Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR Amyloidosis

This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).

Inclusion Criteria:

• Body mass index must be between 17 kg/m^2 and ≤ 33 kg/m^2;
• Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;
• Males agree to use appropriate contraception;
• Diagnosis of TTR amyloidosis;
• Adequate blood counts, liver and renal function;
• Willing to give written informed consent and are willing to comply with the study requirements.

Exclusion Criteria:

• Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
• Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to first dose study drug administration;
• Prior liver transplant;
• Poor cardiac function;
• Considered unfit for the study by the Principal Investigator;
• Employee or family member of the sponsor or the clinical study site personnel.