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

Alnylam Pharmaceuticals

Status and phase

Completed

Phase 3

Conditions

Familial Amyloid Polyneuropathies

TTR-mediated Amyloidosis

Amyloidosis, Hereditary, Transthyretin-Related

Amyloidosis, Hereditary

Amyloid Neuropathies

Amyloid Neuropathies, Familial

Treatments

Drug: patisiran (ALN-TTR02)

Drug: Sterile Normal Saline (0.9% NaCl)

Study type

Interventional

Funder types

Industry

Identifiers

NCT01960348

2013-002987-17 (EudraCT Number)

ALN-TTR02-004

Details and patient eligibility

About

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.

Enrollment

225 patients

Sex

All

Ages

18 to 85 years old

Volunteers

No Healthy Volunteers

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