Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT06449001
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
A Phase 3 study testing Danicopan in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 4 sites in 3 countries.
Trial summary
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH). Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- What is being tested?
- Danicopan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 11 Aug 2025to 10 Mar 2028
- 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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Confirmed diagnosis of PNH.
- CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
- Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
- all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
- vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae
Who cannot join
- Platelet count < 30000/μL or there is a need for platelet transfusions.
- ANC < 500/μL.
- Clinically significant laboratory abnormalities related to liver function, including:
- ALT > 2 × ULN or ALT > 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
- Direct bilirubin > 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
- Current evidence of biliary cholestasis.
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- Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.
- History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
- Known or suspected complement deficiency.
- Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.
Trial locations and contacts
4 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 3 countries
United Kingdom2 sites, 2 recruiting
- Recruiting
Research Site
Leeds, LS9 7TF
- Recruiting
Research Site
London, SE5 9RS
Canada1 site, 1 recruiting
- Recruiting
Research Site
Saskatoon, Saskatchewan, S7N 0W8
About this study
Official title: A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
- Being tested
- Danicopan
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis, Ravulizumab, Eculizumab, Danicopan
- 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
Participants will receive danicopan on a weight-based dosing regimen.
| Study group | Role | What they receive |
|---|---|---|
| Danicopan | Experimental | Participants will receive a 12-week weight-based open-label treatment period and up to 1 year open-label long term extension period. Receives: Drug: Danicopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12
Measured: Baseline, Week 12
Secondary outcomes (9)
Maximum Plasma Concentration (Cmax) of Danicopan
Measured: Day 1 up to Week 12
Number of Participants With Transfusion Avoidance Through Weeks 12 and 24
Measured: Weeks 12 and 24
Change From Baseline in Absolute Reticulocyte Count at Weeks 12 and 24
Measured: Baseline, Weeks 12 and 24
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales Score at Weeks 12 and 24
Measured: Baseline, Weeks 12 and 24
Change from Baseline in Pediatric Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 12 and 24
Measured: Baseline, Weeks 12 and 24
Acceptability and Palatability Questionnaire Score
Measured: Week 2
Change from Baseline in Hgb Concentration at Week 24
Measured: Baseline, Week 24
Change from Baseline in Serum Alternative Pathway (AP) Activity
Measured: Baseline up to Week 64
Change from Baseline in Plasma Bb Concentrations
Measured: Baseline up to Week 64
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2024
- Study start11 Aug 2025
- Primary completion28 May 2027 (estimated)
- Study completion10 Mar 2028 (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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AstraZeneca
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06449001
- Sponsor's protocol ID
- D7332C00006
- Other IDs
- ALXN2040-PNH-302
- Status last verified
- Dec 2025
- First submitted
- 3 Jun 2024
- Last updated
- 23 Dec 2025
- 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 23 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
- ClinicalTrials.gov ID
- NCT06449001
- Acronym / sponsor ID
- D7332C00006
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis, Ravulizumab, Eculizumab, Danicopan
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- AstraZeneca
- Study start
- 11 Aug 2025 (actual)
- Primary completion
- 28 May 2027 (estimated)
- Study completion
- 10 Mar 2028 (estimated)
- First posted
- 7 Jun 2024 (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.
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Danicopan | Experimental | Drug: DanicopanParticipants will receive a 12-week weight-based open-label treatment period and up to 1 year open-label long term extension period. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Confirmed diagnosis of PNH.
- CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
- Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
- all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
- vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae
Key exclusion criteria (10 listed)
- Platelet count < 30000/μL or there is a need for platelet transfusions.
- ANC < 500/μL.
- Clinically significant laboratory abnormalities related to liver function, including:
- Current evidence of biliary cholestasis.
- Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.
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 |
|---|---|---|
| DanicopanParticipants will receive danicopan on a weight-based dosing regimen. | Drug | Danicopan |
Primary outcome
Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12
Time frame: Baseline, Week 12
9 secondary outcomes
Maximum Plasma Concentration (Cmax) of Danicopan
Time frame: Day 1 up to Week 12
Number of Participants With Transfusion Avoidance Through Weeks 12 and 24
Time frame: Weeks 12 and 24
Change From Baseline in Absolute Reticulocyte Count at Weeks 12 and 24
Time frame: Baseline, Weeks 12 and 24
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales Score at Weeks 12 and 24
Time frame: Baseline, Weeks 12 and 24
Change from Baseline in Pediatric Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 12 and 24
Time frame: Baseline, Weeks 12 and 24
Acceptability and Palatability Questionnaire Score
Time frame: Week 2
Change from Baseline in Hgb Concentration at Week 24
Time frame: Baseline, Week 24
Change from Baseline in Serum Alternative Pathway (AP) Activity
Time frame: Baseline up to Week 64
Change from Baseline in Plasma Bb Concentrations
Time frame: Baseline up to Week 64
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 23 Dec 2025.
- 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.
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
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Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
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.
4 sites in 3 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 and PNH, run by Alexion Pharmaceuticals, Inc. at 4 sites in 3 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 5 inclusion and 10 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: looking for participants now, as of the sponsor's last update on 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United Kingdom, Canada, France. 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?
Contact Alexion Pharmaceuticals, Inc. (Sponsor) (1-855-752-2356, clinicaltrials@alexion.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12 (time frame: Baseline, Week 12). 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 23 Dec 2025.