Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis

• Status: Completed
• Trial end date: 2012-02-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Diagnosis of TTR amyloidosis

• Adequate blood counts, liver and renal function

• Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control

• Males agree to use appropriate contraception

• Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent.

Exclusion Criteria:

• Known human immunodeficiency virus (HIV) positive status

• Receiving antibiotics for bacterial infection within 7 days of screening

• Known or suspected systemic viral, parasitic or fungal infection

• Receiving an investigational agent within 30 days prior to study drug administration

• Poor cardiac function

• Considered unfit for the study by the Principal Investigator

• Known sensitivity to oligonucleotides

• Employee or family member of the sponsor or the clinical study site personnel.