Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

Phase 1

• Conditions: Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH); MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2019-003829-18-FR
• Lead Sponsor: Alexion Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-22
• Last Update Posted: 11 September 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 84

Inclusion Criteria

• 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 (µL)
• Absolute neutrophil counts =750/µL.
• Documentation of/or willingness to receive vaccinations and prophylactic antibiotics as required.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 82
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

• History of a major organ transplant or hematopoietic stem cell transplantation (HSCT).
• Participants with 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:
- Alkaline phosphatase >2 x upper limit of normal (ULN).
- 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified