Median duration of response
was not reached at the time of the analysis, with probability of no progression or death above 80% at the 20-month mark, according to Dr.
These indications are approved under accelerated approval based on tumor response rate and duration of response
. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Out of 394 patients in the trial, 188 were responders to medication and 154 we re-evaluated after one, two and three years (following initial treatment) for the duration of response
to medication and the possible occurrence of relapse.
The duration of response
for 2 of the patients was 9 and 12 months; 3 patients are continuing to respond at an earlier stage.
Among these long-term responders, the median duration of response
was 28.1 months.
Longer follow-up is needed to assess the duration of response
. As part of an accelerated approval program, Novartis is required to conduct further studies to determine whether the treatment results in improved survival and other clinical benefits.
Others experience a reduction in relief from spasm and a shorter duration of response
This is a remarkable magnitude and duration of response
for a heavily pretreated population.
Primary efficacy endpoints consist of complete response rate and duration of response
for patients in Cohort 1.
The primary efficacy endpoint will be progression-free survival, and secondary efficacy endpoints will include overall survival, disease control rate, overall response rate and duration of response
. The confirmatory study will involve a safety run-in that is likely to start in the second half of 2019.
Secondary endpoints include safety, duration of response
, and progression-free survival.