A Phase 2 interventional study of Danicopan and Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals, Inc.. Completed at 5 sites in 3 countries. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2023-12-12.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 2, Interventional, and Treatment
To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH. Danicopan dose may be increased within each participant, to a maximum of 200 milligrams (mg) three times daily (TID) based on safety and efficacy at protocol-specified time points.
The purpose of this study is to determine the effectiveness of danicopan in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a drug commonly used for treatment of PNH) for 24 weeks in participants with PNH.
The 24-week treatment period was followed by a long-term extension phase. In the extension phase, participants received the same danicopan dose plus eculizumab as they were receiving at the end of 24-week treatment phase.
Results are reported for the 24-week treatment period.
192 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's enrollment of 12 is below the median of 34 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; 24 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 73 (72%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Additional inclusion/exclusion criteria may apply, per protocol.
Starting dose of 100 mg danicopan TID in combination with eculizumab.
Drug: Danicopan · Drug: Eculizumab
Starting dose of 100 or 150 mg danicopan TID in combination with eculizumab.
Drug: Danicopan · Drug: Eculizumab
Starting dose of 100, 150, or 200 mg danicopan TID in combination with eculizumab.
Drug: Danicopan · Drug: Eculizumab
Optimal dose (starting dose of either 100, 150, or 200 mg, as determined from Groups 1-3) of danicopan TID in combination with eculizumab.
Drug: Danicopan · Drug: Eculizumab
Participants received a daily oral dose of danicopan TID during the treatment period.
Participants received intravenous eculizumab administered at the participant's usual dose and schedule.
Also known as: Soliris
Change From Baseline In Hemoglobin At Week 24
Time frame: Baseline, Week 24
Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment
Time frame: Within 24 weeks prior to first dose and during 24-week treatment period
Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment
Time frame: Within 24 weeks prior to first dose and during 24-week treatment period
Change From Baseline In Lactate Dehydrogenase At Week 24
Time frame: Baseline, Week 24
Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug
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.
Time frame: Day 1 (after dosing) through end of study (maximum exposure: 1631 days)
| Milestone | Danicopan + Eculizumab |
|---|---|
| Started | 12 |
| Safety population: received at least 1 dose of study drug | 12 |
| Efficacy population: received study drug for at least 4 weeks | 11 |
| Completed | 11 |
| Not completed | 1 |
| Withdrew: Adverse event | 1 |
| Milestone | Danicopan + Eculizumab |
|---|---|
| Started | 11 |
| Completed | 11 |
| Not completed | 0 |
| g/dL | Danicopan + Eculizumab |
|---|---|
| Baseline | 7.94 ± 1.425 |
| Week 24 | 10.33 ± 1.661 |
| Change from Baseline | 2.39 ± 1.333 |
| RBC units | Danicopan + Eculizumab |
|---|---|
| Within 24 Weeks Prior to First Dose | 4.5 ± 3.96 |
| During 24-Week Treatment Period | 0.2 ± 0.60 |
| Participants | Danicopan + Eculizumab |
|---|---|
| Within 24 Weeks Prior to First Dose | 1 |
| During 24-Week Treatment Period | 10 |
| IU/L | Danicopan + Eculizumab |
|---|---|
| Baseline | 244.5 ± 74.40 |
| Week 24 | 239.5 ± 48.48 |
| Change from Baseline | -5.0 ± 48.60 |
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.
| Participants | 24-Week Treatment Period: Danicopan + Eculizumab | LTE Period: Danicopan + Eculizumab |
|---|---|---|
| SAEs | 2 | 7 |
| Grade 3 AEs | 4 | 6 |
| Grade 4 AEs | 1 | 1 |
| AEs Leading to Discontinuation of Study Drug | 1 | 0 |
Collected over Day 1 (after dosing) through end of study (maximum exposure: 1631 days). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 24-Week Treatment Period: Danicopan + Eculizumab | 0/12 (0%) | 2/12 (16.7%) | 10/12 (83.3%) |
| LTE Period: Danicopan + Eculizumab | 0/11 (0%) | 7/11 (63.6%) | 11/11 (100%) |
| Event | 24-Week Treatment Period: Danicopan + Eculizumab | LTE Period: Danicopan + Eculizumab |
|---|---|---|
| Device related infectionInfections and infestations | 0/12 | 1/11 |
| PyelonephritisInfections and infestations | 0/12 | 1/11 |
| Tracheobronchitis viralInfections and infestations | 0/12 | 1/11 |
| Influenza like illnessGeneral disorders | 0/12 | 1/11 |
| PyrexiaGeneral disorders | 0/12 | 1/11 |
| Febrile neutropeniaBlood and lymphatic system disorders | 0/12 | 1/11 |
| HaemolysisBlood and lymphatic system disorders | 0/12 | 1/11 |
| PancreatitisGastrointestinal disorders | 0/12 | 1/11 |
| SchwannomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/12 | 1/11 |
| HaemoglobinuriaRenal and urinary disorders | 0/12 | 1/11 |
| Event | 24-Week Treatment Period: Danicopan + Eculizumab | LTE Period: Danicopan + Eculizumab |
|---|---|---|
| COVID-19Infections and infestations | 0/12 | 6/11 |
| Upper respiratory tract infectionInfections and infestations | 5/12 | 5/11 |
| HeadacheNervous system disorders | 3/12 | 5/11 |
| PyrexiaGeneral disorders | 1/12 | 4/11 |
| FatigueGeneral disorders | 2/12 | 3/11 |
| NasopharyngitisInfections and infestations | 0/12 | 2/11 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 2/12 | 2/11 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 2/12 | 2/11 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/12 | 2/11 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 1/12 | 2/11 |
Safety Population: Received at least 1 dose of study drug.
| Age, Continuous(years) | Danicopan + Eculizumab |
|---|---|
| Mean | 45.59 ± 16.385 |
| Sex: Female, Male(Participants) | Danicopan + Eculizumab |
|---|---|
| Female | 10 |
| Male | 2 |
| Ethnicity (NIH/OMB)(Participants) | Danicopan + Eculizumab |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 12 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Danicopan + Eculizumab |
|---|---|
| White | 7 |
| Asian | 1 |
| Black or African American | 3 |
| Other | 1 |
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Studies of the drugs tested here: Danicopan · Eculizumab
Alexion Pharmaceuticals, Inc. →