Status:
COMPLETED
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
Lead Sponsor:
Pfizer
Conditions:
Familial Amyloid Polyneuropathy
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
PHASE3
Brief 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. De...
Detailed Description
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. T...
Eligibility Criteria
Inclusion
- Amyloid documented by biopsy.
- Documented V30M TTR mutation.
- Peripheral and/or autonomic neuropathy with a Karnofsky Performance Status ≥50.
- Patient is 18-75 years old.
- If female, patient is post-menopausal, surgically sterilized, or willing to use an acceptable method of birth control. If male with a female partner of childbearing potential, willing to use an acceptable method 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.
Exclusion
- Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs).
- Primary amyloidosis.
- If female, patient is pregnant or breast feeding.
- Prior liver transplantation.
- No recordable sensory threshold for vibration perception in both feet, as measured by CASE IV.
- Positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (HCV), and/or human immunodeficiency virus (HIV).
- Renal insufficiency or liver function test abnormalities.
- New York Heart Association (NYHA) Functional Classification ≥III.
- Other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic inflammatory diseases).
- Co-morbidity anticipated to limit survival to less than 18 months.
- Patient received an investigational drug/device and/or participated in another clinical investigational study within 60 days before Baseline.
Key Trial Info
Start Date :
January 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2009
Estimated Enrollment :
128 Patients enrolled
Trial Details
Trial ID
NCT00409175
Start Date
January 1 2007
End Date
May 1 2009
Last Update
December 17 2012
Active Locations (10)
Enter a location and click search to find clinical trials sorted by distance.
1
MGH Neuropathy Laboratory
Boston, Massachusetts, United States, 02114
2
FLENI-Hepatology and Organ Transplant Dept.
Ciudad de Buenos Aires, Buenos Aires, Argentina, C1428AQK
3
Hospital Universitário Prof. Clementino Fraga Filho-UFRJ
Rio de Janeiro, Southeast, Brazil, Cep 21945-560
4
CHU de Bicetre
Le Kremlin-Bicêtre, Île-de-France Region, France, 94275