Study of NM8074 in Patients With aHUS

• Status: Not yet recruiting
• Trial end date: 2025-10-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase II, open-label study designed to determine if intravenously administered NM8074 results in remission from TMA in treatment-naïve aHUS patients.

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

Details

• Lead Sponsor: NovelMed Therapeutics

Criteria

Inclusion Criteria:

• Patients ≥ 18 years at the time of consent

• Patients with evidence of resistant or relapsed complement-mediated aHUS with symptoms of Thrombocytopenia, hemolysis, ongoing Thrombotic Microangiopathy and acute kidney injury.

• Evidence of ongoing Thrombotic Microangiopathy which includes Haptoglobinundetectable and/or presence of schistocytes

• Acute kidney injury (proteinuria/creatinuria > ULN and/or reduced eGFR)

• Platelets less than 150,000 per microliter (Thrombocytopenia)

• Anemia (Hemoglobin ≤10 g/dL) due to hemolysis

• 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.

• 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.

• 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 month after stopping the investigational drug.

Exclusion Criteria:

• History of bone marrow, hematopoietic stem cell, or solid organ transplantation

• Treatment with complement blockers

• Patients with infections

• HUS due to ADAMTS-13 deficiency (<5%)

• Kidney disease other than aHUS

• Chronic dialysis (hemo or peritoneal)

• Liver disease or other major autoimmune diseases

• Typical HUS (Shiga toxin +)

• Known Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, or antiphospholipid antibody positivity or syndrome

• 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 currently active or known history of meningococcal disease or N. meningitidis infection

• Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, chronic dialysis)

• Females who have a positive pregnancy test result at Screening or on Day 1.

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