modifier, who are able to sit but have no historical or current ability to stand or walk. One patient in this clinical trial has been dosed to date.
dosage strengths will be evaluated and patients will be stratified into two age groups: patients less than 24 months and patients at least 24 months but less than
60 months. There will be at least a four-week interval between the dosing of the first three patients for each dose being studied and, based on the available safety data, a decision will be
made whether to proceed.
- Patients in Cohort 1 will receive a dose of 6.0 × 1013 vg of AVXS-101, or Dose A. Cohort 1 will consist
of three patients who are less than 24 months of age.
- If safety is established according to the Data Safety Monitoring Board, or the DSMB, the trial will proceed to Cohort 2.
- Patients in Cohort 2 will receive a dose of 1.2 X 1014 vg of AVXS-101, or Dose B. Cohort 2 will consist of three patients who are
less than 60 months of age.
- If safety is established according to the DSMB, an additional 21 patients will be enrolled until the trial includes a total of
(i) 12 patients less than 24 months of age and (ii) 12 patients at least 24 months, but less than 60 months, of age who have received Dose B.
to the well-characterized natural history of the disease by the Pediatric Neuromuscular Clinical Research Network, 100 percent of children with SMA Type 2 will
never walk without support, 95 percent of children will never stand without assistance and more than 30 percent will die by 25 years of age. Additionally, children with SMA
Type 2, who are between 24 and 60 months of age with 3 copies of the SMN2 gene, experienced a mean decrease of 0.33 points on the Hammersmith Function Motor Scale Expanded over a
12-month period at first evaluation.
trial is projected to be conducted at 11 sites in the United States, including Ann and Robert H. Lurie Children's Hospital of Chicago, Boston Children's Hospital, Children's
Hospital of Philadelphia, David Geffen School of Medicine at UCLA, Johns Hopkins Pediatric Neurology, Nationwide Children's Hospital, Stanford University Medical Center, University of Central Florida