Status:
COMPLETED
The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis
Lead Sponsor:
Pfizer
Conditions:
Transthyretin-associated Amyloidosis With Polyneuropathy
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
This is an open-label, multicenter, international study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients with non-V...
Eligibility Criteria
Inclusion
- Patient has amyloid documented by biopsy (in accordance with institutional site standard of care).
- Patient has documentation of one of the following targeted TTR mutations: Ser77Tyr, Thr60Ala, Tyr114Cys, Leu58His, Glu89Gln, Ser77Phe, Thr49Ala, Ile107Val, Val30Ala, Gly47Ala, Gly47Glu, Leu55Arg, Lys70Asn, Ile84Thr, Ile107Met. Patients with mutations other than those listed may be enrolled only after approval by the Sponsor.
- Patient has peripheral and/or autonomic neuropathy and/or cardiomyopathy with a Karnofsky Performance Status ≥ 50.
- Patient is aged ≥18 to 75 years, inclusive.
- If female, patient is post-menopausal, surgically sterilized, or willing to use two acceptable methods of birth control (i.e., 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 childbearing potential, willing to use two acceptable methods of birth control for the duration of the study. For both females and males, acceptable 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), defined as greater than 3-4 times/month (ibuprofen and nimesulide will be permitted).
- Patient has primary or secondary amyloidosis.
- Patient has TTR-associated amyloidosis with V30M mutation.
- If female, patient is pregnant or breast feeding.
- Patient has received prior liver transplantation.
- Patient is expected to undergo liver transplantation within 12 months after enrollment.
- Patient with positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (HCV), and/or human immunodeficiency virus (HIV).
- Patient has renal insufficiency (creatinine clearance \< 30 ml/min).
- 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 a New York Heart Association (NYHA) Functional Classification ≥ III.
- Patient has other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic inflammatory diseases).
- Patient has prior non-amyloid cardiac disease such as: myocardial infarction due to obstructive coronary artery disease, active non-amyloid cardiomyopathy (e.g., symptomatic left ventricular dysfunction from any cause other than amyloid, patients with a primary diagnosis of symptomatic valvular heart disease)
- Patient has a co-morbidity anticipated to limit survival to less than 12 months.
- Patient has received an investigational drug/device and/or participated in another clinical investigational study within 60 days before Baseline (Day 0).
Key Trial Info
Start Date :
June 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2010
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT00630864
Start Date
June 1 2008
End Date
January 1 2010
Last Update
January 18 2013
Active Locations (4)
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1
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21205
2
CHU de Bicetre
Paris, France
3
Universitatsklinikum Munster, Transplant Hepatology
Münster, Germany
4
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche
Irccs - Policlinico San Matteo, Pavia, Italy, 19 - 27100