Trial · NCT04469465
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
NCT04469465, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2023-07-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA). Trials, record NCT04469465. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04469465
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
- NCT number
- NCT04469465
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 86 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Danicopan, Placebo, C5 Inhibitor
- First posted
- 2020-07-14
- Last updated
- 2025-05-04
- Other registries' IDs
- eudract:2019-003829-18
- Results first posted
- 2023-07-24
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Hgb at Week 12 | 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. |
| Secondary | 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 | 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. |
| Secondary | Percentage of Participants With Transfusion Avoidance Through Week 12 | 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. |
| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12 | Baseline, 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. |
| Secondary | Change From Baseline in Absolute Reticulocyte Count at Week 12 | Baseline, Week 12 | LS mean and SE were produced using MMRM. |
| Secondary | 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 | 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment | |
| Secondary | Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment | 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment | |
| Secondary | Percentage of Participants With Transfusion Avoidance Through Week 24 | 24 weeks | 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. |
| Secondary | Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment | 12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment | LS mean and SE were produced using analysis of covariance (ANCOVA). |
| Secondary | Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment | 12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks) | LS mean and SE were produced using ANCOVA. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Hgb at Week 12 | Baseline, Week 12 | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | g/L | LEAST_SQUARES_MEAN |
| SECONDARY | 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 | Week 12 | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Transfusion Avoidance Through Week 12 | Week 12 | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12 | Baseline, Week 12 | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Absolute Reticulocyte Count at Week 12 | Baseline, Week 12 | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure. | 10^12 cells/L | LEAST_SQUARES_MEAN |
| SECONDARY | 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 | 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment | Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure. | RBC units | MEAN |
| SECONDARY | Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment | 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment | Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure. | transfusion instances | MEAN |
| SECONDARY | Percentage of Participants With Transfusion Avoidance Through Week 24 | 24 weeks | Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment | 12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. | RBC units | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment | 12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks) | Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. | transfusion instances | LEAST_SQUARES_MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Hgb at Week 12 | Danicopan (TP1) | Interim Efficacy Analysis | 29.40 | Standard Error 2.107 | g/L |
| Change From Baseline in Hgb at Week 12 | Placebo (TP1) | Interim Efficacy Analysis | 4.96 | Standard Error 3.128 | g/L |
| Change From Baseline in Hgb at Week 12 | Danicopan (TP1) | Full Analysis | 28.08 | Standard Error 1.957 | g/L |
| Change From Baseline in Hgb at Week 12 | Placebo (TP1) | Full Analysis | 4.62 | Standard Error 3.018 | g/L |
| 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 | Danicopan (TP1) | Interim Efficacy Analysis | 59.5 | 95% Confidence Interval 43.28 to 74.37 | percentage of participants |
| 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 | Placebo (TP1) | Interim Efficacy Analysis | 0 | 95% Confidence Interval 0.00 to 16.11 | percentage of participants |
| 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 | Danicopan (TP1) | Full Analysis | 54.4 | 95% Confidence Interval 40.66 to 67.64 | percentage of participants |
| 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 | Placebo (TP1) | Full Analysis | 0 | 95% Confidence Interval 0.00 to 11.94 | percentage of participants |
| Percentage of Participants With Transfusion Avoidance Through Week 12 | Danicopan (TP1) | Interim Efficacy Analysis | 83.3 | 95% Confidence Interval 68.64 to 93.03 | percentage of participants |
| Percentage of Participants With Transfusion Avoidance Through Week 12 | Placebo (TP1) | Interim Efficacy Analysis | 38.1 | 95% Confidence Interval 18.11 to 61.56 | percentage of participants |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow26
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period 1 (TP1) | STARTED | Danicopan-Danicopan | 57 | |
| Treatment Period 1 (TP1) | STARTED | Placebo-Danicopan | 29 | |
| Treatment Period 1 (TP1) | Received at Least 1 Dose of Study Drug | Danicopan-Danicopan | 57 | |
| Treatment Period 1 (TP1) | Received at Least 1 Dose of Study Drug | Placebo-Danicopan | 29 | |
| Treatment Period 1 (TP1) | Interim Efficacy Analysis Set | Danicopan-Danicopan | 42 | |
| Treatment Period 1 (TP1) | Interim Efficacy Analysis Set | Placebo-Danicopan | 21 | |
| Treatment Period 1 (TP1) | COMPLETED | Danicopan-Danicopan | 55 | |
| Treatment Period 1 (TP1) | COMPLETED | Placebo-Danicopan | 27 | |
| Treatment Period 1 (TP1) | NOT COMPLETED | Danicopan-Danicopan | 2 | |
| Treatment Period 1 (TP1) | NOT COMPLETED | Placebo-Danicopan | 2 |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 participant flowAdverse events432
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Haemolysis | Blood and lymphatic system disorders | Danicopan (TP1) | 2 | 57 | 2 |
| other | Haemolysis | Blood and lymphatic system disorders | Placebo (TP1) | 0 | 29 | 0 |
| other | Haemolysis | Blood and lymphatic system disorders | Danicopan-Danicopan (TP2) | 0 | 55 | 0 |
| other | Haemolysis | Blood and lymphatic system disorders | Placebo-Danicopan (TP2) | 0 | 27 | 0 |
| other | Haemolysis | Blood and lymphatic system disorders | Danicopan-Danicopan (LTE) | 2 | 54 | 2 |
| other | Haemolysis | Blood and lymphatic system disorders | Placebo-Danicopan (LTE) | 2 | 26 | 4 |
| other | Neutropenia | Blood and lymphatic system disorders | Danicopan (TP1) | 2 | 57 | 2 |
| other | Neutropenia | Blood and lymphatic system disorders | Placebo (TP1) | 0 | 29 | 0 |
| other | Neutropenia | Blood and lymphatic system disorders | Danicopan-Danicopan (TP2) | 3 | 55 | 3 |
| other | Neutropenia | Blood and lymphatic system disorders | Placebo-Danicopan (TP2) | 1 | 27 | 1 |
1 to 10 of 432Source ClinicalTrials.gov
See all 432 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Danicopan (TP1) | 0 | 57 |
| Total, all-cause mortality | Placebo (TP1) | 0 | 29 |
| Total, all-cause mortality | Danicopan-Danicopan (TP2) | 0 | 55 |
| Total, all-cause mortality | Placebo-Danicopan (TP2) | 0 | 27 |
| Total, all-cause mortality | Danicopan-Danicopan (LTE) | 0 | 54 |
| Total, all-cause mortality | Placebo-Danicopan (LTE) | 1 | 26 |
| Total, other adverse events | Danicopan (TP1) | 43 | 57 |
| Total, other adverse events | Placebo (TP1) | 18 | 29 |
| Total, other adverse events | Danicopan-Danicopan (TP2) | 40 | 55 |
| Total, other adverse events | Placebo-Danicopan (TP2) | 18 | 27 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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