Clinical Trial: A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria
Brief Summary: The purpose of this study is to determine the safety and effectiveness of ACH-0144471 in currently untreated patients with PNH.
Detailed Summary:
Sponsor: Achillion Pharmaceuticals
Current Primary Outcome: Change-from-baseline in serum lactate dehydrogenase (LDH) levels [ Time Frame: Day 28 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change-from-baseline in hemoglobin (Hgb) [ Time Frame: Day 28 ]
- Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), and AEs leading to discontinuation [ Time Frame: Up to Day 28 ]
- Grade 3 and Grade 4 laboratory abnormalities [ Time Frame: Up to Day 28 ]
- Profile of Pharmacokinetics: Area under the curve (AUC) [ Time Frame: Up to Day 28 ]
- Profile of Pharmacokinetics: Maximum plasma concentration (Cmax) [ Time Frame: Up to Day 28 ]
- Profile of Pharmacokinetics: Time to maximum concentration (Tmax) [ Time Frame: Up to Day 28 ]
- Profile of Pharmacodynamics: Complement pathway biomarkers (AP-Wieslab, factor D, and Bb) [ Time Frame: Up to Day 28 ]
Original Secondary Outcome: Same as current
Information By: Achillion Pharmaceuticals
Dates:
Date Received: February 1, 2017
Date Started: March 31, 2017
Date Completion: August 2017
Last Updated: May 15, 2017
Last Verified: May 2017
