital Coordinator of this site? Confirm its status | Edegem | Belgium | Active, not recruiting |
| Universiteit Gent Coordinator of this site? Confirm its status | Gent | Belgium | Active, not recruiting |
| UZ Leuven Coordinator of this site? Confirm its status | Leuven | Belgium | Active, not recruiting |
| Fakultni nemocnice Brno Coordinator of this site? Confirm its status | Brno | Czechia | Active, not recruiting |
| Fakultni nemocnice v Motole Coordinator of this site? Confirm its status | Prague | Czechia | Active, not recruiting |
| Universitaetsklinikum Essen (AoR) Coordinator of this site? Confirm its status | Essen | Germany | Active, not recruiting |
| Medical Center- University of Freiburg Coordinator of this site? Confirm its status | Freiburg im Breisgau | Germany | Active, not recruiting |
| Klinikum der Universitaet Muenchen AöR Coordinator of this site? Confirm its status | Munich | Germany | Active, not recruiting |
| Hospital Universitario Donostia Coordinator of this site? Confirm its status | Donostia | Spain | Active, not recruiting |
| Hospital Sant Joan De Deu Barcelona Coordinator of this site? Confirm its status | Esplugues de Llobregat | Spain | Active, not recruiting |
| Hospital Universitario Regional de Málaga Coordinator of this site? Confirm its status | Málaga | Spain | Active, not recruiting |
| Hospital Universitario Y Politecnico La Fe Coordinator of this site? Confirm its status | Valencia | Spain | Active, not recruiting |
| Royal Hospital for Children Coordinator of this site? Confirm its status | Glasgow | United Kingdom | Unknown status |
| John Radcliffe Hospital Coordinator of this site? Confirm its status | Oxford | United Kingdom | Unknown status |
| Azienda Unita Sanitaria Locale Di Bologna Coordinator of this site? Confirm its status | Bologna | Italy | Active, not recruiting |
| Centro Clinico Nemo Coordinator of this site? Confirm its status | Gussago | Italy | Active, not recruiting |
| Fondazione Istituto Neurologico Nazionale Casimiro Mondino Coordinator of this site? Confirm its status | Pavia | Italy | Active, not recruiting |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Rome | Italy | Active, not recruiting |
| Universitair Medisch Centrum Utrecht Coordinator of this site? Confirm its status | Utrecht | Netherlands | Active, not recruiting |
| Uniwersyteckie Centrum Kliniczne Coordinator of this site? Confirm its status | Gdansk | Poland | Active, not recruiting |
| Uniwersytecki Szpital Kliniczny w Poznaniu Coordinator of this site? Confirm its status | Poznan | Poland | Active, not recruiting |
| Instytut Pomnik Centrum Zdrowia Dziecka Coordinator of this site? Confirm its status | Warsaw | Poland | Active, not recruiting |
| Dolnośląski Szpital Specjalistyczny im. T. Marciniaka - Centrum Medycyny Ratunkowej Coordinator of this site? Confirm its status | Wroclaw | Poland | Active, not recruiting |
| Rare Disease Research Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Children's Hospital Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| UT Pediatric Neurosciences/Dell Children's Medical Center Coordinator of this site? Confirm its status | Austin | United States | Unknown status |
| Boston Children's Hospital - Harvard Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| University of Virginia Children's Hospital Coordinator of this site? Confirm its status | Charlottesville | United States | Unknown status |
| Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| Cincinnati Children's Hospital Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Nationwide Children's Hospital Coordinator of this site? Confirm its status | Columbus | United States | Unknown status |
| Neurology Rare Disease Center Coordinator of this site? Confirm its status | Denton | United States | Unknown status |
| Duke University Medicine Coordinator of this site? Confirm its status | Durham | United States | Unknown status |
| University of Kansas Medical Center Coordinator of this site? Confirm its status | Fairway | United States | Unknown status |
| Cook Children's Hospital Coordinator of this site? Confirm its status | Fort Worth | United States | Unknown status |
| UF Health Shands Hospital Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| BSHS Office of Research Coordinator of this site? Confirm its status | Grand Rapids | United States | Unknown status |
| Penn State College of Medicine Coordinator of this site? Confirm its status | Hershey | United States | Unknown status |
| Indiana University -Riley Research Coordinator of this site? Confirm its status | Indianapolis | United States | Unknown status |
| University of Iowa Coordinator of this site? Confirm its status | Iowa City | United States | Unknown status |
| UCSD & Rady Children's Coordinator of this site? Confirm its status | La Jolla | United States | Unknown status |
| Children's Hospital of Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Medical College of Wisconsin Coordinator of this site? Confirm its status | Milwaukee | United States | Unknown status |
| Vanderbilt University Medical Center Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Columbia University Medical Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Children's Hospital of The King's Daughters Coordinator of this site? Confirm its status | Norfolk | United States | Unknown status |
| CHOP Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Phoenix Children's Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| UPMC Children's Hospital of Pittsburgh Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| UCSF Benioff Children's Hospital, Medical Center Coordinator of this site? Confirm its status | San Francisco | United States | Unknown status |
| Washington University in St. Louis Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
| Stony Brook University Hospital Coordinator of this site? Confirm its status | Stony Brook | United States | Unknown status |
| MultiCare Institute of Research and Innovation Coordinator of this site? Confirm its status | Tacoma | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.