A Phase 3 interventional study of Danicopan and Placebo in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 62 sites in 18 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-05-04.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 3, Interventional, and Treatment
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.
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.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's enrollment of 86 is above the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Clinically Evident EVH defined by:
Exclusion Criteria:
Laboratory abnormalities at screening, including:
>2 x (>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Drug: Danicopan · Drug: C5 Inhibitor
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).
Drug: Placebo · Drug: C5 Inhibitor
Oral tablet
Also known as: ALXN2040
Oral tablet
Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.
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.
Time frame: Baseline, Week 12
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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, Week 12
Change From Baseline in Absolute Reticulocyte Count at Week 12
LS mean and SE were produced using MMRM.
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
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.
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
LS mean and SE were produced using analysis of covariance (ANCOVA).
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
LS mean and SE were produced using ANCOVA.
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
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.
Time frame: 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.
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
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.
Time frame: 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.
Time frame: 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.
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
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Week 24
| Milestone | Danicopan-Danicopan | Placebo-Danicopan |
|---|---|---|
| Started | 57 | 29 |
| Received at least 1 dose of study drug | 57 | 29 |
| Interim efficacy analysis set | 42 | 21 |
| Completed | 55 | 27 |
| Not completed | 2 | 2 |
| Withdrew: Adverse event | 2 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
| Milestone | Danicopan-Danicopan | Placebo-Danicopan |
|---|---|---|
| Started | 55 | 27 |
| Received at least 1 dose of study drug | 55 | 27 |
| Completed | 54 | 26 |
| Not completed | 1 | 1 |
| Withdrew: Adverse event | 1 | 1 |
| Milestone | Danicopan-Danicopan | Placebo-Danicopan |
|---|---|---|
| Started | 54 | 26 |
| Received at least 1 dose of study drug | 54 | 26 |
| Completed | 46 | 24 |
| Not completed | 8 | 2 |
| Withdrew: Withdrawal by subject | 3 | 0 |
| Withdrew: Physician decision | 3 | 0 |
| Withdrew: Noncompliance with study intervention | 1 | 0 |
| Withdrew: Death | 0 | 1 |
| Withdrew: Adverse event | 1 | 1 |
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.
| g/L | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | 29.40 ± 2.107 | 4.96 ± 3.128 |
| Full Analysis | 28.08 ± 1.957 | 4.62 ± 3.018 |
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.
| percentage of participants | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | 59.5 (43.28 to 74.37) | 0 (0.00 to 16.11) |
| Full Analysis | 54.4 (40.66 to 67.64) | 0 (0.00 to 11.94) |
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.
| percentage of participants | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | 83.3 (68.64 to 93.03) | 38.1 (18.11 to 61.56) |
| Full Analysis | 78.9 (66.11 to 88.62) | 27.6 (12.73 to 47.24) |
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.
| units on a scale | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | 7.97 ± 1.128 | 1.85 ± 1.581 |
| Full Analysis | 8.13 ± 0.919 | 2.35 ± 1.289 |
LS mean and SE were produced using MMRM.
| 10^12 cells/L | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | -0.0838 ± 0.00893 | 0.0035 ± 0.01268 |
| Full Analysis | -0.0925 ± 0.00816 | -0.0008 ± 0.01184 |
| RBC units | Danicopan-Danicopan (TP2) |
|---|---|
| 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 | -2.7 ± 4.86 |
| transfusion instances | Danicopan-Danicopan (TP2) |
|---|---|
| Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment | -1.5 ± 2.41 |
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.
| percentage of participants | Danicopan-Danicopan (TP2) |
|---|---|
| Percentage of Participants With Transfusion Avoidance Through Week 24 | 69.1 (55.19 to 80.86) |
LS mean and SE were produced using analysis of covariance (ANCOVA).
| RBC units | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | -1.48 ± 0.271 | -0.18 ± 0.383 |
| Full Analysis | -1.44 ± 0.212 | -0.14 ± 0.297 |
LS mean and SE were produced using ANCOVA.
| transfusion instances | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | -0.92 ± 0.174 | -0.21 ± 0.246 |
| Full Analysis | -0.91 ± 0.138 | -0.11 ± 0.193 |
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.
| units on a scale | Danicopan-Danicopan | Placebo-Danicopan |
|---|---|---|
| Change From Baseline FACIT Fatigue Scores at Week 24 | 6.21 ± 1.046 | 5.64 ± 1.921 |
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.
| percentage of participants | Danicopan-Danicopan |
|---|---|
| Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan | 58.2 (44.11 to 71.35) |
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.
| percentage of participants | Danicopan-Danicopan |
|---|---|
| Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24 | 41.8 (28.65 to 55.89) |
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
| micromoles/L | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Total Bilirubin (Interim Efficacy Analysis) | -9.77 ± 1.692 | -2.15 ± 2.377 |
| Direct Bilirubin (Interim Efficacy Analysis) | -2.88 ± 0.357 | 0.30 ± 0.503 |
| Total Bilirubin (Full Analysis) | -11.55 ± 1.541 | -1.42 ± 2.172 |
| Direct Bilirubin (Full Analysis) | -2.85 ± 0.317 | 0.17 ± 0.447 |
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.
| percentage of the total cell population | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | 24.60 ± 4.180 | -3.04 ± 5.864 |
| Full Analysis | 26.35 ± 2.369 | -0.18 ± 2.960 |
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
| percentage of the total cell population | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | -15.06 ± 2.824 | 0.89 ± 4.394 |
| Full Analysis | -19.00 ± 1.814 | 0.68 ± 2.690 |
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.
| units/L | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | -23.49 ± 8.287 | -2.92 ± 11.914 |
| Full Analysis | -25.60 ± 7.932 | -16.92 ± 11.380 |
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.
| percentage of participants | Danicopan (TP1) | Placebo (TP1) |
|---|---|---|
| Interim Efficacy Analysis | 28.6 (15.72 to 44.58) | 0 (0.00 to 16.11) |
| Full Analysis | 26.3 (15.54 to 39.66) | 0 (0.00 to 11.94) |
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.
| percentage of participants | Danicopan-Danicopan |
|---|---|
| Percentage of Participants With Hgb Normalization at Week 24 | 20.0 (10.43 to 32.97) |
Collected over Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Danicopan (TP1) | 0/57 (0%) | 3/57 (5.3%) | 43/57 (75.4%) |
| Placebo (TP1) | 0/29 (0%) | 2/29 (6.9%) | 18/29 (62.1%) |
| Danicopan-Danicopan (TP2) | 0/55 (0%) | 3/55 (5.5%) | 40/55 (72.7%) |
| Placebo-Danicopan (TP2) | 0/27 (0%) | 6/27 (22.2%) | 18/27 (66.7%) |
| Danicopan-Danicopan (LTE) | 0/54 (0%) | 7/54 (13%) | 48/54 (88.9%) |
| Placebo-Danicopan (LTE) | 1/26 (3.8%) | 6/26 (23.1%) | 24/26 (92.3%) |
| Event | Danicopan (TP1) | Placebo (TP1) | Danicopan-Danicopan (TP2) | Placebo-Danicopan (TP2) | Danicopan-Danicopan (LTE) | Placebo-Danicopan (LTE) |
|---|---|---|---|---|---|---|
| COVID-19Infections and infestations | 1/57 | 0/29 | 0/55 | 0/27 | 1/54 | 2/26 |
| HaemolysisBlood and lymphatic system disorders | 0/57 | 0/29 | 0/55 | 2/27 | 0/54 | 0/26 |
| Haemorrhagic diathesisBlood and lymphatic system disorders | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| Pericardial effusionCardiac disorders | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| Abdominal pain upperGastrointestinal disorders | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| DiarrhoeaGastrointestinal disorders | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| CystitisInfections and infestations | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| Neutropenic sepsisInfections and infestations | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| PneumoniaInfections and infestations | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| Body temperature increasedInvestigations | 0/57 | 0/29 | 0/55 | 0/27 | 0/54 | 1/26 |
| Event | Danicopan (TP1) | Placebo (TP1) | Danicopan-Danicopan (TP2) | Placebo-Danicopan (TP2) | Danicopan-Danicopan (LTE) | Placebo-Danicopan (LTE) |
|---|---|---|---|---|---|---|
| COVID-19Infections and infestations | 1/57 | 0/29 | 1/55 | 2/27 | 11/54 | 7/26 |
| PyrexiaGeneral disorders | 3/57 | 0/29 | 7/55 | 0/27 | 13/54 | 3/26 |
| ConstipationGastrointestinal disorders | 2/57 | 1/29 | 1/55 | 1/27 | 0/54 | 4/26 |
| AstheniaGeneral disorders | 0/57 | 4/29 | 2/55 | 2/27 | 4/54 | 4/26 |
| HeadacheNervous system disorders | 6/57 | 2/29 | 6/55 | 1/27 | 8/54 | 1/26 |
| NasopharyngitisInfections and infestations | 1/57 | 0/29 | 1/55 | 0/27 | 7/54 | 1/26 |
| ThrombocytopeniaBlood and lymphatic system disorders | 0/57 | 1/29 | 1/55 | 1/27 | 2/54 | 3/26 |
| InsomniaPsychiatric disorders | 1/57 | 3/29 | 1/55 | 0/27 | 1/54 | 3/26 |
| NauseaGastrointestinal disorders | 5/57 | 3/29 | 1/55 | 3/27 | 3/54 | 1/26 |
| DiarrhoeaGastrointestinal disorders | 4/57 | 3/29 | 6/55 | 2/27 | 1/54 | 2/26 |
Full Analysis Set: all enrolled participants who were randomized to either the danicopan or placebo treatment group.
| Age, Continuous(years) | Danicopan-Danicopan | Placebo-Danicopan | Total |
|---|---|---|---|
| Mean | 52.8 ± 17.00 | 52.9 ± 14.34 | 52.8 ± 16.07 |
| Sex: Female, Male(Participants) | Danicopan-Danicopan | Placebo-Danicopan | Total |
|---|---|---|---|
| Female | 34 | 20 | 54 |
| Male | 23 | 9 | 32 |
| Ethnicity (NIH/OMB)(Participants) | Danicopan-Danicopan | Placebo-Danicopan | Total |
|---|---|---|---|
| Hispanic or Latino | 6 | 1 | 7 |
| Not Hispanic or Latino | 46 | 24 | 70 |
| Unknown or Not Reported | 5 | 4 | 9 |
| Race (NIH/OMB)(Participants) | Danicopan-Danicopan | Placebo-Danicopan | Total |
|---|---|---|---|
| American Indian or Alaska Native | 1 | 0 | 1 |
| Asian | 22 | 10 | 32 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 2 | 0 | 2 |
| White | 28 | 14 | 42 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 4 | 5 | 9 |
| Hemoglobin (Hgb)(g/L) | Danicopan-Danicopan | Placebo-Danicopan | Total |
|---|---|---|---|
| Mean | 76.7 ± 9.47 | 78.9 ± 10.11 | 77.5 ± 9.69 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2020 to primary completion in Jun 2022, 18 months.
Results were first posted in Jul 2023, 1 year after primary completion.
Of 35 industry-sponsored phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 41. This study's 18 months is below that.
Counting every sponsor, half of 35 studies reached it within 23 months; a quarter within 15 and three quarters within 41.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Danicopan
Alexion Pharmaceuticals, Inc. →