RESILIENT
The purpose of this study is to see if the investigational medication helps increase muscle strength and function in children and adults from 4 – 21 years of age with SMA, as compared to placebo.
Length of Study
# Study Visits
Post-study Follow Up
Who’s Included?
Children; Adults; All genders
4–21
Drug & Formulation
Taldegrobep Alpha
Injection
Possibility of Placebo
Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease that robs children and adults of physical strength. SMA attacks and destroys motor nerve cells, taking away the ability to walk, eat and even breathe. Onset typically occurs in childhood. It impacts four out of every 100,000 people and is the number one genetic cause of death for infants.
- Have SMA confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
- Be ambulant or non-ambulant
- Be 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
- Have previously taken anti-myostatin therapies
- Have a body weight of less than 15kg
- Have 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)
- Have history of spinal fusion within 6 months of screening (MAGEC rod nonsurgical adjustments are allowed during the study)
- Have an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
Learn more about the benefits of joining our trial and see if you qualify today!
Frequently Asked Questions
About 180 people will participate.
Up to 6 weeks screening and 28 weeks of study drug treatment