For Healthcare Professionals

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


About the study

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.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:


Key Inclusion Criteria:

  1. All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment.
  2. 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.
  3. 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:

  1. 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.
  2. Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study.
  3. Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.

pin location

Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Paroxysmal Nocturnal Hemoglobinuria

Age (in years)



Phase 3

Participants needed


Est. Completion Date

Feb 26, 2027

Treatment type



Alexion Pharmaceuticals, Inc. identifier


Study number


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