NCT01148953

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

A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Transthyretin Mediated Amyloidosis (ATTR)

Interventions

Type	Name	Description
DRUG	ALN-TTR01	Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion
DRUG	Sterile Normal Saline (0.9% NaCl)	Calculated volume to match active comparator

Timeline

• Start date: 2010-06-01
• Primary completion: 2012-01-01
• Completion: 2012-02-01
• First posted: 2010-06-23
• Last updated: 2012-05-24

Locations

4 sites across 4 countries: France, Portugal, Sweden, United Kingdom