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

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

NCT05389449•Alexion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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.

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.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

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.

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.

Locations (45)

Research Site

Los Angeles, California, United States

Research Site

Chicago, Illinois, United States

Research Site

Baltimore, Maryland, United States

Research Site

Kansas City, Missouri, United States

Research Site

Long Island City, New York, United States

Research Site

Cleveland, Ohio, United States

Research Site

Dallas, Texas, United States

Research Site

Porto Alegre, Brazil

Research Site

Rio de Janeiro, Brazil

Research Site

Toronto, Ontario, Canada

Key Dates

Start Date

October 28, 2022

Primary Completion Date

July 31, 2026

Completion Date

July 31, 2026

Last Updated

March 17, 2026

Enrollment

ACTUAL participants

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

Danicopan

DRUG

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

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Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

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RECRUITING

National Longitudinal Cohort of Hematological Diseases

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Multiple MyelomaAcute Myeloid Leukemia+15 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 17, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

National Longitudinal Cohort of Hematological Diseases

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

Home Reported Outcomes in PNH

Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors