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

Alnylam Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3

Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Treatments: Drug: Placebo; Drug: Patisiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT03997383

ALN-TTR02-011

2019-001458-24 (EudraCT Number)

Details and patient eligibility

About

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

Enrollment: 360 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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 <8500 ng/L; in participants with permanent or persistent atrial fibrillation, screening NT-proBNP> 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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360 participants in 2 patient groups, including a placebo group

Patisiran

Experimental group

Description:

Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.

Treatment:

Drug: Patisiran

Placebo

Placebo Comparator group

Description:

Participants will be administered multiple doses of placebo in the double-blind period. In the open-label extension period, participants will be administered multiple doses of patisiran.

Treatment:

Drug: Patisiran

Drug: Placebo