Phase 4
Completed N=53
A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Source: ClinicalTrials.gov NCT04814186 ↗Enrolled (actual)
53
Serious AEs
39.6%
Results posted
Dec 2024
Primary outcomePrimary: Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) — 45; 21; 17; 6 Participants
◆ Published Evidence
Not yet cited
0citations
Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy.
Summary
This is a national, multi-center, single-arm study, open-label to patients with symptomatic Transthyretin amyloid cardiomyopathy (ATTR-CM) who are tafamidis naïve. This study is to obtain safety, descriptive efficacy, Pharmacokinetics (PK) and Pharmacodynamics (PD) data for tafamidis orally once daily.
Subject eligibility for participation in the study will receive tafamidis once daily or 12 months following the assessment as the screening and baseline, month 1, 3, 6, 9 and 12 visits (or Early Study Discontinuation).
Linked Publications
-
Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) |
45; 21; 17; 6; 1; 1 | — |
| PRIMARY Number of Participants With Treatment-Related TEAEs |
3; 0; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline (CFB) for Distance Walked During 6 Minute Walk Test (6MWT) at Months 6 and 12 |
-19.3; 1.5 | — |
| SECONDARY Change From Baseline for N Terminal Prohormone B Type Natriuretic Peptide (NT-proBNP) at Months 6 and 12 |
452.9; -422.2 | — |
| SECONDARY Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12 |
96.4; 96.4; 96.2; 94.7 | — |
| SECONDARY TTR Concentration at Baseline, Months 1, 6, and 12 |
12.70; 19.73; 19.58; 21.48; 21.79 | — |
| SECONDARY Change From Baseline for Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Scores at Months 6 and 12 |
-0.4; -0.3 | — |
| SECONDARY Change From Baseline for EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Index Values at Months 6 and 12 |
0.0; 0.0 | — |
| SECONDARY Change From Baseline for EuroQol VAS at Months 6 and 12 |
-0.6; 3.4 | — |
| SECONDARY Change From Baseline for Physical Component Summary (PCS) for Short-Form Survey 12 (SF-12) at Months 6 and 12 |
0.1; -0.0 | — |
| SECONDARY Change From Baseline for Mental Component Summary (MCS) for SF-12 at Months 6 and 12 |
1.4; 1.5 | — |
| SECONDARY Plasma Concentrations of Tafamidis at Months 1, 6, and 12 |
10410; 12040; 14100; 11950 | — |
Eligibility Criteria
Inclusion Criteria
- Subject has documented ATTR-CM.
- For the reproductive criteria for male and female participants, please refer to relevant protocol sections.
Exclusion Criteria
- Other acute or chronic medical or psychiatric condition including recent or active suicidal ideation or behavior or laboratory abnormality, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
- Participants who have prior liver and/or heart transplant.
- Participants with primary (light chain) or secondary amyloidosis.
- Previous administration with an investigational drug within 30 days or 5 half lives preceding the first dose of study intervention used in this study (whichever is longer).
Data sourced from ClinicalTrials.gov (NCT04814186) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.