Ignite Creation Date:
2025-12-25 @ 3:37 AM
Ignite Modification Date:
2025-12-26 @ 2:21 AM
Study NCT ID:
NCT03053102
Status:
COMPLETED
Last Update Posted:
2022-06-23
First Post:
2017-02-01
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Sponsor:
Alexion Pharmaceuticals, Inc.
Organization:
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2022-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
Detailed Description:
After 12 weeks of treatment, participants deriving clinical benefit were offered enrollment in a separate long-term extension study (ACH471-103, NCT03181633).
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?: