Phase 3 N=360 Randomized Quadruple-blind Treatment

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Bottom Line

View on ClinicalTrials.gov: NCT03997383 ↗

Enrolled (actual)

360

Serious AEs

17.9%

Results posted

Oct 2023

Primary outcome: Primary: Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT) — -21.345; -8.150 meters — p=0.0162

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Placebo (Drug); Patisiran (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Jun 2022

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

Summary

The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03997383). 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.