Study of NM8074 in Adult C3 Glomerulopathy Patients

NovelMed Therapeutics

Status and phase: Begins enrollment in 5 months; Phase 2; Phase 1

Conditions: C3 Glomerulopathy

Treatments: Drug: NM8074

Study type: Interventional

Funder types: Industry

Identifiers

NCT05647811

NM8074-C3G-101

Details and patient eligibility

About

This is a Phase Ib, open-label, dose-escalation study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to patients with C3 Glomerulopathy.

Full description

The proposed study, NM8074-C3G-101, will enroll a planned number of 18 patients, with the potential to enroll more patients. There will be 3 cohorts with 6 patients each dosed at 5, 10, or 20 mg/kg depending on which cohort they are assigned to. Enrollment in the subsequent higher dose level cohort will occur after the previous cohort has been evaluated for safety.

Enrollment: 18 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients ≥ 18 and ≤ 65 years at the time of consent
• Diagnosis of C3 Glomerulopathy as confirmed by C3 nephropathy in biopsy within 12 months prior to enrollment
• Reduced serum C3 levels (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening
• Patients with confirmed proteinuria
• 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
• Women 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 of investigational drug
• Males must agree to use contraceptives and refrain from donating sperm for the duration of the study
• Patients must have documentation of previous vaccination or be willing to be vaccinated prior to dosing with NM8074. All patients will be vaccinated against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae according to the most current Advisory Committee on Immunization Practices (ACIP) recommendations.
• Estimated glomerular filtration (eGFR) rate of ≤ 60 ml/min but ≥ 20 ml/min

Exclusion criteria

• Use of other investigational drugs at the time of enrollment
• Patients with other renal diseases that would interfere with interpretation of the study
• Estimated glomerular filtration rate of ≤ 20 mL/min/1.73m2 based on Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) at Screening
• Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5 times the upper limit of normal (xULN)
• Has a known history of meningococcal disease or N. meningitidis
• 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 renal organ transplantation
• Pregnant, planning to become pregnant, or nursing female subjects
• C3G patients currently under complement blocker treatments
• 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)

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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18 participants in 3 patient groups

Cohort 1

Experimental group

Description:

6 subjects will receive NM8074 at 5 mg/kg weekly.

Treatment:

Drug: NM8074

Cohort 2

Experimental group

Description:

6 subjects will receive NM8074 at 10 mg/kg every 2 weeks.

Treatment:

Drug: NM8074

Cohort 3

Experimental group

Description:

6 subjects will receive NM8074 at 20 mg/kg every 2 weeks.

Treatment:

Drug: NM8074