2 results
Approved WMOPending
Primary ObjectivesSafety run-in Part 1:To confirm the recommended dose of alpelisib in combination with trastuzumab and pertuzumab for Part 2.Double-blind, randomized, placebo-controlled Part 2:To determine whether treatment with alpelisib in…
Approved WMORecruiting
The primary aim of the trial is to demonstrate the non-inferiority of AMT-061 (2 × 1013 gc/kg) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (AMT-061) follow-up compared to standard of…