Trials / Completed
CompletedNCT03053102
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 10 (actual)
- Sponsor
- Alexion Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Danicopan | Danicopan was administered as multiple oral doses over a period of at least 28 days. |
Timeline
- Start date
- 2017-03-31
- Primary completion
- 2018-11-14
- Completion
- 2018-11-14
- First posted
- 2017-02-14
- Last updated
- 2022-06-23
- Results posted
- 2021-06-02
Locations
5 sites across 4 countries: Italy, New Zealand, South Korea, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03053102. Inclusion in this directory is not an endorsement.