NCT01617967

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

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).

Conditions

• TTR-mediated Amyloidosis

Interventions

Type	Name	Description
DRUG	Patisiran	Participants received a single dose of patisiran as an intravenous (IV) infusion on Day 0 and Day 28 (Q4W). Optional cohorts received an alternative dosing regimen (once every 3 weeks \[Q3W\]: Day 0 and Day 21) and an alternative premedication regimen.

Timeline

• Start date: 2012-05-01
• Primary completion: 2013-10-01
• Completion: 2014-01-01
• First posted: 2012-06-13
• Last updated: 2024-04-19
• Results posted: 2018-11-21

Locations

10 sites across 7 countries: United States, Brazil, France, Germany, Portugal, Spain, Sweden

Regulatory

• FDA-regulated drug study