Phase 3
Completed N=360
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Source: ClinicalTrials.gov NCT03997383 ↗
Enrolled (actual)
360
Serious AEs
17.9%
Results posted
Oct 2023
Primary outcomePrimary: Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT) — -21.345; -8.150 meters — p=0.0162
◆ Published Evidence
Highly cited
309citations · ~103 / year
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.
Summary
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
Linked Publications (2)
-
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.
-
Patisiran Treatment in the Brazilian Subpopulation of the Phase 3 APOLLO-B Study in Transthyretin Amyloidosis with Cardiomyopathy: Post Hoc Analysis.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT) |
-21.345; -8.150 | 0.0162 sig |
| SECONDARY Change From Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score |
-3.396; 0.478 | 0.0397 sig |
| SECONDARY Composite Endpoint of All-Cause Mortality, Frequency of Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) and Change From Baseline in 6-MWT Analyzed by Win Ratio |
1.22; 1.28 | 0.0574 |
| SECONDARY Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits in Participants Not on Tafamidis at Baseline |
0.997 | 0.9888 |
| SECONDARY Composite Endpoint of All-cause Mortality and Frequency of All-cause Hospitalizations and Urgent HF Visits in All Participants |
0.883 | 0.5609 |
Eligibility Criteria
Inclusion Criteria
- Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with cardiomyopathy
- Medical history of heart failure with at least 1 prior hospitalization for heart failure, or current clinical evidence (signs and symptoms of heart failure)
- Clinically stable with no cardiovascular related hospitalizations within 6 weeks of study start
- Has never taken tafamidis before (tafamidis naïve) or currently on tafamidis for ≥6 months with evidence of disease progression while on tafamidis treatment
- Able to complete ≥150 m on the 6-minute walk test
- Screening N-terminal pro B-type natriuretic peptide (NT-proBNP), a blood marker of heart failure severity, >300 ng/L and 600 ng/L and <8500 ng/L
Exclusion Criteria
- Known primary amyloidosis (AL) or leptomeningeal amyloidosis.
- Received prior TTR lowering treatment
- New York Heart Association heart failure classification of III and at high risk
- New York Heart Association heart failure classification of IV
- Neuropathy requiring cane or stick to walk, or is wheelchair bound
- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2
- Abnormal liver function
- Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- Has non-amyloid disease that significantly affects ability to walk (e.g., severe chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular disease affecting ambulation)
- Prior or planned heart, liver, or other organ transplant
- Other cardiomyopathy not related to ATTR amyloidosis
Data sourced from ClinicalTrials.gov (NCT03997383) 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.