Trial · NCT04170023
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
NCT04170023, Phase 2, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2024-08-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy. Trials, record NCT04170023. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04170023
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
- NCT number
- NCT04170023
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- ALXN2050
- First posted
- 2019-11-20
- Last updated
- 2024-11-20
- Other registries' IDs
- eudract:2019-003830-17
- Results first posted
- 2024-08-09
Source ClinicalTrials.gov
Registered outcomes14
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 | Hgb baseline was defined as the lowest Hgb value observed between and including screening and first dose date. To address the impact of transfusion, Hgb values collected within 4 weeks after transfusion were not included in the primary efficacy analysis. Change from Baseline = Hgb at Week 12 - Baseline Hgb. |
| Secondary | Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050 | Baseline up to Week 12 | Transfusion avoidance: participants remained transfusion-free and did not require a transfusion during the period of interest. |
| Secondary | Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment | Baseline up to Week 12 | |
| Secondary | Number of Transfusion Instances During 12 Weeks of Treatment | Baseline up to Week 12 | |
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12 | Baseline, Week 12 | Change from Baseline = Serum LDH levels at Week 12 - Baseline Serum LDH levels. |
| Secondary | Change From Baseline in Absolute Reticulocyte Count at Week 12 | Baseline, Week 12 | Change from Baseline = absolute reticulocyte count at Week 12 - Baseline reticulocyte count. |
| Secondary | Change From Baseline in Direct and Total Bilirubin at Week 12 | Baseline, Week 12 | |
| Secondary | Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12 | Baseline, Week 12 | The PNH RBC clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 12 - Baseline PNH clone size. |
| Secondary | Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12 | Baseline, Week 12 | C3 fragment deposition on PNH RBC was used as a marker of intra and extravascular hemolysis. Data are presented for the change from baseline to Week 12 in percentage of PNH RBCs with C3 fragment deposition. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication | From first dose of study drug up to Week 217 | An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), or an important medical event or reaction. A TEAE was defined as an AE that emerged during treatment, had been absent prior to treatment, or worsened relative to the pretreatment state. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
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 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | grams/liter | MEAN |
| SECONDARY | Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050 | Baseline up to Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment | Baseline up to Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. | RBC units | MEAN |
| SECONDARY | Number of Transfusion Instances During 12 Weeks of Treatment | Baseline up to Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. | transfusion instances | MEAN |
| SECONDARY | Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12 | Baseline, Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units/liter | MEAN |
| SECONDARY | Change From Baseline in Absolute Reticulocyte Count at Week 12 | Baseline, Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | 10^3 cells/microliter (μL) | MEAN |
| SECONDARY | Change From Baseline in Direct and Total Bilirubin at Week 12 | Baseline, Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for the specified category. | micromoles (μmol)/liter | MEAN |
| SECONDARY | Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12 | Baseline, Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | percentage of the total cell population | MEAN |
| SECONDARY | Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12 | Baseline, Week 12 | Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | percentage of PNH RBC | MEAN |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication | From first dose of study drug up to Week 217 | Safety set included all participants who received at least 1 dose of ALXN2050 in this study. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group51
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 | Group 1: Treatment Naive | 35.6 | Standard Deviation 14.70 | grams/liter | |
| Change From Baseline in Hgb at Week 12 | Group 2: Eculizumab Switch | 32.5 | Standard Deviation 20.03 | grams/liter | |
| Change From Baseline in Hgb at Week 12 | Group 3: Danicopan Rollover | -3.7 | Standard Deviation 15.77 | grams/liter | |
| Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050 | Group 1: Treatment Naive | 9 | Participants | ||
| Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050 | Group 2: Eculizumab Switch | 10 | Participants | ||
| Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050 | Group 3: Danicopan Rollover | 5 | Participants | ||
| Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment | Group 1: Treatment Naive | 0.6 | Standard Deviation 1.24 | RBC units | |
| Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment | Group 2: Eculizumab Switch | 0.1 | Standard Deviation 0.30 | RBC units | |
| Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment | Group 3: Danicopan Rollover | 0.8 | Standard Deviation 2.04 | RBC units | |
| Number of Transfusion Instances During 12 Weeks of Treatment | Group 1: Treatment Naive | 0.4 | Standard Deviation 0.90 | transfusion instances |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period | STARTED | Group 1: Treatment Naive | 12 | |
| Treatment Period | STARTED | Group 2: Eculizumab Switch | 11 | |
| Treatment Period | STARTED | Group 3: Danicopan Rollover | 6 | |
| Treatment Period | Received at Least 1 Dose of Study Drug | Group 1: Treatment Naive | 12 | |
| Treatment Period | Received at Least 1 Dose of Study Drug | Group 2: Eculizumab Switch | 11 | |
| Treatment Period | Received at Least 1 Dose of Study Drug | Group 3: Danicopan Rollover | 6 | |
| Treatment Period | COMPLETED | Group 1: Treatment Naive | 12 | |
| Treatment Period | COMPLETED | Group 2: Eculizumab Switch | 11 | |
| Treatment Period | COMPLETED | Group 3: Danicopan Rollover | 6 | |
| Treatment Period | NOT COMPLETED | Group 1: Treatment Naive | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events591
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 | Anaemia | Blood and lymphatic system disorders | Group 1: Treatment Naive | 1 | 12 | |
| other | Anaemia | Blood and lymphatic system disorders | Group 2: Eculizumab Switch | 2 | 11 | |
| other | Anaemia | Blood and lymphatic system disorders | Group 3: Danicopan Rollover | 0 | 6 | |
| other | Breakthrough haemolysis | Blood and lymphatic system disorders | Group 1: Treatment Naive | 3 | 12 | |
| other | Breakthrough haemolysis | Blood and lymphatic system disorders | Group 2: Eculizumab Switch | 2 | 11 | |
| other | Breakthrough haemolysis | Blood and lymphatic system disorders | Group 3: Danicopan Rollover | 1 | 6 | |
| other | Haemolysis | Blood and lymphatic system disorders | Group 1: Treatment Naive | 0 | 12 | |
| other | Haemolysis | Blood and lymphatic system disorders | Group 2: Eculizumab Switch | 4 | 11 | |
| other | Haemolysis | Blood and lymphatic system disorders | Group 3: Danicopan Rollover | 0 | 6 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Group 1: Treatment Naive | 1 | 12 |
1 to 10 of 591Source ClinicalTrials.gov
See all 591 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Group 1: Treatment Naive | 0 | 12 |
| Total, all-cause mortality | Group 2: Eculizumab Switch | 0 | 11 |
| Total, all-cause mortality | Group 3: Danicopan Rollover | 0 | 6 |
| Total, other adverse events | Group 1: Treatment Naive | 12 | 12 |
| Total, other adverse events | Group 2: Eculizumab Switch | 10 | 11 |
| Total, other adverse events | Group 3: Danicopan Rollover | 6 | 6 |
| Total, serious adverse events | Group 1: Treatment Naive | 7 | 12 |
| Total, serious adverse events | Group 2: Eculizumab Switch | 5 | 11 |
| Total, serious adverse events | Group 3: Danicopan Rollover | 2 | 6 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41124654 | DERIVED |
Source PubMed
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.
For software
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The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04170023
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