A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study.

Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.18 sites in 6 countries25 target enrollmentJuly 4, 2024

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Not specified
Sponsor: Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.
Enrollment: 25
Locations: 18
Primary Endpoint: Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs
Status: Active, Not Recruiting
Last Updated: last year

To characterize the long-term safety of treatment with danicopan as an add-on therapy to a complement component 5 inhibitor (C5i)

Registry

euclinicaltrials.eu

Start Date

July 4, 2024

End Date

TBD

Last Updated

last year

Sponsor

Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.

Responsible Party

Principal Investigator

European Clinical Trial Information

Scientific

Alexion Pharmaceuticals Inc.

•All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment
•Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.

•Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant.
•Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.

Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs

Change in hemoglobin (Hgb) values over time
Proportion of patients with Hgb increase of ≥ 2g/dL in the absence of transfusion over time
Change in absolute reticulocyte count over time
Change in lactate dehydrogenase (LDH) over time
Proportion of patients with LDH ≤ 1.5 × upper limit of normal (ULN) over time
Proportion of patients with transfusion avoidance (TA), defined as patients who remain transfusion free and do not require transfusion as per protocol specified guidelines
Change in FACIT Fatigue scores over time
Change in EORTC-QLQ-C30 scores over time
Change in safety laboratory parameters over time
TEAEs leading to discontinuation