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

Status:	Completed
Conditions:	Neurology, Hematology
Therapuetic Areas:	Hematology, Neurology
Healthy:	No
Age Range:	18 - 85
Updated:	12/13/2018
Start Date:	November 2013
End Date:	August 2017

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

We found this trial at

sites

Clinical Trial Site

Durham, North Carolina

from

Durham, NC