Neuromuscular Diseases Clinical Trial
— RESILIENTOfficial 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
Verified date | November 2023 |
Source | Biohaven Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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 or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.
Status | Active, not recruiting |
Enrollment | 269 |
Est. completion date | January 2025 |
Est. primary completion date | January 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 4 Years to 21 Years |
Eligibility | Key Inclusion Criteria: - 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, risdiplam, and/or a history of onasemnogene abeparvovec Key Exclusion Criteria: - Cannot have previously taken anti-myostatin therapies - Must weigh at least 15kg - 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 - Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter |
Country | Name | City | State |
---|---|---|---|
Belgium | University Hospital Antwerp | Edegem | |
Belgium | University Hospital Ghent | Ghent | |
Belgium | University Hospital Leuven | Leuven | |
Czechia | University Hospital Brno - Dept. of Pediatric Neurology | Brno | |
Czechia | Motol University Hospital | Prague | |
Germany | University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I | Essen | |
Germany | University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders | Freiburg | |
Germany | Dr. Von Haunersches Children'S Hospital - Lmu Munich | Munich | |
Italy | Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital | Bologna | |
Italy | Nemo-Brescia Clinical Center For Neuromuscular Diseases | Gussago | |
Italy | IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT | Pavia | |
Italy | Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience | Roma | |
Netherlands | University Medical Center Utrecht | Utrecht | |
Poland | University Clinical Centre in Gdansk - Dept. of Developmental Neurology | Gdansk | |
Poland | Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic | Poznan | |
Poland | The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology | Warsaw | |
Poland | T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept. | Wroclaw | |
Spain | Donostia University Hospital | Donostia | |
Spain | Hospital Sant Joan de Déu | Esplugues De Llobregat | |
Spain | Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit | Málaga | |
Spain | La Fe University and Polytechnic Hospital | Valencia | |
United Kingdom | Royal Hospital For Children | Glasgow | Scotland |
United Kingdom | John Radcliffe Hospital | Oxford | |
United States | Rare Disease Research | Atlanta | Georgia |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | UT Pediatric Neurosciences/Dell Children's Medical Center | Austin | Texas |
United States | Boston Children's Hospital - Harvard | Boston | Massachusetts |
United States | University of Virginia Children's Hospital | Charlottesville | Virginia |
United States | Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Nationwide Children's Hospital | Columbus | Ohio |
United States | Neurology Rare Disease Center | Denton | Texas |
United States | Duke University Medicine | Durham | North Carolina |
United States | University of Kansas Medical Center | Fairway | Kansas |
United States | Cook Children's Hospital | Fort Worth | Texas |
United States | UF Health, Shands Hospital | Gainesville | Florida |
United States | BSHS Office of Research | Grand Rapids | Michigan |
United States | Penn State College of Medicine | Hershey | Pennsylvania |
United States | Indiana University -Riley Research | Indianapolis | Indiana |
United States | University of Iowa | Iowa City | Iowa |
United States | UCSD & Rady Children's | La Jolla | California |
United States | Children's Hospital of Los Angeles | Los Angeles | California |
United States | Medical College of Wisconsin | Milwaukee | Wisconsin |
United States | Vanderbilt University Medical Center | Nashville | Tennessee |
United States | Columbia University Medical Center | New York | New York |
United States | Children's Hospital of The King's Daughters | Norfolk | Virginia |
United States | CHOP Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Phoenix Children's | Phoenix | Arizona |
United States | UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Washington University in St. Louis | Saint Louis | Missouri |
United States | UCSF Benioff Children's Hospital, Medical Center | San Francisco | California |
United States | Stony Brook University Hospital | Stony Brook | New York |
United States | MultiCare Institute of Research and Innovation | Tacoma | Washington |
Lead Sponsor | Collaborator |
---|---|
Biohaven Pharmaceuticals, Inc. |
United States, Belgium, Czechia, Germany, Italy, Netherlands, Poland, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score | Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The higher the score, indicates higher functioning. | Baseline to Week 48 |
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