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Study of NM8074 in Patients with AHUS with Evidence of Ongoing Thrombotic Microangiopathy | Clinical Trials | Clareo Health

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Study of NM8074 in Patients with AHUS with Evidence of Ongoing Thrombotic Microangiopathy

A Phase II, Open-Label Study of NM8074 in Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

NCT05684159•NovelMed Therapeutics

View on ClinicalTrials.gov

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.

Detailed Description

The proposed study, NM8074-aHUS-401,will initially assign six (6) patients per cohort in a 2-cohort trial. In the first cohort, we will evaluate a biweekly dosing regimen whereas in the second cohort, we will evaluate a weekly dose (10 mg/kg) followed by the biweekly dose (20 mg/kg) over a 3-month period. These studies will determine if NM8074 results in remission from TMA in aHUS patients. If the study shows efficacy in aHUS, additional patients may be added per cohort.

Eligibility

Age

18 - 65 years

Sex

ALL

Healthy Volunteers

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 Haptoglobin \<LLN or undetectable 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.
+1 more criteria

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
+5 more criteria

Key Dates

Start Date

July 1, 2025

Primary Completion Date

December 1, 2025

Completion Date

February 1, 2027

Last Updated

November 18, 2024

Enrollment

ESTIMATED participants

Conditions

AHUS - Atypical Hemolytic Uremic Syndrome

Interventions

NM8074

DRUG

Contacts

Rekha Bansal, PhD

CONTACT

2164402696 clinicalsae@novelmed.com