NCT03181633

A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

An Open-Label Study to Evaluate Efficacy and Safety of Long-Term Treatment With ACH-0144471 in Participants Who Completed Clinical Study ACH471-100

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is designed to include up to 12 participants.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	ACH-0144471	ACH-0144471 will be administered to all participants enrolled in the study.

Timeline

• Start date: 2017-06-22
• Primary completion: 2022-01-04
• Completion: 2022-01-04
• First posted: 2017-06-09
• Last updated: 2023-03-14
• Results posted: 2023-03-14

Locations

4 sites across 3 countries: Italy, New Zealand, South Korea

Regulatory

• FDA-regulated drug study