Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT04469465
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
A Phase 3 study testing Danicopan and C5 Inhibitor in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 62 sites in 18 countries.
Trial summary
The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Danicopan and C5 Inhibitor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 86 participants
- Where
- 62 sitesin 18 countries
- Study dates
- 16 Dec 2020to 16 Jan 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of PNH
- Clinically Evident EVH defined by:
- Anemia (Hgb ≤9.5 gram/deciliter) with absolute reticulocyte count ≥120 x 10^9/liter
- Receiving an approved C5 inhibitor for at least 6 months prior to Day 1
- Platelet count ≥30,000/microliters (µL)
- Absolute neutrophil counts ≥500/μL
- Documentation of/or willingness to receive vaccinations for N. meningiditis and prophylactic antibiotics as required
Who cannot join
- History of a major organ transplant or hematopoietic stem cell transplantation (HSCT)
- Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants
- Known or suspected complement deficiency
- Laboratory abnormalities at screening, including:
- Alanine aminotransferase >2 x ULN (>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values
- 500 ng/ML)
Show all 10Show fewer
- Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
- Current evidence of biliary cholestasis
- Estimated glomerular filtration rate of <30 milliliters/minute/1.73 meter squared and/or are on dialysis
- Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening
Trial locations and contacts
62 sites in 18 countries.
62 sites in 18 countries
Japan12 sites
Research Site
Bunkyō City, 113 8603
Research Site
Fukuoka, 812-8582
Research Site
Kashiwa-shi, 277-8567
Research Site
Kyoto, 605-0981
Research Site
Nagakute-shi, 480-1195
Research Site
Ogaki-shi, 503-8502
Research Site
Osaka, 530-8480
Research Site
Sayama, 589-8511
Research Site
Shibuya-ku, 150-8935
Research Site
Tanabe-shi, 646-8588
Research Site
Toyoake-shi, 470-1192
Research Site
Tsukuba, 305-8576
United States7 sites
Research Site
Chicago, Illinois, 60612
Research Site
Dallas, Texas, 75390
Research Site
Kalamazoo, Michigan, 49007
Research Site
Los Angeles, California, 90089
Research Site
Milwaukee, Wisconsin, 53212
Research Site
New York, New York, 10065
Research Site
Weston, Florida, 33331
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, 00161
Spain6 sites
Research Site
Barcelona, 08036
Research Site
Barcelona, 08916
Research Site
Las Palmas de Gran Canaria, 35020
Research Site
Madrid, 28040
Research Site
Majadahonda, 28222
Research Site
Seville, 41013
Brazil5 sites
Research Site
Belém, 66053-000
Research Site
Curitiba, 80810-050
Research Site
Goiânia, 74605-020
Research Site
Porto Alegre, 90110-270
Research Site
Rio de de Janeiro, 20211030
South Korea5 sites
Research Site
Daejeon, 35015
Research Site
Seoul, 03722
Research Site
Seoul, 06351
Research Site
Seoul, 06591
Research Site
Suwon, 16247
France4 sites
Research Site
Lille, 59037
Research Site
Paris, 75010
Research Site
Pessac, 33604
Research Site
Pierre-Bénite, 69495
Malaysia3 sites
Research Site
Kota Kinabalu, 88586
Research Site
Kuching, 93586
Research Site
Miri, 98000
United Kingdom3 sites
Research Site
Airdrie, ML6 0JS
Research Site
Leeds, BD7 1DP
Research Site
London, SE5 9NU
About this study
Official title: A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.
Read the detailed description
This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (eculizumab or ravulizumab).
Participants will be randomized to receive danicopan or placebo, in a 2:1 ratio for 12 weeks (Treatment Period 1) in addition to their C5 inhibitor (eculizumab or ravulizumab) therapy. At Week 12, participants randomized to receive placebo will be switched to danicopan in addition to their C5 inhibitor for an additional 12 weeks (Treatment Period 2) and participants randomized to danicopan will continue on danicopan for an additional 12 weeks, while remaining on their ongoing C5 inhibitor therapy.
At the end of the 2 treatment periods (Week 24), participants may enter a Long-Term Extension (LTE) Period and continue to receive danicopan in addition to their C5 inhibitor therapy. The Long-Term Extension period will consist of a first year of LTE(Year1) and a second year of optional LTE(Year2).All patients will complete 72 weeks of LTE(Year 1) assessments. After Week 72 (at the end of the first year of LTE), patients have the choice to complete participation in this study or continue to the optional second year (Year2) of LTE.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Danicopan, Placebo, C5 Inhibitor
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, 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
Oral tablet
PlaceboDrug
Oral tablet
C5 InhibitorDrug
Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.
| Study group | Role | What they receive |
|---|---|---|
| Danicopan + C5 Inhibitor | Experimental | Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2). Receives: Drug: Danicopan; Drug: C5 Inhibitor |
| Placebo + C5 Inhibitor | Placebo comparator | Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2). Receives: Drug: Placebo; Drug: C5 Inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Hgb at Week 12
Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.
Measured: Baseline, Week 12
Secondary outcomes (18)
Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 12 and remaining transfusion free during the 12-Week TP1. Participants who withdrew from the study early during the 12-Week TP1 or had missing Hgb value at Week 12 were considered as not achieving the criterion.
Measured: Week 12
Percentage of Participants With Transfusion Avoidance Through Week 12
Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 12. Participants who discontinued study treatment early before Week 12 were considered as not achieving transfusion avoidance.
Measured: Week 12
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue and better health-related quality of life. LS mean and SE were produced using MMRM.
Measured: Baseline, Week 12
Change From Baseline in Absolute Reticulocyte Count at Week 12
LS mean and SE were produced using MMRM.
Measured: Baseline, Week 12
Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Measured: 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Measured: 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
Percentage of Participants With Transfusion Avoidance Through Week 24
Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 24. Participants who discontinued study treatment early before Week 24 were considered as not achieving transfusion avoidance.
Measured: 24 weeks
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using analysis of covariance (ANCOVA).
Measured: 12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using ANCOVA.
Measured: 12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks)
Change From Baseline FACIT Fatigue Scores at Week 24
The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue and better health-related quality of life. LS mean and SE were produced using MMRM.
Measured: Baseline, Week 24
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
The criterion was defined as Hgb stabilization avoidance of a >1 g/dL (>10 g/L) decrease in Hgb level at Week 24 from Week 12. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb stabilization regardless of the actual value observed at Week 24.
Measured: Week 12 to Week 24
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 24 and remaining transfusion free during the 12-Week TP2. Participants who withdrew from the study early during the 12-Week TP2 or had missing Hgb value at Week 24 were considered as not achieving the criterion.
Measured: Week 24
Change From Baseline in Total and Direct Bilirubin at Week 12
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Measured: Baseline, Week 12
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Measured: Baseline, Week 12
Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Measured: Baseline, Week 12
Change From Baseline in Lactate Dehydrogenase at Week 12
Baseline was defined as the average of all available assessments prior to the first dose of study intervention. LS mean and SE were produced using MMRM.
Measured: Baseline, Week 12
Percentage of Participants With Hgb Normalization at Week 12
Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 12 were considered as not meeting Hgb normalization regardless of actual value observed at Week 12.
Measured: Week 12
Percentage of Participants With Hgb Normalization at Week 24
Hgb normalization was defined as Hgb value above LLN reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb normalization regardless of actual value observed at Week 24.
Measured: Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2020
- Study start16 Dec 2020
- Primary completion29 Jun 2022
- Study completion16 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 86 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
- NCT04469465
- Sponsor's protocol ID
- ALXN2040-PNH-301
- Other IDs
- 2019-003829-18, 2019
- Status last verified
- Apr 2025
- First submitted
- 9 Jul 2020
- Last updated
- 4 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Lee JW, Griffin M, Kim JS, Lee Lee LW, Piatek C, Nishimura JI, Carrillo Infante C, Jain D, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Kulasekararaj AG; ALXN2040-PNH-301 Investigators. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol. 2023 Dec;10(12):e955-e965. doi: 10.1016/S2352-3026(23)00315-0.
PubMed 38030318Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, Schrezenmeier H, Barcellini W, Panse J, Gaya A, Patel Y, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Lee JW. Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH. Blood. 2025 Feb 20;145(8):811-822. doi: 10.1182/blood.2024026299.
PubMed 39700502
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
- ClinicalTrials.gov ID
- NCT04469465
- Acronym / sponsor ID
- ALXN2040-PNH-301
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, C5 inhibitor
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Dec 2020 (actual)
- Primary completion
- 29 Jun 2022 (actual)
- Study completion
- 16 Jan 2024 (actual)
- First posted
- 14 Jul 2020 (actual)
- Results
- Posted 24 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.
Detailed description
This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (eculizumab or ravulizumab).
Participants will be randomized to receive danicopan or placebo, in a 2:1 ratio for 12 weeks (Treatment Period 1) in addition to their C5 inhibitor (eculizumab or ravulizumab) therapy. At Week 12, participants randomized to receive placebo will be switched to danicopan in addition to their C5 inhibitor for an additional 12 weeks (Treatment Period 2) and participants randomized to danicopan will continue on danicopan for an additional 12 weeks, while remaining on their ongoing C5 inhibitor therapy.
At the end of the 2 treatment periods (Week 24), participants may enter a Long-Term Extension (LTE) Period and continue to receive danicopan in addition to their C5 inhibitor therapy. The Long-Term Extension period will consist of a first year of LTE(Year1) and a second year of optional LTE(Year2).All patients will complete 72 weeks of LTE(Year 1) assessments. After Week 72 (at the end of the first year of LTE), patients have the choice to complete participation in this study or continue to the optional second year (Year2) of LTE.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Danicopan + C5 Inhibitor | Experimental | Drug: Danicopan, Drug: C5 InhibitorParticipants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2). |
| Placebo + C5 Inhibitor | Placebo comparator | Drug: Placebo, Drug: C5 InhibitorParticipants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of PNH
- Clinically Evident EVH defined by:
- Receiving an approved C5 inhibitor for at least 6 months prior to Day 1
- Platelet count ≥30,000/microliters (µL)
- Absolute neutrophil counts ≥500/μL
Key exclusion criteria (10 listed)
- History of a major organ transplant or hematopoietic stem cell transplantation (HSCT)
- Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants
- Known or suspected complement deficiency
- Laboratory abnormalities at screening, including:
- Current evidence of biliary cholestasis
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: ALXN2040Oral tablet | Drug | Danicopan + C5 Inhibitor |
| PlaceboOral tablet | Drug | Placebo + C5 Inhibitor |
| C5 InhibitorParticipants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule. | Drug | Danicopan + C5 Inhibitor, Placebo + C5 Inhibitor |
Primary outcome
Change From Baseline in Hgb at Week 12
Time frame: Baseline, Week 12
Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.
18 secondary outcomes
Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
Time frame: Week 12
Percentage of Participants With Transfusion Avoidance Through Week 12
Time frame: Week 12
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Absolute Reticulocyte Count at Week 12
Time frame: Baseline, Week 12
Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Time frame: 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Time frame: 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
Percentage of Participants With Transfusion Avoidance Through Week 24
Time frame: 24 weeks
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
Time frame: 12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
Time frame: 12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks)
Change From Baseline FACIT Fatigue Scores at Week 24
Time frame: Baseline, Week 24
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
Time frame: Week 12 to Week 24
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
Time frame: Week 24
Change From Baseline in Total and Direct Bilirubin at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Lactate Dehydrogenase at Week 12
Time frame: Baseline, Week 12
Percentage of Participants With Hgb Normalization at Week 12
Time frame: Week 12
Percentage of Participants With Hgb Normalization at Week 24
Time frame: Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 38030318Lee JW, Griffin M, Kim JS, Lee Lee LW, Piatek C, Nishimura JI, Carrillo Infante C, Jain D, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Kulasekararaj AG; ALXN2040-PNH-301 Investigators. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol. 2023 Dec;10(12):e955-e965. doi: 10.1016/S2352-3026(23)00315-0.
- Derived from this study: PubMed 39700502Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, Schrezenmeier H, Barcellini W, Panse J, Gaya A, Patel Y, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Lee JW. Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH. Blood. 2025 Feb 20;145(8):811-822. doi: 10.1182/blood.2024026299.
- Danicopan (ACH-4471) in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase 2, Open-label, Proof of Concept Study of the Oral, Small Molecule Factor D InhibitorLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
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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.
62 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Danicopan and C5 Inhibitor in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 62 sites in 18 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 7 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: finished, as of the sponsor's last update on 4 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 62 sites in Japan, United States, Italy, Spain, Brazil and 13 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 24 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Hgb 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?
On the ClinicalTrials.gov record for NCT04469465, which also lists 2 linked publications.
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 4 May 2025.