ACTIMMUNE INTERFERON GAMMA COPAY ASSISTANCE PROGRAM TRIAL
The study was completed on time, and the analysis conducted efficiently.Īlthough the Phase 3 trial was unsuccessful, both Horizon and FARA view their partnership's ability to bring to life CTTI's PGE recommendations as a model for other organizations to emulate. In addition, there were no screening failures, no amendments across the duration of the study (virtually unheard of in clinical research), and no adjustments for eligibility criteria. Enrollment was gated, so no site was overwhelmed. The study had a compressed timeline, which Horizon largely credits to FARA's patient registry and community support. Horizon and FARA successfully developed and executed the Phase 3 placebo-controlled ACTIMMUNE trial, which enrolled 92 patients at four clinical sites, representing FARA's largest study involvement to date. FARA also helped Horizon with subject selection given the therapeutic approach for ACTIMMUNE, as well as selection of clinical trial sites in FARA's Collaborative Clinical Research Network for FA. This included scoping discussions on FARA's natural history database (a 15-year endeavor, which FARA considers to be its most valuable resource), mining for biomarkers, and collaboration with patients into the toleration of procedures in the protocol design to increase the shots on goal for ACTIMMUNE. The FA community also offered significant input to the ACTIMMMUNE clinical team in developing the protocol for the Phase 3 study. Horizon and FARA collaborated on disease characterization, access to clinical experts, and validated outcome measures for clinical planning. The partners met with key opinion leaders and leveraged FARA's patient ambassador program to get the FA community more engaged.
Per CTTI's recommendations for research sponsors and patient groups, Horizon brought FARA's patient insights into for the ACTIMMUNE protocol development process early on.
0 kommentar(er)
