A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2023-506498-36-00
- Lead Sponsor
- F. Hoffmann-La Roche AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 209
Body weight = 40 kg, Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry evaluation of WBCs, LDH level =2 x ULN at screening (as per local assessment), Platelet count >= 30,000/mm*3 at screening without transfusion support within 7 days of lab testing, ANC > 500/micro L at screening, For female patients of childbearing potential: agreement to remain abstinent or use contraception
Current or previous treatment with a complement inhibitor, History of allogeneic bone marrow transplantation, History of Neisseria meningitidis infection within 6 months prior to screening and up to first study drug administration, History of myelodysplastic syndrome with Revised International Prognostic Scoring System (IPSS-R) prognostic risk categories of intermediate, high and very high, Splenectomy <= 6 months prior to screening, History of or ongoing cryoglobulinemia at screening
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of crovalimab compared to eculizumab based on the non-inferiority assessment of the following co-primary endpoints;Secondary Objective: To evaluate the efficacy of crovalimab compared to eculizumab based on the non-inferiority assessment of the following assessment of the secondary and exploratory efficacy endpoints, To evaluate the overall safety and tolerability of crovalimab compared to eculizumab, To evaluate the pharmacokinetics of crovalimab and eculizumab, To evaluate the immune response to crovalimab, To identify and/or evaluate biomarkers that can potentially provide evidence of crovalimab and eculizumab activity;Primary end point(s): 1. Proportion of patients who achieve transfusion avoidance, 2. Proportion of patients with hemolysis control, measured by LDH <=1.5×
- Secondary Outcome Measures
Name Time Method