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] - Immune System Diseases [C20]
- Registration Number
- EUCTR2019-004931-21-IT
- Lead Sponsor
- F. HOFFMANN - LA ROCHE LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 200
- Age >= 12 years
- 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)
- Vaccination against Neisseria meningitidis < 3 years prior to initiation of study treatment, in accordance with most current local guidelines or standard-of-care as applicable in patients with complement deficiency
- For women of childbearing potential: agreement to remain abstinent or use contraception
Are the trial subjects under 18? yes
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 160
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 36
- Current or previous treatment with a complement inhibitor
- Platelet count < 30000/mm3 at screening
- ANC < 500/microlitre at screening
- History of allogeneic bone marrow transplantation
- History of Neisseria meningitidis infection within 6 months prior to screening and up to first study drug administration
- Pregnant or breastfeeding, or intending to become pregnant during the study or within 6 months after the final dose of study treatment
- Concurrent disease, treatment, procedure, or surgery or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose any additional risk for the patient, or would, in the opinion of the Investigator, preclude the patient’s safe participation in and completion of the study
- Splenectomy <= 6 months prior to screening
- Positive for hepatitis B surface antigen at screening
- Positive for hepatitis C virus antibody at screening (confirmed by detectable viral RNA)
- 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;Secondary Objective: • To evaluate the efficacy of crovalimab compared with eculizumab<br>• To evaluate the overall safety and tolerability of crovalimab compared to eculizumab<br>• To evaluate the pharmacokinetics of crovalimab and eculizumab <br>• To evaluate the immune response to crovalimab<br>• 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 <br>2. Proportion of patients with hemolysis control, measured by LDH <=1.5×ULN<br>;Timepoint(s) of evaluation of this end point: 1. From Baseline to Week 25<br>2. From Week 5 to Week 25
- Secondary Outcome Measures
Name Time Method