Danicopan Early Access Program
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age18+
SponsorAlexion Pharmaceuticals, Inc.
About this trial
This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female, aged 18 years and older
Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L.
Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment.
Platelet count ≥ 30,000/µL without the need for platelet transfusions.
Disqualifiers
Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan).
Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN)
participants with increased bilirubin due to EVH in the opinion of the Treating Physician or
participants with documented Gilbert's syndrome
Trial design
Treatments tested in this trial
Danicopan
DrugParticipants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect.
Treatment groups
No treatment groups listed
Trial outcomes
This trial has no outcomes listed.Sponsors and contacts
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Source ClinicalTrials.gov