Transthyretin Amyloidosis (ATTR) MedDRA version: 14.1 Level: LLT Classification code 10057949 Term: Familial amyloid polyneuropathy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Tafamidis meglumine
Product Code: Fx-1006A
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: tafamidis
CAS Number: 951395-08-7
Current Sponsor code: Fx-1006A
Other descriptive name
Sponsors
Pfizer Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient has successfully completed either Protocol Fx-006 or Fx1A-201. 2. If female, patient is post-menopausal, surgically sterilized, or willing to use two acceptable methods of birth control (i.e., 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.). 3. Patient is, in the opinion of the investigator, willing and able to comply with the investigational product regimen and all other study requirements. Sub-study: 4.Decrease in creatinine clearance to =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patient has not successfully completed either Protocol Fx-006 or Fx1A-201. 2. Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than 3 to 4 times/month. The following NSAIDs are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac. 3. If female, patient is pregnant or breast feeding. 4. Clinically significant medical condition that, in the opinion of the investigator, would place the patient at an increased risk to participate in the study. 5. An alanine transaminase (ALT) and aspartate transaminase, (AST) value >3X ULN that in the medical judgement of the investigator is due to reduced liver function or active disease. 6. The patient has received a liver or heart transplant. Sub-study: 7. Unwilling to provide written informed consent for this sub-study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin (TTR) amyloidosis (ATTR).;Secondary Objective: To continue to provide the investigational product Fx-1006A until market availability or until 31 December 2012 or whichever is earliest, to patients with ATTR who have completed Protocol Fx-006 or Protocol Fx1A-201.;Primary end point(s): Safety endpoints: - The incidence of treatment-emergent adverse events (TEAEs), physical examinations, clinical laboratory testing, use of concomitant medications and vital signs. Efficacy endpoints: - The Neuropathy Impairment Score (NIS) - The Total Quality of Life (TQOL) score as measured using the Norfolk Quality of Life for diabetic neuropathy (QOL-DN) questionnaire - The Karnofsky Performance Scale Index -Assessment of Patient Ambulation;Timepoint(s) of evaluation of this end point: End of study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): None described at time of submission;Timepoint(s) of evaluation of this end point: Not applicable | — |
Countries
Argentina, Brazil, France, Germany, Italy, Portugal, Sweden, United States
Contacts
Public ContactClinical Trials.gov Call Center
Pfizer Inc.
Outcome results
None listed