Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
Status:
Completed
Trial end date:
2009-05-01
Target enrollment:
Participant gender:
Summary
This study will examine whether Fx-1006A is effective in halting the progression of Familial
Amyloid Polyneuropathy (FAP).
Deposition of TTR amyloid is associated with a variety of human diseases. Deposition of
amyloid fibrils of variant TTR (primarily V30M) in peripheral nerve tissue produces the
condition called FAP.
The prevention of the formation of amyloid by stabilization of the TTR native state should
constitute an effective therapy for amyloid diseases. Therapeutic intervention with a TTR
stabilizer drug, such as Fx-1006A, is hypothesized to stop progression of the disease in FAP
patients. FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless
neurological deterioration FAP patients experience.
This Phase 2/3 study will enroll early to mid-stage FAP patients in order to interrupt and
stabilize the disease at a point in time where progression of motor and autonomic dysfunction
can be maximally effected. Male and female patients with FAP with documented V30M TTR
mutation will receive Fx-1006A or placebo once daily for a period of eighteen (18) months.