Phase 2
N=12
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Bottom Line
View on ClinicalTrials.gov: NCT03472885 ↗Enrolled (actual)
12
Serious AEs
39.1%
Results posted
Jun 2021
Primary outcome: Primary: Change From Baseline In Hemoglobin At Week 24 — 7.94; 10.33; 2.39 g/dL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Danicopan (Drug); Eculizumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Sep 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline In Hemoglobin At Week 24 |
7.94; 10.33; 2.39 | — |
| SECONDARY Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment |
4.5; 0.2 | — |
| SECONDARY Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment |
1; 10 | — |
| SECONDARY Change From Baseline In Lactate Dehydrogenase At Week 24 |
244.5; 239.5; -5.0 | — |
| 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 |
2; 7; 4; 6; 1; 1 | — |
Summary
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.
Eligibility Criteria
Key Inclusion Criteria
- Diagnosed with PNH
- Have received at least one red blood cell transfusion within last 12 weeks
- Anemia with adequate reticulocytosis
- Must be on a stable regimen of eculizumab
- Platelet count ≥ 40, 000/microliter without the need for platelet transfusions
- Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae or willingness to receive vaccinations based on local guidelines
- Willingness to receive antibiotic prophylaxis
- Female participants must use highly effective birth control to prevent pregnancy during the clinical trial and for 30 days after their last dose of study drug
- Male participants must use a highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 90 days after the last dose of study drug
Key Exclusion Criteria
- Current evidence of bone marrow failure or aplastic anemia requiring treatment
- History of a major organ transplant or hematopoietic stem cell/marrow transplant
- Received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater
- Documented C5 complement protein mutations
- Known or suspected complement deficiency
- Contraindication to any of the required vaccinations
- Active bacterial infection or clinically significant active viral infection, a body temperature >38°C, or other evidence of infection
- History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
- History of hypersensitivity reactions to commonly used antibacterial agents
Note: Additional inclusion/exclusion criteria may apply, per protocol.
Data sourced from ClinicalTrials.gov (NCT03472885). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.