RAVULIZUMAB for Paroxysmal nocturnal hemoglobinuria (PNH)

Inclusion criteria

• {"criterion_text":"- The patient is eligible for treatment with ravulizumab in line with relevant Dutch guidelines or is already being treated with ravulizumab according to the label and Dutch guidelines.\n- Willing to provide written informed consent\n- 16 years or older"}

Exclusion criteria

• {"criterion_text":"- Patients with thrombosis during treatment with complement inhibitors in the medical history\n- Patients with other concomitant hematological diseases, with the exception for aplastic anemia/PNH\n- Unstable medical conditions (e.g. myocardial infarction, bleeding)\n- Pregnant or lactating patients\n- An LDH >1.5x ULN during the 8 weeks prior to screening for inclusion\n- Use of high dose corticosteroids(defined as >30mg prednisolone equivalent daily during the 8 weeks prior to the screening for inclusion)"}

Primary endpoints

• {"endpoint_text":"- Percentage of change in LDH compared to baseline LDH between standard dosing and individualized dosing","definition_or_measurement_approach":"Change in lactate dehydrogenase (LDH) level compared to baseline LDH between treatment arms (LDH measurement vs baseline)."}
• {"endpoint_text":"- Difference in percentage target attainment (Ravulizumab concentration >100 mg/L) between the standard dosing regimen and the individualized dosing regimen","definition_or_measurement_approach":"Proportion of patients achieving ravulizumab concentration >100 mg/L (measured ravulizumab serum concentration), comparison between regimens."}

Secondary endpoints

• {"endpoint_text":"- The proportion of patients with breakthrough hemolysis, defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2× the ULN after prior reduction of LDH to <1.5× the ULN on treatment","definition_or_measurement_approach":"Breakthrough hemolysis defined as ≥1 new/worsening symptom or sign listed above in presence of LDH ≥2× ULN after prior reduction to <1.5× ULN."}
• {"endpoint_text":"- Quality of life assessed with the FACIT-Fatigue Scale and patient preference","definition_or_measurement_approach":"Quality of life measured using the FACIT-Fatigue Scale; patient preference captured by study-specific preference measures."}
• {"endpoint_text":"- Differences between number of transfusions between both dosing strategies","definition_or_measurement_approach":"Number of transfusions per patient compared between dosing strategies (count of transfusion events)."}
• {"endpoint_text":"- Differences in cumulative ravulizumab dose over 52 weeks, number of infusions and appointments with the physician as surrogate marker for treatment costs.","definition_or_measurement_approach":"Cumulative ravulizumab dose over 52 weeks (mg), count of infusions and physician appointments used as surrogate markers for treatment costs."}

Netherlands

Earliest CTIS Part Ii Submission Date

03-02-2025

Latest Decision Or Authorization Date

05-01-2026

Processing Time Days

336

Number Of Sites

3

Number Of Participants

25

Primary sponsor

Full Name

Radboud universitair medisch centrum Stichting

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands