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

NCT05389449 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: ALXN2040-PNH-303
• Study Type: interventional
• Target: 80
• Locations: 15 countries
• Sites: 45

Brief 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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Paroxysmal Nocturnal Hemoglobinuria (PNH); Extravascular Hemolysis (EVH); Factor D inhibitor; Complement; Danicopan; ALXN2040; C5 inhibitor

Outcome Measures

Primary Outcomes (1)

• Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs

  Up to 3 years

Secondary Outcomes (9)

• 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

  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

Study Arms (1)

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.

Drug: Danicopan

Interventions

Danicopan DRUG

Oral tablet

Also known as: ALXN2040, ACH-0144471

Danicopan

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (45)

Research Site

Los Angeles, California, 90089, United States

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Chicago, Illinois, 60612, United States

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Baltimore, Maryland, 21231, United States

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Kansas City, Missouri, 64111, United States

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Long Island City, New York, 11101, United States

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Cleveland, Ohio, 44195, United States

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Dallas, Texas, 75390-9047, United States

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Porto Alegre, 90110-270, Brazil

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Rio de Janeiro, 20211-080, Brazil

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Toronto, Ontario, M5G 2C4, Canada

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Brno, 625 00, Czechia

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Lille, 59037, France

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Paris, 75010, France

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Pessac, 33604, France

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Pierre-Bénite, 69495, France

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Athens, 11527, Greece

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Thessaloniki, 57010, Greece

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Haifa, 31048, Israel

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Jerusalem, 91120, Israel

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Avellino, 83100, Italy

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Bassano del Grappa, 36061, Italy

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Florence, 50134, Italy

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Milan, 20122, Italy

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Reggio Calabria, 89131, Italy

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Roma, 161, Italy

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Bunkyō City, 113 8603, Japan

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Kyoto, 605-0981, Japan

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Osaka, 530-8480, Japan

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Shibuya-ku, 150-8935, Japan

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Tsukuba, 305-8576, Japan

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Kota Kinabalu, 88586, Malaysia

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Kuching, 93200, Malaysia

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Miri, 98000, Malaysia

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Gdansk, 80-952, Poland

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Seoul, 03722, South Korea

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Seoul, 06591, South Korea

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Suwon, 16247, South Korea

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Badalona, 8916, Spain

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Barcelona, 08036, Spain

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Majadahonda, 28222, Spain

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Seville, 41013, Spain

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Bangkok, 10330, Thailand

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Airdrie, ML6 0JS, United Kingdom

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Leeds, LS9 7TF, United Kingdom

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London, SE5 9RS, United Kingdom

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MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Hemolysis

Interventions

danicopan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2022
• First Posted: May 25, 2022
• Study Start: October 28, 2022
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): July 31, 2026
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR

Locations

ES (4); CA (1); BR (2); FR (4); US (7); MY (3); CZ (1); JP (5); KR (3); TH (1); IL (2); IT (6); GB (3); PL (1); GR (2)