Phase 2
N=10
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Bottom Line
View on ClinicalTrials.gov: NCT03053102 ↗Enrolled (actual)
10
Serious AEs
10.0%
Results posted
Jun 2021
Primary outcome: Primary: Change From Baseline In Serum LDH Levels At Day 28 — 1416; 444.3; -971.7 international units per liter (IU/L)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Danicopan (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Nov 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline In Serum LDH Levels At Day 28 |
1416; 444.3; -971.7 | — |
| SECONDARY Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84 |
9.76; 10.94; 1.18; 11.45; 1.80 | — |
| SECONDARY Change From Baseline In Serum LDH Levels At Day 84 |
1416; 537.3; -865.9 | — |
| SECONDARY Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size |
31.5363; 43.6279; 56.2953 | — |
| SECONDARY Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation |
1; 1; 1; 1 | — |
| SECONDARY Grade 3 And Grade 4 Laboratory Abnormalities |
1; 1; 1; 4; 1 | — |
| SECONDARY Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8) |
1432.611; 2370.421; 2278.038 | — |
| SECONDARY PK: Maximum Plasma Concentration (Cmax) |
347.707; 583.626; 516.8788 | — |
| SECONDARY PK: Time To Maximum Concentration (Tmax) |
4.17; 3.67; 4.11 | — |
| SECONDARY Complement Alternative Pathway (AP) Functional Activity |
65.216; 12.730 | — |
| SECONDARY Complement Bb |
2.24160; 0.83913 | — |
Summary
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
Eligibility Criteria
Inclusion Criteria
- Currently untreated PNH participants with PNH Type III erythrocyte and/or granulocyte clone size ≥10% and anemia (hemoglobin 38°Celsius, or other evidence of infection on Day 1, or with a history of febrile illness within 14 days prior to first study drug administration.
- History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection.
- Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.
Data sourced from ClinicalTrials.gov (NCT03053102). 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.