Study of NM8074 in Adult PNH Patients with Inadequate Response to Soliris

NCT ID: NCT05646563

Last Updated: 2025-03-11

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-31
• Study Completion Date: 2028-01-31

Brief Summary

This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.

Detailed Description

The proposed study, NM8074-PNH-106, will enroll a planned number of 12 Soliris-treated PNH patients who have been diagnosed with hemolytic anemia and meet the defined inclusion criteria. This study will evaluate the safety, efficacy, and immunogenicity of NM8074 as both a mono- and combination therapy with complement component C5 blocker Soliris. Patients will be evenly divided into two cohorts.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1

6 Soliris-treated patients will receive an intravenous (IV) dose of NM8074 at 10 mg/kg weekly for 4 weeks. Patients will then discontinue Soliris treatment and be administered NM8074 at 20 mg/kg IV every 2 weeks for the remainder of the treatment period (8 weeks). At the end of the treatment period, patients will resume Soliris monotherapy as prescribed.

Group Type: EXPERIMENTAL

NM8074

Intervention Type: DRUG

NM8074 is an anti-Factor Bb humanized monoclonal antibody that will be administered as an intravenous infusion. Doses will be administered over a treatment period of 13 weeks.

Soliris

Intervention Type: DRUG

Complement C5 blocker administered intravenously

Cohort 2

6 Soliris-treated patients will receive an intravenous (IV) dose of NM8074 at 10 mg/kg for 4 weeks. Patients will then continue receiving Soliris while being administered NM8074 as a combination therapy at 20 mg/kg IV every 2 weeks for the remainder of the treatment period (8 weeks). At the end of the treatment period, patients will resume Soliris monotherapy as prescribed.

Group Type: EXPERIMENTAL

NM8074

Intervention Type: DRUG

NM8074 is an anti-Factor Bb humanized monoclonal antibody that will be administered as an intravenous infusion. Doses will be administered over a treatment period of 13 weeks.

Soliris

Intervention Type: DRUG

Complement C5 blocker administered intravenously

Interventions

NM8074

NM8074 is an anti-Factor Bb humanized monoclonal antibody that will be administered as an intravenous infusion. Doses will be administered over a treatment period of 13 weeks.

Intervention Type: DRUG

Soliris

Complement C5 blocker administered intravenously

Intervention Type: DRUG

Other Intervention Names

Eculizumab

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 white blood cells (WBCs), with neutrophil, granulocyte and/or monocyte clone size of ≥10%.
• Evidence of ongoing hemolysis.
• ≥1 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.
• Treatment with Soliris
• 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

• Subjects currently or previously under other complement inhibitor treatments other than Soliris less than 3 months prior to study Day 1
• 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
• 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 (CKD stage 4, dialysis)
• 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 not donate sperm for the duration of the study.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

NovelMed Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Rekha Bansal

Role: CONTACT

clinicalsae@novelmed.com

216-440-2696

Other Identifiers

NM8074-PNH-106

Identifier Type: -

Identifier Source: org_study_id