Phase 1/2 Dose Escalation Study in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia
Status:
Terminated
Trial end date:
2017-11-01
Target enrollment:
Participant gender:
Summary
Recent reports have identified a specific oncogenic mutation L265P of the MYD88 gene in
approximately 90% of the patients with Waldenström's macroglobulinemia. MYD88 is a key linker
protein in the signaling pathway of Toll Like Receptors (TLRs) 7, 8, and 9, and IMO-8400 is
an oligonucleotide specifically designed to inhibit TLRs 7,8, and 9. The scientific
hypothesis for use of IMO-8400 to treat patients with Waldenström's macroglobulinemia depends
on the inhibition of mutant MYD88 signaling in the TLR pathway, thereby interrupting the
proliferation of cell populations responsible for the propagation of the disease.