Safety and efficacy of Orally Administered Fx-1006A in Patients with Familial Amyloid Polyneuropathy (FAP): a Phase II/III, Randomised, Double-Blind, Placebo-Controlled Study

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 120

Inclusion Criteria

- Documented amyloid by biopsy
- Documented V30M TTR mutation.
- Peripheral and/or autonomic neuropathy with a Karnofsky Performance Status >50.
- Aged not less than 18 through 75 years
- Female patients must be post-menopausal, surgically sterilised, or willing to use two acceptable methods of birth control (ie. a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide) throughout the study and for 3 months from the end of the study. (A condom alone is not considered an acceptable method of birth control.) If male with a female partner of child bearing potential, willing to use two acceptable methods of birth control for the duration of the study. For both females and males, birth control must be used for at least 3 months after the last dose of study medication.
- Patient is, in the opinion of the Investigator, willing and able to comply with the study medication regimen and all other study requirements.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Chronic use of NSAIDs, defined as greater than 3-4 times/month (ibuprofen and nimesulid will be permitted)
- Patient has primary amyloidosis
- Patient is pregnant or breast feeding
- Prior liver transplantation
- No recordable sensory threshold for vibration perception in both feet, as measured by CASE IV
- Patients with positive results for HbsAg, anti-HCV, and/or HIV
- Renal insufficiency (creatnine clearance < 30 ml/min)
- Liver function test abnormalities; alanine transaminase (ALT) and/or aspartate transaminase (AST) > 2 times upper limit of normal that in the medical judgement of the investigator are due to reduced liver function or active liver disease.
- NYHA Class > 3
- Other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse and chronic inflammatory diseases)
- Comorbidity anticipated to limit survival to less than 18 months
- Patient has received an investigational drug/device and/or participated in another clinical investigation within 60 days before baseline (Day 0).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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