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Researching an Investigational Treatment Option for Spinal Muscular Atrophy (SMA).

 

What is SMA?

SMA is a genetic disease that affects nerve cells in the spinal cord and brain stem, producing muscle weakness and atrophy (wasting/shrinking) that affects essential voluntary muscle activity such as breathing, swallowing, speaking, and walking.  

Doctors are working with Biohaven Pharmaceuticals, in conducting this research study, or clinical trial, to test a potential new investigational treatment option for SMA. 

Despite current therapies, a high unmet need for safe and effective treatments for SMA remains, as many patients still experience significant weakness and reduced levels of functioning.

boy in wheelchair next to a doctor and nurse

We named the study “                                       ” because we are in absolute awe of the resilience, perseverance, and never-give-up attitude of children and adults with SMA, as well as their parents, guardians, and caregivers.

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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. A placebo is an inactive material that looks like the study drug but does not contain any active study drug.

This study includes participants who are able to sit independently as well as those who are able to walk.

The study medication, taldefgrobep alfa, or a placebo, will be given while you or your child is taking your regular medication for SMA (nusinersen, risdiplam, or previous gene therapy onasemnogene abeparvovec-xioi). The study is testing the effectiveness and safety of this medication, taldefgrobep alfa, as an additional treatment. You or your child will have a 66% chance (2:1) to receive the study medication. 

Approximately 180 participants with SMA are expected to enter the treatment phase of this study.

If you or your child have SMA, and are currently taking nusinersen, risdiplam, and/or have received onasemnogene abeparvovec-xioi, you or your child may be eligible for the Resilient study.

​BHV2000-301: 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 (RESILIENT)

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What to Expect

If you or your child qualifies and are enrolled in the study, you will receive the following at no cost:

  • The investigational medication, or a placebo

  • All study-related care, testing, and monitoring

  • Ongoing appointments with the study doctors and qualified medical providers specializing in SMA care

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You or your child will continue to see your regular doctors for routine office visits and checkups.

After completion of the 48-week initial study phase, you or your child may be eligible to participate in an open-label extension study, where all participants will receive taldefgrobep alfa, and study-related care, at no cost.