The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: TTR-mediated Amyloidosis

Treatments: Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion

Study type: Interventional

Funder types: Industry

Identifiers

NCT01961921

ALN-TTR02-003

2013-001644-65 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Enrollment: 27 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
• Adequate Karnofsky performance status, liver function, and renal function.

Exclusion criteria

• Pregnant or nursing.
• Has had a liver transplant.
• Has a New York Heart Association heart failure classification >2.
• Has unstable angina.
• Has uncontrolled clinically significant cardiac arrhythmia.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 1 patient group

ALN-TTR02 (patisiran)

Experimental group

Treatment:

Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion