APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)

The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis. An open-label, single-arm, long-term follow-up extension study NCT02510261 (ALN-TTR02-006) was initiated to provide participants who completed this study with continued patisiran-LNP (lipid nanoparticle) treatment.

Inclusion Criteria:

• Male or female of 18 to 85 years of age (inclusive);
• Have a diagnosis of FAP
• Neuropathy Impairment Score requirement of 5-130
• Meet Karnofsky performance status requirements
• Have adequate complete blood counts and liver function tests
• Have adequate cardiac function
• Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)

Exclusion Criteria:

• Had a prior liver transplant or is planned to undergo liver transplant during the study period;
• Has untreated hypo- or hyperthyroidism;
• Has known human immunodeficiency virus (HIV) infection;
• Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
• Recently received an investigational agent or device
• Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid