A new phase 2, multidose, open-label study will investigate the safety and efficacy of NM8074 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were previously treated with eculizumab (Soliris®). The study is expected to start in June 2025 and is not yet recruiting.
Six to 10 eligible participants will be recruited during the initial screening period of 8 weeks. Each of them will receive 6 biweekly intravenous infusions of 15 mg/kg NM8074 during the 12-week treatment period, followed by a 2-week washout period.
According to the current study protocol, 5 primary outcome measures will be evaluated. The researchers will compare baseline and after-treatment levels of hemoglobin and LDH, as well as the indicators of complement activity and blood transfusions.
Moreover, they will evaluate markers of anemia including reticulocyte count and bilirubin levels and changes of C3b deposition on PNH type 2 and 3 red blood cells as secondary outcome measures.
Read more about PNH treatment
Eligible participants must fulfill the following inclusion criteria: confirmed PNH diagnosis with at least 1 of the relevant PNH-related signs or symptoms within 3 months of screening, eculizumab treatment for at least 3 months prior to screening, and LDH level of at least 1.5 times the upper limit of normal during screening, as well as proof of vaccination against meningococcal infections.
Female patients of childbearing potential must declare to not be planning pregnancy while part of the study and submit a negative pregnancy test during screening. All male and eligible female participants must agree to use a highly effective form of contraception.
In addition, patients might be excluded from the study based on their blood cell count, liver or kidney function, history of past meningococcal disease, immunological disorders, surgery under general anesthesia, bone marrow or solid organ transplantation, currently active systemic infections, pregnancy, or malignancy.
Individuals who are currently on complement blocker treatments other than eculizumab, as well as those who concomitantly use anticoagulants, will not be considered eligible.
The trial is sponsored by NovelMed Therapeutics.
Study of NM8074 in Soliris-treated patients with paroxysmal nocturnal hemoglobinuria (PNH). ClinicalTrials.gov. February 16, 2023. Updated February 16, 2023. Accessed February 21, 2023.