APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
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|ClinicalTrials.gov Identifier: NCT03997383|
Recruitment Status : Active, not recruiting
First Posted : June 25, 2019
Last Update Posted : May 23, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Transthyretin Amyloidosis (ATTR) With Cardiomyopathy||Drug: Placebo Drug: Patisiran||Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||360 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)|
|Actual Study Start Date :||September 4, 2019|
|Actual Primary Completion Date :||June 20, 2022|
|Estimated Study Completion Date :||June 2025|
Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.
Patisiran will be administered by intravenous (IV) infusion.
Placebo Comparator: Placebo
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.
Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously.
Patisiran will be administered by intravenous (IV) infusion.
- Change from Baseline at Month 12 in Six-Minute Walk Test (6-MWT) [ Time Frame: Baseline, Month 12 ]
- Change from Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score [ Time Frame: Baseline, Month 12 ]The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
- 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 [ Time Frame: Up to Month 12 ]This composite endpoint will be analyzed using the win ratio method. This method combines all-cause mortality, frequency of CV events (CV hospitalizations and HF visits) and change from baseline in 6-MWT in a hierarchical fashion. The method uses pairwise comparisons for all possible active/placebo patient pairs. A 'win' represents a patient doing better based on the hierarchical comparison. The win ratio is the total number of 'winners' divided by the total number of 'losers' in the active group.
- Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits [ Time Frame: Up to Month 12 ]The hazard rate of all-cause mortality and all-cause hospitalizations and urgent HF visits will be compared between treatment groups using an Andersen-Gill model.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years to 85 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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 patients with permanent or persistent atrial fibrillation, screening NT-proBNP> 600 ng/L and <8500 ng/L
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03997383
|Study Director:||Medical Director||Alnylam Pharmaceuticals|
|Responsible Party:||Alnylam Pharmaceuticals|
|Other Study ID Numbers:||
2019-001458-24 ( EudraCT Number )
|First Posted:||June 25, 2019 Key Record Dates|
|Last Update Posted:||May 23, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Amyloid Neuropathies, Familial
Heredodegenerative Disorders, Nervous System
Nervous System Diseases
Peripheral Nervous System Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors