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) (APOLLO)

ID Number 14-1779

Principal Investigator(s)
Peter D Gorevic

Department(s) or Division(s)


The purpose of this study is to collect information about how patients with FAP respond to the study drug Patisiran (ALN-TTR02) over 18 months. In this study the study drug subjects receive either the active investigational drug patisiran (at a dose of 0.3 mg/kg) or placebo (a dummy infusion that only contains saline no active drug). Whether subjects receive patisiran or placebo will be determined randomly (by chance like flipping a coin). They will have a 2 in 3 chance of receiving patisiran and a 1 in 3 chance of receiving placebo. Neither the subject nor the study doctor will know whether they receive patisiran or placebo until the data are submitted and reviewed by regulatory authorities. 

Study drug (referring to both patisiran and placebo) will be given every 3 weeks for a total of 18 months. Study drug will be given through their vein over a 70-minute period by an infusion pump device. If the dose is late or missed it cannot be made up. The number of people expected to take part in this research study at this site is 10. The total number of people expected to take part in this research study is 200.

Contact Information
Garnette Mason
(212) 241-4824

Recruiting Patients: No