Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Status: Not yet recruiting
• Trial end date: 2026-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NovelMed Therapeutics

Criteria

Inclusion Criteria:

• Male or female patients ≥ 18 years at the time of consent

• Confirmation of PNH diagnosis by flow cytometry evaluation of red blood cells (RBCs) and white blood cells (WBCs), with granulocyte or monocyte clone size of ~5%

• Presence of one or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia: hemoglobin < 10 g/dL, history of a major adverse vascular event (including thrombosis), dysphagia, erectile dysfunction, PNH-mediated pRBC transfusions

• PNH patients must be undergoing treatment with Soliris for at least 3 months prior to screening, and must have a lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (ULN) during Screening

• 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.

• Female subjects of childbearing potential must have a negative pregnancy test at Screening, and must not be planning pregnancy throughout the extent of the study term

• Female subjects of child-bearing potential and all male subjects must agree to use of effective contraception during study

• Soliris treated individuals must be able to provide documentation of vaccination against meningococcal infections.

Exclusion Criteria:

• Platelet count < 30,000/µL at Screening

• Absolute neutrophil count (ANC) < 500 cells/µL at Screening

• Body weight < 85 lbs. (38 kg) at Screening

• Estimated glomerular filtration rate of < 30 mL/min/1.73m2 based on modification of diet in renal disease (MDRD) equation, creatinine clearance, or CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) at Screening

• Elevation of liver function tests: alanine aminotransferase (ALT) > 2xULN or direct bilirubin and alkaline phosphatase (ALP) both > 2xULN

• Has a known history of meningococcal disease or N. meningitidis infection

• Has an immunological disorder, such as, but not limited to, human immunodeficiency virus (HIV) infection (as evident by HIV-1 or HIV-2 antibody titer) or any acute or chronic infection including, but not limited to, hepatitis B virus (HBV) or hepatitis C virus (HCV)

• Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection that requires antibiotic, antifungal, antiparasitic, or antiviral mediations

• Temperature > 38°C for more than two weeks prior to screening

• History of bone marrow or solid organ transplantation

• Pregnant, planning to become pregnant, or nursing female subjects

• Recent surgery requiring general anesthesia within the 2 weeks prior to Screening, or expected to have surgery requiring general anesthesia during the 12-week treatment period

• Active malignancy requiring surgery, chemotherapy, or radiation within the prior 12- months (subjects with a history of malignancy who have undergone curative resection or otherwise not requiring treatment for at least 12-months prior to screening with no detectable recurrences are allowed)

• History of any significant major medical conditions (cardiac, pulmonary, renal, e endocrine, or hepatic), or psychiatric disorder that, in the opinion of the Investigator, would make the subject unsuitable for participation in the study

• PNH patients currently under complement blocker treatments other than Soliris

• Concomitant use of anticoagulants is prohibited, if not on a stable regimen for at least 2 weeks prior to Day 1

• Participation in any experimental small molecule or non-antibody therapy within 60 days prior to dosing on Day 1 (participation in observational studies and/or registry studies is permitted)

• Known or suspected history of illegal recreational drug or alcohol abuse within 1 year prior to start of screening

• Hypersensitivity or history of allergy to excipients in NM8074 formulation

• Unable or unwilling to comply with the requirements of the study