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

Phase 1

• Conditions: Patients with Paroxysmal Nocturnal Hemoglobinuria; MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2021-004253-22-GR
• Lead Sponsor: Alexion Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-11-29
• Last Update Posted: 29 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 95

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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 93
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

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
Secondary Objective: -To characterize the long-term efficacy of danicopan as an add-on therapy to a C5i <br>-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 (EORTC-QLQ-C30) scores<br>-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 ;Timepoint(s) of evaluation of this end point: Over time throughout the study. Planned time points for all safety assessments are provided in the SoA (Section 1.3 of the protocol);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 Outcome Measures

Name	Time	Method
Secondary end point(s): • Change in hemoglobin (Hgb) values over time<br>• Change in absolute reticulocyte count over time<br>• Change in lactate dehydrogenase (LDH) over time<br>• Proportion of patients with LDH = 1.5 × upper limit of normal (ULN) over time<br>• Proportion of patients with transfusion avoidance (TA), defined as patients who remain transfusion free and do not require transfusion as per protocol specified guidelines<br>• Change in FACIT Fatigue scores over time<br>• Change in EORTC-QLQ-C30 scores over time<br>• Change in safety laboratory parameters over time<br>• TEAEs leading to discontinuation<br>• Proportion of patients with Hgb increase of = 2g/dL in the absence of transfusion over time<br><br>;Timepoint(s) of evaluation of this end point: Over time throughout the study. Planned time points for all safety assessments are provided in the SoA (Section 1.3 of the protocol)