Research Project
7365316
[unreadable] DESCRIPTION (provided by applicant): [unreadable] The long-term objective of the development program for Fx-1006A is to provide a treatment for FAP, a relentlessly progressive, debilitating and ultimately fatal orphan disease. The proposed study is intended to provide the primary data to support a marketing application for Fx-1006A in the treatment of FAP. [unreadable] [unreadable] Deposition of amyloid fibrils of variant transthyretin (TTR) (primarily V30M) in peripheral nerve tissue produces the condition called FAP. Mutation in TTR destabilizes the protein and causes amyloid deposition. The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for FAP. Therapeutic intervention with a TTR stabilizer drug, such as Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience. [unreadable] [unreadable] The international Phase 2/3 study will enroll 120 early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of sensorimotor 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. [unreadable] [unreadable] The primary objectives of the study are (1) to evaluate the effect of Fx-1006A on disease progression in patients with FAP; and (2) to evaluate the safety and tolerability of chronic administration of Fx1006A in patients with FAP. The secondary study objectives are: (1) to determine the pharmacodynamic stabilization effect of Fx-1006A on human V30M TTR; and (2) to determine the population pharmacokinetics in patients with FAP. [unreadable] [unreadable] The co-primary efficacy endpoints for the study are: [unreadable] Response to treatment at Month 18, as indicated by either improvement (decrease from baseline) or stabilization (change from baseline of 0 to <2) in the Neurologic Impairment Score-Lower Limb (NIS-LL) score. [unreadable] Change from Baseline to 18 months in the Total Quality of Life (TQOL) score, as measured by the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN). [unreadable] [unreadable] Efficacy, safety and pharmacokinetic assessments will be conducted throughout the study. [unreadable] [unreadable] [unreadable]
