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

• Status: Completed
• Trial end date: 2018-11-14
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Achillion Pharmaceuticals; Alexion Pharmaceuticals
• Collaborator: Achillion, a wholly owned subsidiary of Alexion

Criteria

Inclusion Criteria:

• Currently untreated PNH participants with PNH Type III erythrocyte and/or granulocyte clone size ≥10% and anemia (hemoglobin <12 grams/deciliter) with adequate reticulocytosis (as determined by the Investigator).

• LDH ≥1.5 x the upper limit of normal.

• Platelets ≥50,000/microliter without the need for platelet transfusions.

• Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae, or willingness to receive vaccinations during the screening period.

• Negative pregnancy test for females prior to dosing and throughout the study.

Exclusion Criteria:

• History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.

• Participants who had received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater.

• Participants who had received eculizumab at any dose or interval within the past 75 days before study entry.

• Participants with known or suspected complement deficiency.

• Participants with active bacterial infection or clinically significant active viral infection, a body temperature >38°Celsius, or other evidence of infection on Day 1, or with a history of febrile illness within 14 days prior to first study drug administration.

• History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection.

• Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.