Clinical Trials List
Protocol NumberB3461045
NCT Number(ClinicalTrials.gov Identfier)NCT02791230
2019-02-27 - 2023-12-31
Phase III
Recruiting2
ICD-10E85.0
Non-neuropathic heredofamilial amyloidosis
ICD-10E85.1
Neuropathic heredofamilial amyloidosis
ICD-10E85.2
Heredofamilial amyloidosis, unspecified
ICD-10E85.3
Secondary systemic amyloidosis
ICD-10E85.4
Organ-limited amyloidosis
ICD-10E85.8
Other amyloidosis
ICD-10E85.9
Amyloidosis, unspecified
ICD-9277.3
Amyloidosis
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) 61 MG] IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (ATTR-CM)
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Trial Applicant
PPD DEVELOPMENT (HK) LIMITED
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Sponsor
Pfizer
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- Shih-Hsien Sung Division of Cardiovascular Diseases
- Kon-Ping Lin Division of Cardiovascular Diseases
- 廖若男 Division of Cardiovascular Diseases
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- JYH-MING JIMMY JUANG Division of General Internal Medicine
- Chi-Chao Chao Division of Neurology
- Ming-Jen Lee Division of Neurology
- SUNG-TSANG HSIEH Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Transthyretin (TTR) Amyloid Cardiomyopathy (ATTR-CM)
Objectives
Primary Objectives:
To obtain additional, long-term, safety data for tafamidis in subjects with transthyretin amyloid cardiomyopathy (ATTR-CM).
To provide investigational product, tafamidis, to enrolled subjects until local availability by prescription for the ATTR-CM indication
Test Drug
Tafamidis (PF-06291826)
Active Ingredient
Tafamidis (PF-06291826)
Dosage Form
capsule
Dosage
61mg/20mg
Endpoints
Primary Endpoints
Safety as measured by:
All-cause mortality.
Incidence of treatment-emergent adverse events.
Other Endpoints
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 Summary score, domain scores (Physical limitation, Symptom stability,
Symptom frequency, Symptom burden, Total symptom, Self-efficacy, Social
limitation, and Quality of life), and Clinical summary score.
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.
Safety as measured by:
All-cause mortality.
Incidence of treatment-emergent adverse events.
Other Endpoints
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 Summary score, domain scores (Physical limitation, Symptom stability,
Symptom frequency, Symptom burden, Total symptom, Self-efficacy, Social
limitation, and Quality of life), and Clinical summary score.
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.
Inclution Criteria
1. Male or female subject of at least 18 years of age (or the minimum country specific
age of consent if >18) and participates at a study site in an eligible country listed in
Protocol Appendix 5.
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. A female subject is eligible to participate if she is not pregnant or breastfeeding, and
at least 1 of the following conditions applies:
Is not a Woman of Childbearing Potential (WOCBP) (see definition
Section 4.3.1).
OR
Is a WOCBP and using a contraceptive method that is highly effective (with a
failure rate of <1% per year) (see Section 4.3) during the intervention period and
for at least 28 days after the last dose of study intervention, which corresponds to
the time needed to eliminate any study intervention. The investigator should
evaluate the effectiveness of the contraceptive method in relationship to the first
dose of study intervention.
The investigator is responsible for review of medical history, menstrual history, and
recent sexual activity to decrease the risk for inclusion of a woman with an early
undetected pregnancy.
5. Documentation of the genetic testing for transthyretin amyloidosis (ie, original
laboratory result, or copy).
6. Documentation of diagnosis and criteria used (eg, congestive heart failure and
scintigraphy with tracer eg, 99mTC DPD [99mTC 3,3 diphosphono 1,2 propano
dicarboxylic acid], 99mTC PYP [Pyrophosphate] and also 99mTC HMDP
[hydroxymethylene diphosphonate] or Congestive heart failure and presence of
amyloid deposits in biopsy tissue, eg, fat aspirate, salivary gland, median nerve
connective tissue sheath, or cardiac [amyloid demonstrated per appropriate stain
such as Congo red or alcian blue stain]).
7. Documentation that primary (light chain) amyloidosis disease has been evaluated and
ruled out (ie, original laboratory result, or copy).
8. Evidence of NYHA classification I, II, III, or IV.
age of consent if >18) and participates at a study site in an eligible country listed in
Protocol Appendix 5.
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. A female subject is eligible to participate if she is not pregnant or breastfeeding, and
at least 1 of the following conditions applies:
Is not a Woman of Childbearing Potential (WOCBP) (see definition
Section 4.3.1).
OR
Is a WOCBP and using a contraceptive method that is highly effective (with a
failure rate of <1% per year) (see Section 4.3) during the intervention period and
for at least 28 days after the last dose of study intervention, which corresponds to
the time needed to eliminate any study intervention. The investigator should
evaluate the effectiveness of the contraceptive method in relationship to the first
dose of study intervention.
The investigator is responsible for review of medical history, menstrual history, and
recent sexual activity to decrease the risk for inclusion of a woman with an early
undetected pregnancy.
5. Documentation of the genetic testing for transthyretin amyloidosis (ie, original
laboratory result, or copy).
6. Documentation of diagnosis and criteria used (eg, congestive heart failure and
scintigraphy with tracer eg, 99mTC DPD [99mTC 3,3 diphosphono 1,2 propano
dicarboxylic acid], 99mTC PYP [Pyrophosphate] and also 99mTC HMDP
[hydroxymethylene diphosphonate] or Congestive heart failure and presence of
amyloid deposits in biopsy tissue, eg, fat aspirate, salivary gland, median nerve
connective tissue sheath, or cardiac [amyloid demonstrated per appropriate stain
such as Congo red or alcian blue stain]).
7. Documentation that primary (light chain) amyloidosis disease has been evaluated and
ruled out (ie, original laboratory result, or copy).
8. Evidence of NYHA classification I, II, III, or IV.
Exclusion Criteria
1. Chronic use of diflunisal, TTR stabilizer, tauroursodeoxycholate, doxycycline,
digitalis, 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.
2. Use of certain non-steroidal anti-inflammatory drugs (NSAIDs) [Section 5.8.1].
3. Liver and/or heart transplant, or implanted cardiac mechanical assist device.
4. Require initiation of treatment with calcium channel blockers.
5. Urinary retention requiring chronic self-catheterization.
6. Subjects with heart failure that in the opinion of the investigator is on the basis of
ischemic heart disease (eg, prior myocardial infarction with documented history of
cardiac enzymes and ECG changes), or uncorrected valvular disease and not
primarily due to transthyretin amyloid cardiomyopathy.
7. 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
the study.
8. Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality 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
inappropriate for entry into this study.
digitalis, 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.
2. Use of certain non-steroidal anti-inflammatory drugs (NSAIDs) [Section 5.8.1].
3. Liver and/or heart transplant, or implanted cardiac mechanical assist device.
4. Require initiation of treatment with calcium channel blockers.
5. Urinary retention requiring chronic self-catheterization.
6. Subjects with heart failure that in the opinion of the investigator is on the basis of
ischemic heart disease (eg, prior myocardial infarction with documented history of
cardiac enzymes and ECG changes), or uncorrected valvular disease and not
primarily due to transthyretin amyloid cardiomyopathy.
7. 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
the study.
8. Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality 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
inappropriate for entry into this study.
The Estimated Number of Participants
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Taiwan
20 participants
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Global
2000 participants
