NCT03862807

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.

Conditions

• Amyloidosis, Familial
• Transthyretin Amyloidosis

Interventions

Type	Name	Description
DRUG	Patisiran	Patisiran was administered via IV infusion.

Timeline

• Start date: 2019-03-27
• Primary completion: 2020-10-06
• Completion: 2020-10-20
• First posted: 2019-03-05
• Last updated: 2024-04-22
• Results posted: 2021-12-21

Locations

9 sites across 7 countries: France, Germany, Italy, Portugal, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study