The trial, sponsored by NovelMed Therapeutics, is targeted to enroll 12 adult treatment-naive patients with PNH. The number of patients could potentially be increased to 18.
Patients will be divided into 2 cohorts to receive slightly different treatment regimens. The first cohort will receive 20 mg/kg intravenous (IV) infusions every 2 weeks for 13 weeks total. The second cohort will receive 10 mg/kg IV infusions weekly for 4 weeks followed by 20 mg/kg infusions every 2 weeks for a total of 13 weeks.
Read more about experimental therapies for PNH.
The study will measure the number of adverse events and serious adverse events experienced by patients over 105 days of the study. The number of patients who develop antidrug antibodies against NM8074 will also be measured.
Additional primary outcomes include changes from baseline in hemoglobin and lactate dehydrogenase levels, the number of packed red blood cell transfusions, and levels of membrane attack complex (MAC) and complement component C3b via the alternative pathway.
Secondary outcomes measured will include changes in reticulocyte count and bilirubin levels, as well as changes in quality of life assessed using the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale (QLQ- C30).
The study is estimated to begin in February 2023 with a primary completion date of February 2024 and full completion in June 2024.
According to the developer, “NM8074 is an anti-Factor Bb humanized monoclonal antibody” designed to inhibit portions of the alternative pathway. It is also being investigated in another clinical trial for PNH in patients being treated with eculizumab (NCT05646563) and also a trial in patients with C3 glomerulopathy (NCT05647811).
Study of NM8074 in adult treatment-naive PNH patients. ClinicalTrials.gov. Accessed December 22, 2022.