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)

ALN-TTR02-004

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.

2013-11-01

2017-08-01

2017-08-01

Completed

Early phase I

225

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 225, 'type': 'ACTUAL'}}

2024-04-22