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

• Status: Completed
• Trial end date: 2017-08-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

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