Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT05389449
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
A Phase 3 study testing Danicopan in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 45 sites in 15 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Danicopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 45 sitesin 15 countries
- Study dates
- 28 Oct 2022to 24 Aug 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
45 sites in 15 countries.
45 sites in 15 countries
United States7 sites
Research Site
Baltimore, Maryland, 21231
Research Site
Chicago, Illinois, 60612
Research Site
Cleveland, Ohio, 44195
Research Site
Dallas, Texas, 75390-9047
Research Site
Kansas City, Missouri, 64111
Research Site
Long Island City, New York, 11101
Research Site
Los Angeles, California, 90089
Italy6 sites
Research Site
Avellino, 83100
Research Site
Bassano del Grappa, 36061
Research Site
Florence, 50134
Research Site
Milan, 20122
Research Site
Reggio Calabria, 89131
Research Site
Roma, 161
Japan5 sites
Research Site
Bunkyō City, 113 8603
Research Site
Kyoto, 605-0981
Research Site
Osaka, 530-8480
Research Site
Shibuya-ku, 150-8935
Research Site
Tsukuba, 305-8576
France4 sites
Research Site
Lille, 59037
Research Site
Paris, 75010
Research Site
Pessac, 33604
Research Site
Pierre-Bénite, 69495
Spain4 sites
Research Site
Badalona, 8916
Research Site
Barcelona, 08036
Research Site
Majadahonda, 28222
Research Site
Seville, 41013
Malaysia3 sites
Research Site
Kota Kinabalu, 88586
Research Site
Kuching, 93200
Research Site
Miri, 98000
South Korea3 sites
Research Site
Seoul, 03722
Research Site
Seoul, 06591
Research Site
Suwon, 16247
United Kingdom3 sites
Research Site
Airdrie, ML6 0JS
Research Site
Leeds, LS9 7TF
Research Site
London, SE5 9RS
Brazil2 sites
Research Site
Porto Alegre, 90110-270
Research Site
Rio de Janeiro, 20211-080
About this study
Official title: A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical 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.
Read the detailed description
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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Danicopan
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, ALXN2040, C5 inhibitor
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Hemolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanicopanDrug
Also called ALXN2040, ACH-0144471
Oral tablet
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Danicopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Measured: Up to 3 years
Secondary outcomes (9)
Change In Hemoglobin Values
Measured: Up to 3 years
Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
Measured: Up to 3 years
Change In Absolute Reticulocyte Count
Measured: Up to 3 years
Change In Lactate Dehydrogenase (LDH)
Measured: Up to 3 years
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
Measured: Up to 3 years
Proportion Of Participants With Transfusion Avoidance
Measured: 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.
Measured: 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).
Measured: Up to 3 years
Participants Experiencing TEAEs Leading To Discontinuation
Measured: Up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2022
- Study start28 Oct 2022
- Primary completion24 Aug 2026 (estimated)
- Study completion24 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05389449
- Sponsor's protocol ID
- ALXN2040-PNH-303
- Status last verified
- Jun 2026
- First submitted
- 20 May 2022
- Last updated
- 18 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study
- ClinicalTrials.gov ID
- NCT05389449
- Acronym / sponsor ID
- ALXN2040-PNH-303
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, ALXN2040, C5 inhibitor
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Oct 2022 (actual)
- Primary completion
- 24 Aug 2026 (estimated)
- Study completion
- 24 Aug 2026 (estimated)
- First posted
- 25 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Danicopan | Experimental | Drug: DanicopanParticipants 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DanicopanAlso: ALXN2040, ACH-0144471Oral tablet | Drug | Danicopan |
Primary outcome
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Time frame: Up to 3 years
9 secondary outcomes
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
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
Time frame: Up to 3 years
Participants Experiencing TEAEs Leading To Discontinuation
Time frame: Up to 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2026.
- Individual participant dataSharing planned: 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.
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Enrolling by invitationObservational study
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RecruitingPhase 3
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Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
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RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
45 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Danicopan in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 45 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 45 sites in United States, Italy, Japan, France, Spain and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs (time frame: Up to 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2026.