A Long term Safety and Efficacy Study of Danicopan as an Add-on Therapy to C5i in Patients with PNH.

Phase 1

Recruiting

• Conditions: Patients with Paroxysmal Nocturnal Hemoglobinuria; MedDRA version: 21.1Level: PTClassification code: 10034042Term: Paroxysmal nocturnal haemoglobinuria Class: 100000004857; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: CTIS2023-504867-18-00
• Lead Sponsor: Alexion Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-07
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

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.

Exclusion Criteria

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To characterize the long-term safety of treatment with danicopan as an add-on therapy to a complement component 5 inhibitor (C5i);Secondary Objective: To characterize long-term efficacy of danicopan as an add-on therapy to a C5i, To characterize the long-term effect of treatment with danicopan as an add-on therapy to a C5i on Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue scores and on European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire-Core 30 Scale (EORTC-QLQ-C30) scores, To further characterize the safety of danicopan as an add-on therapy to a C5i;Primary end point(s): Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs

Secondary Outcome Measures

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