Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Launched by NOVELMED THERAPEUTICS · Dec 3, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called NM8074 for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The goal is to see how safe and effective NM8074 is when given through an intravenous (IV) infusion to patients who have not received any prior treatments that target the complement system, which is part of the immune system.

To be eligible for this trial, participants must be at least 18 years old, weigh at least 45 kg, and have a confirmed diagnosis of PNH. They should also have signs of ongoing hemolysis (the breakdown of red blood cells), which can lead to anemia. This means their hemoglobin levels need to be 10.5 g/dL or lower. Participants will need to have received at least one blood transfusion in the past year and must be vaccinated for specific infections before starting the treatment. Throughout the study, participants will regularly visit the clinic to monitor their health and response to the treatment. It’s important to note that certain medical histories or current health conditions might exclude someone from participating, so potential volunteers should discuss their eligibility with their healthcare provider.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patients ≥ 18 years (males and females), weight ≥ 45 kg at the time of consent
• • Confirmation of PNH diagnosis by flow cytometry evaluation of white blood cells (WBCs), with neutrophil, granulocyte and/or monocyte clone size of ≥10%
• • Evidence of ongoing hemolysis
• • ≥1 packed red blood cell (pRBC) transfusion within 12 months prior to screening
• • Anemia (Hemoglobin ≤10.5 g/dL)
• • Lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during Screening
• • All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-135 and MenB meningococcal serogroup B vaccine (Bexsero®). If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics
• • Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and comply with the study visit schedule
• Exclusion Criteria:
• • History of bone marrow, hematopoietic stem cell, or solid organ transplantation
• • History of splenectomy
• • Participation in any other investigational drug trial within 5 elimination half-lives of enrollment, or within 30 days, whichever is longer
• • Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1
• • Participants with known or suspected hereditary or acquired complement deficiency
• • History of currently active primary or secondary immunodeficiency
• • Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections
• • Has a known history of meningococcal disease or N. meningitidis infection
• • Patients on immunosuppressive agents or systemic corticosteroids less than 8 weeks prior to dosing
• • Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study
• • Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (chronic kidney disease (CKD) stage 4, dialysis)
• • Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1
• • Pregnant, planning to become pregnant, or nursing female subjects. Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for 1 week after stopping the investigational drug
• • Females who have a positive pregnancy test result at Screening or on Day 1
• • Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to refrain from donating sperm for the duration of the study