A Phase 3 Multicenter, Open Label Study to Evaluate the Safety of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826-83) 20 mg or 80 mg [or Tafamidis (PF-06291826-00) 61mg] in Subjects Diagnosed with Transthyretin Cardiomyopathy (ATTR-CM))
- Conditions
- chronic heart failuretransthyretin amyloid cardiomyopathy (ATTR-CM)10019280
- Registration Number
- NL-OMON53130
- Lead Sponsor
- Pfizer
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 12
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.
Aanvullend voor cohort B:
5. Documentatie van de genetische tests voor transthyretineamyloïdose
6. Documentatie van diagnose en gebruikte criteria of congestief hartfalen en
de aanwezigheid van
amyloïde afzettingen in biopsieweefsel, b.v. vetaspiratie, speekselklier,
nervus medianus
bindweefselschede of cardiaal
7.Documentatie dat primaire (lichte keten) amyloïdose ziekte is geëvalueerd en
uitgesloten
8. Bewijs van NYHA-classificatie I, II, III of IV
1. Chronic use of diflunisal, TTR stabilizer, tauroursodeoxycholate,
doxycycline, digitalis, patisaran, calcium channel blockers, investigational
drug(s) or other experimental interventions, other than tafamidis,
independently or as part of a study within 30 months prior to enrollment, or
inotersen within 6 months prior to enrolment
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety as measured by:<br /><br>* All cause mortality.<br /><br>* Incidence of treatment emergent adverse events.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Other Endpoints<br /><br>* Cardiovascular related mortality.<br /><br>* Frequency of all cause hospitalization.<br /><br>* Frequency of cardiovascular related hospitalization (including heart failure,<br /><br>arrhythmia, myocardial infarction, stroke and other cardiovascular related<br /><br>events).<br /><br>* Change from baseline at each visit in Kansas City Cardiomyopathy<br /><br>Questionnaire Overall Score and domain scores (Physical limitation, Symptom<br /><br>stability, Symptoms, Self efficacy, Social limitation, and Quality of life) and<br /><br>domain summary scores (Functional summary and Clinical summary).<br /><br>* New York Heart Association classification at each visit.<br /><br>* Change from baseline in Body Mass Index/modified Body Mass Index at each<br /><br>visit.<br /><br>* Assessment of physical examinations, use of concomitant medications,<br /><br>electrocardiograms (ECGs), clinical laboratory testing, vital signs at each<br /><br>visit.</p><br>