Biogen recently presented data about ongoing studies of SPINRAZA (nusinersen) in patients with Spinal Muscular Atrophy. Updates included the NURTURE, ENDEAR, SHINE, DEVOTE, and RESPOND clinical trials.
Oral presentation details:
- Title: Predictive Factors of Nusinersen Treatment Response in Infantile-onset SMA: Results from the ENDEAR/SHINE Studies
- Presenter: Laurent Servais, MD, PhD
- Title: Nusinersen in Children with SMA Who Received Onasemnogene Abeparvovec: Design of the Phase 4, Open-Label RESPOND Study
- Presenter: Julie Parsons, MD
Poster presentation details:
- Title: Nusinersen in children with SMA who received onasemnogene abeparvovec: design of the phase 4, open-label RESPOND study
- Presenter: Julie Parsons, MD
- Title: Title: Nusinersen in children with SMA who received onasemnogene abeparvovec: design of the phase 4, open-label RESPOND study
- Presenter: Samuel Ignacio Pascual Pascual, PhD
- Title: Predictive Factors of Nusinersen Treatment Response in Infantile-onset SMA: Results from the ENDEAR/SHINE Studies
- Presenter: Laurent Servais, MD, PhD
- Title: Preserved swallowing function in infants who initiated nusinersen treatment in the presymptomatic stage of SMA: results from the NURTURE study
- Presenter: Kathryn J. Swoboda, MD
- Title: Ongoing Phase 2/3 DEVOTE (232SM203) Randomized, Controlled Study to Explore High-Dose Nusinersenin SMA: Part A Interim Results and Part B Enrollment Update
- Presenter: Samuel Ignacio Pascual Pascual, PhD
About the Trials
NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before 6 weeks old.
ENDEAR is a randomized, double-blind, sham-procedure controlled 13-month study in individuals with infantile-onset spinal muscular atrophy (SMA). There were 122 participants.
SHINE is an open-label extension study for persons who previously participated in nusinersen investigational studies. There are 292 participants.
DEVOTE is a Phase 2/3 randomized, controlled dose-escalating study that will be conducted at 50 sites around the world with a projected enrollment of 126 individuals with SMA of all ages, including adults. DEVOTE will examine the potential for even greater efficacy, as well as the safety and tolerability of SPINRAZA, when administered at a higher dose. Recruitment is ongoing.
RESPOND is a two-year, open-label study to evaluate the efficacy and safety of SPINRAZA in SMA patients previously treated with Zolgensma to further optimize treatment decisions. Recruitment is ongoing.