Trial · NCT03472885
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
NCT03472885, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2021-06-25
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab. Trials, record NCT03472885. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03472885
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
- NCT number
- NCT03472885
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Danicopan, Eculizumab
- First posted
- 2018-03-21
- Last updated
- 2023-12-12
- Other registries' IDs
- eudract:2016-003526-16
- Results first posted
- 2021-06-25
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline In Hemoglobin At Week 24 | Baseline, Week 24 | |
| Secondary | Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment | Within 24 weeks prior to first dose and during 24-week treatment period | |
| Secondary | Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment | Within 24 weeks prior to first dose and during 24-week treatment period | |
| Secondary | Change From Baseline In Lactate Dehydrogenase At Week 24 | Baseline, Week 24 | |
| Secondary | Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug | Day 1 (after dosing) through end of study (maximum exposure: 1631 days) | An AE was defined 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, 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), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module. |
Source ClinicalTrials.gov
Outcomes5
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 Hemoglobin At Week 24 | Baseline, Week 24 | Efficacy Population: All treated participants who received at least 4 weeks of danicopan. | g/dL | MEAN |
| SECONDARY | Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment | Within 24 weeks prior to first dose and during 24-week treatment period | Efficacy Population: All treated participants who received at least 4 weeks of danicopan. | RBC units | MEAN |
| SECONDARY | Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment | Within 24 weeks prior to first dose and during 24-week treatment period | Efficacy Population: All treated participants who received at least 4 weeks of danicopan. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline In Lactate Dehydrogenase At Week 24 | Baseline, Week 24 | Efficacy Population: All treated participants who received at least 4 weeks of danicopan. | IU/L | MEAN |
| SECONDARY | Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug | Day 1 (after dosing) through end of study (maximum exposure: 1631 days) | Participants who received at least 1 dose of danicopan were included in the safety assessment. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group18
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 Hemoglobin At Week 24 | Danicopan + Eculizumab | Baseline | 7.94 | Standard Deviation 1.425 | g/dL |
| Change From Baseline In Hemoglobin At Week 24 | Danicopan + Eculizumab | Week 24 | 10.33 | Standard Deviation 1.661 | g/dL |
| Change From Baseline In Hemoglobin At Week 24 | Danicopan + Eculizumab | Change from Baseline | 2.39 | Standard Deviation 1.333 | g/dL |
| Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment | Danicopan + Eculizumab | Within 24 Weeks Prior to First Dose | 4.5 | Standard Deviation 3.96 | RBC units |
| Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment | Danicopan + Eculizumab | During 24-Week Treatment Period | 0.2 | Standard Deviation 0.60 | RBC units |
| Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment | Danicopan + Eculizumab | Within 24 Weeks Prior to First Dose | 1 | Participants | |
| Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment | Danicopan + Eculizumab | During 24-Week Treatment Period | 10 | Participants | |
| Change From Baseline In Lactate Dehydrogenase At Week 24 | Danicopan + Eculizumab | Baseline | 244.5 | Standard Deviation 74.40 | IU/L |
| Change From Baseline In Lactate Dehydrogenase At Week 24 | Danicopan + Eculizumab | Week 24 | 239.5 | Standard Deviation 48.48 | IU/L |
| Change From Baseline In Lactate Dehydrogenase At Week 24 | Danicopan + Eculizumab | Change from Baseline | -5.0 | Standard Deviation 48.60 | IU/L |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| 24-Week Treatment Period | STARTED | Danicopan + Eculizumab | 12 | |
| 24-Week Treatment Period | Safety Population: Received at Least 1 Dose of Study Drug | Danicopan + Eculizumab | 12 | |
| 24-Week Treatment Period | Efficacy Population: Received Study Drug for at Least 4 Weeks | Danicopan + Eculizumab | 11 | |
| 24-Week Treatment Period | COMPLETED | Danicopan + Eculizumab | 11 | |
| 24-Week Treatment Period | NOT COMPLETED | Danicopan + Eculizumab | 1 | |
| LTE Period (Maximum Exposure: 1463 Days) | STARTED | Danicopan + Eculizumab | 11 | |
| LTE Period (Maximum Exposure: 1463 Days) | COMPLETED | Danicopan + Eculizumab | 11 | |
| LTE Period (Maximum Exposure: 1463 Days) | NOT COMPLETED | Danicopan + Eculizumab | 0 |
Source ClinicalTrials.gov
Adverse events228
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 | 24-Week Treatment Period: Danicopan + Eculizumab | 1 | 12 | |
| other | Anaemia | Blood and lymphatic system disorders | LTE Period: Danicopan + Eculizumab | 0 | 11 | |
| other | Neutropenia | Blood and lymphatic system disorders | 24-Week Treatment Period: Danicopan + Eculizumab | 2 | 12 | |
| other | Neutropenia | Blood and lymphatic system disorders | LTE Period: Danicopan + Eculizumab | 1 | 11 | |
| other | Palpitations | Cardiac disorders | 24-Week Treatment Period: Danicopan + Eculizumab | 1 | 12 | |
| other | Palpitations | Cardiac disorders | LTE Period: Danicopan + Eculizumab | 0 | 11 | |
| other | Sinus tachycardia | Cardiac disorders | 24-Week Treatment Period: Danicopan + Eculizumab | 1 | 12 | |
| other | Sinus tachycardia | Cardiac disorders | LTE Period: Danicopan + Eculizumab | 0 | 11 | |
| other | Periorbital swelling | Eye disorders | 24-Week Treatment Period: Danicopan + Eculizumab | 1 | 12 | |
| other | Periorbital swelling | Eye disorders | LTE Period: Danicopan + Eculizumab | 0 | 11 |
1 to 10 of 228Source ClinicalTrials.gov
See all 228 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 24-Week Treatment Period: Danicopan + Eculizumab | 0 | 12 |
| Total, all-cause mortality | LTE Period: Danicopan + Eculizumab | 0 | 11 |
| Total, other adverse events | 24-Week Treatment Period: Danicopan + Eculizumab | 10 | 12 |
| Total, other adverse events | LTE Period: Danicopan + Eculizumab | 11 | 11 |
| Total, serious adverse events | 24-Week Treatment Period: Danicopan + Eculizumab | 2 | 12 |
| Total, serious adverse events | LTE Period: Danicopan + Eculizumab | 7 | 11 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34314483 | 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| 24-Week Treatment Period: Danicopan + Eculizumab vs LTE Period: Danicopan + Eculizumab: deaths from any cause | Trial comparisons |
| 24-Week Treatment Period: Danicopan + Eculizumab vs LTE Period: Danicopan + Eculizumab: serious adverse events | Trial comparisons |
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
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03472885
GET https://conductscience.org/api/trials/v1/NCT03472885/events?page=2