A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
- Conditions
- Paroxysmal Nocturnal HemoglobinuriaPNH10038158
- Registration Number
- NL-OMON55171
- Lead Sponsor
- Alexion Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 5
* Diagnosis of PNH
* Clinically Evident EVH defined by:
- Anemia (Hgb *9.5 gram/deciliter) with absolute reticulocyte count * 120 x
10^9/liter.
- At least 1 packed red blood cell or whole blood transfusion within 6 months
prior to the start of the study.
* Receiving a C5 inhibitor for at least 6 months prior to Day 1
* Platelet count *30,000/microliters (*L)
* Absolute neutrophil counts *750/*L.
* Documentation of/or willingness to receive vaccinations and prophylactic
antibiotics as required.
* History of a major organ transplant or hematopoietic stem cell
transplantation (HSCT).
* Known aplastic anemia or other bone marrow failure that requires HSCT or
other therapies including anti-thymocyte globulin and/or immunosuppressants.
* Known or suspected complement deficiency.
* Laboratory abnormalities at screening, including:
- Alanine aminotransferase >2 x ULN.
- Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome.
* Current evidence of biliary cholestasis.
* Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter
squared and/or are on dialysis.
* Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C
infection at screening.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Change From Baseline In Hemoglobin (Hgb)</p><br>
- Secondary Outcome Measures
Name Time Method <p>* Percentage Of Participants With Transfusion Avoidance<br /><br>* Change From Baseline In Functional Assessment Of Chronic Illness Therapy<br /><br>(FACIT) Fatigue Scores; Scoring 0-52<br /><br>* Change From Baseline In Absolute Reticulocyte Count</p><br>