A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05389449

Collaborator

(none)

100

Enrollment

Arm

40.3

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.

Condition or Disease	Intervention/Treatment	Phase
Paroxysmal Nocturnal Hemoglobinuria	Drug: Danicopan	Phase 3

Detailed Description

The total duration of the study will be up to 3 years. Eligible participants must complete all study assessments on the parent protocol before starting this study. All participants entering this study will receive danicopan as an add-on to a background C5i therapy. The only allowed C5i therapies are eculizumab and ravulizumab.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

100 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Aug 15, 2022

Anticipated Primary Completion Date :

Dec 25, 2025

Anticipated Study Completion Date :

Dec 25, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Danicopan Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.	Drug: Danicopan Oral tablet Other Names: ALXN2040, ACH-0144471

Arm

Intervention/Treatment

Experimental: Danicopan

Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.

Drug: Danicopan

Oral tablet

Other Names:

ALXN2040, ACH-0144471

Outcome Measures

Primary Outcome Measures

Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs [Up to 3 years]

Secondary Outcome Measures

Change In Hemoglobin Values [Up to 3 years]
Change In Absolute Reticulocyte Count [Up to 3 years]
Change In Lactate Dehydrogenase (LDH) [Up to 3 years]
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal [Up to 3 years]
Proportion Of Participants With Transfusion Avoidance [Up to 3 years]
Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores [Up to 3 years]
Total scores range from 0 to 52, with higher scores indicating better QoL.
Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores [Up to 3 years]
Thirty questions related to QoL, with the first 28 questions scored on a 4-point scale (1 = not at all to 4 = very much) and the final 2 questions that probe the participant's overall health and QoL scored on a scale of 1 (very poor) to 7 (excellent).
Participants Experiencing TEAEs Leading To Discontinuation [Up to 3 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment.
Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol.
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.

Key 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.
Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study.
Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.