Study: A phase 2, open-label, multicenter, multinational, single-arm study
Treatment duration: Ongoing, results from a median of 2.9-year interim analysis
Participants: 25 presymptomatic, genetically diagnosed infants who were likely to develop Type 1 or 2 SMA aged ≤6 weeks at time of first dose
Primary endpoint: Time to death or respiratory intervention
Secondary endpoints: Attainment of WHO motor milestones and change from baseline in the CHOP INTEND motor function scale
Study limitations: Small participant group size and no sham control group; CHOP INTEND score was assessed only until a participant achieved the maximum score of 64. HINE-2 score was assessed until day 778, but not at later study visits
Safety: Consistent with the SPINRAZA prescribing information; no new safety concerns were identified. 80% (20/25) of participants had an adverse event (AE) that was mild or moderate in severity. Eight participants experienced AEs that were possibly or definitely related to the lumbar puncture procedure.
All the following are results after a
median follow-up of 2.9 years in the
The majority of infants achieved the following WHO motor milestones:
Mean CHOP INTEND scores showed SPINRAZA improved motor function, with scores rising steadily until
day 183. The majority of patients achieved a maximum score of 64 and then remained stable over time.
CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders.
**In the original protocol, CHOP INTEND was to be assessed in participants at each visit up to day 778; however, this was amended to be until they had a maximum score of 64. Once a score of 64 was achieved, CHOP INTEND was no longer assessed.
“We are very, very thankful for
the advances in science.”