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.

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-504867-18-00

Acronym

ALXN2040-PNH-303

Enrollment

Registered

2024-07-04

Start date

2022-12-05

Completion date

Unknown

Last updated

2025-12-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Patients with Paroxysmal Nocturnal Hemoglobinuria

Brief summary

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

Detailed description

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

Interventions

DRUGALXN 2040

DRUGSoliris 300 mg concentrate for solution for infusion

DRUGUltomiris 300 mg/30 mL concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs	—

Secondary

Measure	Time frame
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	—

Measure

Time frame

—

Countries

Czechia, France, Greece, Italy, Poland, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026