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

• Status: Completed
• Trial end date: 2014-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

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.