A Phase 3 interventional study of Danicopan in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Alexion Pharmaceuticals, Inc.. Active, not recruiting at 45 sites in 15 countries. Per ClinicalTrials.gov, last updated 2026-06-18.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 3, Interventional, and Treatment
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.
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.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's enrollment of 80 is above the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted after Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Also known as: ALXN2040, ACH-0144471
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Time frame: Up to 3 years
Change In Hemoglobin Values
Time frame: Up to 3 years
Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
Time frame: Up to 3 years
Change In Absolute Reticulocyte Count
Time frame: Up to 3 years
Change In Lactate Dehydrogenase (LDH)
Time frame: Up to 3 years
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
Time frame: Up to 3 years
Proportion Of Participants With Transfusion Avoidance
Time frame: Up to 3 years
Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores
Total scores range from 0 to 52, with higher scores indicating better QoL.
Time frame: Up to 3 years
Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores
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).
Time frame: Up to 3 years
Participants Experiencing TEAEs Leading To Discontinuation
Time frame: Up to 3 years
Plan to share: Yes — Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Supporting information: Study protocol, Clinical study report
No publications or documents are linked to this record.
Started Oct 2022 (actual). Primary completion is estimated for Aug 2026, 45 months after the start.
Of 35 industry-sponsored phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 41. This study's planned 45 months is above that.
Counting every sponsor, half of 35 studies reached it within 23 months; a quarter within 15 and three quarters within 41.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Danicopan
Alexion Pharmaceuticals, Inc. →