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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Danicopan | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Danicopan | Drug | Oral tablet |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs | Up to 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change In Hemoglobin Values | Up to 3 years | |
| Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time | Up to 3 years | |
| Change In Absolute Reticulocyte Count |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Los Angeles | California | 90089 | United States | ||
| Research Site |
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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| 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 | Total scores range from 0 to 52, with higher scores indicating better QoL. | 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). | Up to 3 years |
| Participants Experiencing TEAEs Leading To Discontinuation | Up to 3 years |
| Chicago |
| Illinois |
| 60612 |
| United States |
| Research Site | Baltimore | Maryland | 21231 | United States |
| Research Site | Kansas City | Missouri | 64111 | United States |
| Research Site | Long Island City | New York | 11101 | United States |
| Research Site | Cleveland | Ohio | 44195 | United States |
| Research Site | Dallas | Texas | 75390-9047 | United States |
| Research Site | Porto Alegre | 90110-270 | Brazil |
| Research Site | Rio de Janeiro | 20211-080 | Brazil |
| Research Site | Toronto | Ontario | M5G 2C4 | Canada |
| Research Site | Brno | 625 00 | Czechia |
| Research Site | Lille | 59037 | France |
| Research Site | Paris | 75010 | France |
| Research Site | Pessac | 33604 | France |
| Research Site | Pierre-Bénite | 69495 | France |
| Research Site | Athens | 11527 | Greece |
| Research Site | Thessaloniki | 57010 | Greece |
| Research Site | Haifa | 31048 | Israel |
| Research Site | Jerusalem | 91120 | Israel |
| Research Site | Avellino | 83100 | Italy |
| Research Site | Bassano del Grappa | 36061 | Italy |
| Research Site | Florence | 50134 | Italy |
| Research Site | Milan | 20122 | Italy |
| Research Site | Reggio Calabria | 89131 | Italy |
| Research Site | Roma | 161 | Italy |
| Research Site | Bunkyō City | 113 8603 | Japan |
| Research Site | Kyoto | 605-0981 | Japan |
| Research Site | Osaka | 530-8480 | Japan |
| Research Site | Shibuya-ku | 150-8935 | Japan |
| Research Site | Tsukuba | 305-8576 | Japan |
| Research Site | Kota Kinabalu | 88586 | Malaysia |
| Research Site | Kuching | 93200 | Malaysia |
| Research Site | Miri | 98000 | Malaysia |
| Research Site | Gdansk | 80-952 | Poland |
| Research Site | Seoul | 03722 | South Korea |
| Research Site | Seoul | 06591 | South Korea |
| Research Site | Suwon | 16247 | South Korea |
| Research Site | Badalona | 8916 | Spain |
| Research Site | Barcelona | 08036 | Spain |
| Research Site | Majadahonda | 28222 | Spain |
| Research Site | Seville | 41013 | Spain |
| Research Site | Bangkok | 10330 | Thailand |
| Research Site | Airdrie | ML6 0JS | United Kingdom |
| Research Site | Leeds | LS9 7TF | United Kingdom |
| Research Site | London | SE5 9RS | United Kingdom |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| D006461 | Hemolysis |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000718467 | danicopan |
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