Status:
COMPLETED
The Effects Of Fx-1006A On Transthyretin Stabilization And Clinical Outcome Measures In Patients With V122I Or Wild-Type TTR Amyloid Cardiomyopathy
Lead Sponsor:
Pfizer
Conditions:
Cardiomyopathy
Eligibility:
All Genders
40+ years
Phase:
PHASE2
Brief Summary
Open-label, multicenter, international, single-treatment study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients wit...
Eligibility Criteria
Inclusion
- Patient is \> 40 years-old.
- Patient participated in FoldRx Study Fx-001 (TRACS) OR Patient has documented TTR amyloid cardiomyopathy and NYHA Classification of I or II.
- TTR amyloid cardiomyopathy is defined as:
- Variant TTR amyloid cardiomyopathy as defined as: V122I genotype and presence of amyloid in cardiac biopsy tissue (as determined by congo red stain, alcin blue stain, or immunohistochemical TTR analysis), or
- Variant TTR amyloid cardiomyopathy as defined as: V122I genotype, evidence of cardiac involvement by echocardiography with left ventricle wall thickness \> 12 mm and presence of amyloid in non-cardiac biopsy tissue (as determined by congo red stain, alcin blue stain, or immunohistochemical TTR analysis), or
- Wild-type TTR amyloid cardiomyopathy as defined as: normal TTR genotype and presence of TTR amyloid deposits in cardiac biopsy tissue (as determined by congo red stain and immunohistochemical TTR analysis), or
- Wild-type TTR amyloid cardiomyopathy as defined as: normal TTR genotype, evidence of cardiac involvement by echocardiography with left ventricle wall thickness \> 12 mm and presence of TTR amyloid deposits in non-cardiac biopsy tissue (as determined by congo red stain and immunohistochemical TTR analysis).
- Patient's symptoms of congestive heart failure (CHF) have been optimally managed prior to baseline, as assessed by the Principal Investigator. Optimal CHF management includes stable drug regimen for ≥ 4 weeks prior to enrollment and stable dose of beta blocker for ≥ 3 months prior to enrollment.
- If female, patient is post-menopausal. If male with a female partner of childbearing potential, willing to use an acceptable method of birth control for the duration of the study and 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-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than 3-4 times/month. The following NSAID are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac.
- Patient has a TTR mutation other than V122I.
- Patient has primary or secondary amyloidosis.
- Patient has received prior liver or heart transplantation.
- Patient with positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV), and/or human immunodeficiency virus (HIV).
- Patient has renal failure requiring dialysis.
- Patient has moderate or severe hepatic impairment (assessed by Child-Pugh).
- Patient has liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) \> 2 times upper limit of normal (ULN) that, in the medical judgment of the Investigator, are due to reduced liver function or active liver disease.
- Patient has prior non-amyloid cardiac disease, such as myocardial infarction due to obstructive coronary artery disease, active non-amyloid cardiomyopathy (i.e., symptomatic left ventricular dysfunction from any cause other than amyloid), or symptomatic valvular heart disease that significantly contribute to the patient's underlying cardiac signs or symptoms.
- Patient has a co-morbidity anticipated to limit survival to less than 12 months.
- Patient received an investigational drug/device in another clinical investigational study within 60 days before Baseline (Day 0).
- Patient had active alcohol or substance abuse within 60 days before Baseline (Day 0).
- Patient has a history of documented noncompliance.
Key Trial Info
Start Date :
August 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2010
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT00694161
Start Date
August 1 2008
End Date
January 1 2010
Last Update
January 11 2013
Active Locations (6)
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1
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
2
University of Chicago
Chicago, Illinois, United States, 60637
3
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21205
4
Harvard Vanguard Medical Associates
Boston, Massachusetts, United States, 02215