drugset

drugset / Trial / NCT03053102

Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT03053102 ↗

Phase 2 Completed 10 enrolled Alexion Pharmaceuticals, Inc. Achillion, a wholly owned subsidiary of Alexion · collab

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2017-03-31
Primary completion: 2018-11-14
Completion: 2018-11-14

Publications

Results Risitano AM, Kulasekararaj AG, Lee JW, Maciejewski JP, Notaro R, Brodsky R, Huang M, Geffner M, Browett P. Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Haematologica. 2021 Dec 1;106(12):3188-3197. doi: 10.3324/haematol.2020.261826.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Danicopan	Small molecule	100 mg	Oral
Subject	Danicopan	Small molecule	150 mg	Oral
Subject	Danicopan	Small molecule	200 mg	Oral

Indications