Transthyretin amyloid cardiomyopathy (TTR-CM) MedDRA version: 20.0 Level: LLT Classification code 10002020 Term: Amyloid cardiomyopathy System Organ Class: 100000004849
Conditions
Interventions
Trade Name: Vyndaqel 20 mg soft capsule
Product Name: Tafamidis meglumine
Product Code: PF-06291826-83
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Tafamidis meglumine
CAS Number: 951395-08
Sponsors
Pfizer Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female subjects with TTR amyloid cardiomyopathy who have completed 30 months of study treatment on Protocol B3461028. 2. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study. 3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. 4. Male subjects able to father children and female subjects of childbearing potential and at risk for pregnancy must agree to use 2 highly effective methods of contraception hroughout the study and for at least 28 days after the last dose of assigned treatment. Female subjects who are not of childbearing potential (ie, meet at least 1 of the following criteria): ? Have undergone a documented hysterectomy and/or bilateral oophorectomy; ? Have medically confirmed ovarian failure; or ? Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed by having a serum folliclestimulating hormone (FSH) level confirming the post-menopausal state. All other female subjects (including females with tubal ligations) will be considered o be of childbearing potential. 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 30 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 300
Exclusion criteria
Exclusion criteria: 1. Chronic use of diflunisal, TTR stabilizer, tauroursodeoxycholate, doxycycline, digitalis, patisiran, calcium channel blockers, investigational drug(s) or other experimental interventions, other than tafamidis, independently or as part of a study within 30 days prior to enrollment enrollment or inotersen within 6 months prior to enrollment. 2. Use of certain non-steroidal anti-inflammatory drugs (NSAIDs) 3. Liver and/or heart transplant, or implanted cardiac mechanical assist device. 4. Pregnant females (or planning to become pregnant during the study interval); breastfeeding females; male subjects with partners currently pregnant. 5. Require initiation of treatment with calcium channel blockers. 6. Urinary retention requiring chronic self-catheterization. 7. Breach of compliance with treatment/significant protocol violations during conduct of B3461028 for which the subject was accountable. 8. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees directly involved in the conduct of he study. 9. Other severe acute or chronic medical or psychiatric condition or laboratory bnormality that may increase the risk associated with study participation or investigational product administration, or that may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject an appropriate for entry into this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: ? To obtain additional, long-term, safety data for tafamidis in subjects with transthyretin amyloid cardiomyopathy (TTR-CM). ? To provide investigational product, tafamidis, to TTR-CM subjects who complete 30 months of blinded treatment on protocol B3461028.;Secondary Objective: Not Applicable;Primary end point(s): Safety as measured by: ? All-cause mortality. ? Incidence of treatment-emergent adverse events.;Timepoint(s) of evaluation of this end point: Both primary end points will be assessed throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Cardiovascular-related mortality. Frequency of all-cause hospitalization. Frequency of cardiovascular-related hospitalization (including heart failure, arrhythmia, myocardial infarction, stroke and other cardiovascular-related events). Change from baseline at each visit in Kansas City Cardiomyopathy Questionnaire. Overall Score and domain scores (Physical limitation, Symptom stability, Symptoms, Self-efficacy, Social limitation, and Quality of life) and domain summary scores (Functional summary and Clinical summary). ? New York Heart Association classification at each visit. Change from baseline in Body Mass Index/modified Body Mass Index at each visit. Assessment of physical examinations, use of concomitant medications, electrocardiograms (ECGs), clinical laboratory testing, vital signs at each visit.;Timepoint(s) of evaluation of this end point: - 12-lead ECG: An annual basis (every 12 months) at clinic visits Months 12, 24, 36, 48 and 6at 0 month or early study termination - NYHA Classification: At day 0, evry 6 months, at 60 month or early study termination - KCCQ: At day 0, evry 6 months, at 60 month or early study termination | — |
Countries
Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactClinicaltrials.gov Call Center
Pfizer Inc
Outcome results
None listed