Study: A phase 3, multicenter, randomized (2:1), double-blind, sham procedure–controlled trial
Treatment duration: 15 months
Participants: 126 patients with later-onset SMA aged 2 to 9 years at screening
Primary endpoint: Change in motor function as measured by HFMSE
Secondary endpoint: Clinically meaningful change in HFMSE ≥3 points and change in upper limb function as measured by RULM
Study limitations: Differences in dosing compared to the approved SPINRAZA schedule
Safety: The most common side effects were fever (43%), headache (29%), vomiting (29%), and back pain (25%)
Study: A phase 1b/2a, open-label, multiple-dose, dose-escalation integrated analysis of CS2 and CS12 trials
Treatment duration: ~3 years (38 months)
Participants: 28 ambulatory and nonambulatory patients with SMA aged 2 to 16 years at time of first dose
Primary endpoint: Safety of SPINRAZA
Study limitations: Differences in dosing compared to the approved SPINRAZA schedule and no control group
Safety: Adverse reactions were similar to those reported in the pivotal trials
Study design: An independent, prospective, multicenter, observational cohort study
Study duration: Up to 14 months. Assessments made at 6, 10, and 14 months
Participants: 139 patients with genetically confirmed 5q later-onset SMA ages 16-65
Primary endpoint: Change from baseline in motor function measured by HFMSE at 6, 10, and 14 months. Patients with missing baseline HFMSE scores were excluded from these analyses
Secondary endpoints: Change from baseline in upper limb and walking ability measured by RULM and 6MWT at 6, 10, and 14 months
Study limitations: No control group; observational design. Study powered on primary endpoint only. Statistics for other endpoints are descriptive only
Safety: The majority of adverse events (AEs) were generally consistent with those reported in the SPINRAZA clinical trials. Other reported AEs were:
Study design: An independent, retrospective, multicenter, observational cohort study
Study duration: Up to 14 months. Assessments made at 6, 10, and 14 months
Participants: 116 patients with later-onset SMA Type 2 (n=13) and Type 3 (n=103) aged 18-72
Primary outcomes: Change from baseline in motor function measured by HFMSE, RULM, and 6MWT at 6, 10 and 14 months
Study limitations: No control group, retrospective observational design, missing data for some clinical assessment variables, and a small number of patients with SMA Type 2 (n=13)
Safety: The majority of adverse events (AEs) were generally consistent with those in the SPINRAZA clinial trials
Not Biogen-sponsored studies. SPINRAZA pivotal trials did not include adults with SMA.