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Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

NCT00409175 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 128

Last updated 2012-12-17

Study results available
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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.

Conditions

  • Familial Amyloid Polyneuropathy

Interventions

DRUG

Fx-1006A

Fx-1006A 20mg or matched placebo once daily (at the same time each day) for a period of 18 Months

DRUG

Placebo

Fx-1006A 20mg or matched placebo once daily (at the same time each day) for a period of 18 Months

Sponsors & Collaborators

Principal Investigators

  • Jeff Packman · FoldRx Pharmaceuticals, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-01-31
Primary Completion
2009-05-31
Completion
2009-05-31

Countries

  • United States
  • Argentina
  • Brazil
  • France
  • Germany
  • Portugal
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00409175 on ClinicalTrials.gov