ClinicalTrials.gov record NCT05646563
Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris
A Phase II, Open-Label Study of NM8074 in Soliris®-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase II, open-label study evaluates NM8074 in people with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with Soliris. Twelve enrolled patients will be split into two groups: one group will stop Soliris after initial NM8074 doses and continue NM8074 alone, the other will continue Soliris while receiving NM8074. The study collects safety, blood tests, anti-drug antibody, quality-of-life, and drug-level data.
What it is studying. The study is testing the safety, efficacy, and immunogenicity of NM8074 given by intravenous infusion, both as monotherapy and combined with the complement C5 blocker Soliris. Key measurements include adverse events, anti-drug antibodies, hemoglobin and LDH levels, number of packed red blood cell transfusions, complement activity markers (MAC and C3b), quality-of-life surveys, and NM8074 pharmacokinetics (drug blood levels).
What the researchers hope to learn. To evaluate NM8074's safety and side-effect profile, measure immune responses to the drug, describe its blood levels over time (pharmacokinetics), and assess effects on hemolysis markers (hemoglobin, LDH), transfusion needs, and complement activity in PNH patients on Soliris.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- NM8074
- Soliris
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will receive intravenous (IV) NM8074 infusions according to one of two cohort schedules alongside their Soliris treatment: initial weekly NM8074 10 mg/kg for 4 weeks followed by NM8074 20 mg/kg every 2 weeks either with continued Soliris (combination cohort) or after stopping Soliris (switch cohort). The treatment period spans about 13 weeks with safety monitoring through Study Day 105. Study visits will include blood draws for safety labs, hemoglobin and LDH measurements, tests for anti-drug antibodies and drug levels, recording of transfusions, and completion of quality-of-life questionnaires (FACIT‑Fatigue and EORTC QLQ-C30). Participants must meet meningococcal vaccination requirements (or receive prophylactic antibiotics if vaccination timing is short) and follow pregnancy-testing and contraception rules described in the eligibility criteria.
How the study is structured. Phase II, interventional, open-label, non-randomized study with two parallel cohorts. Twelve Soliris-treated PNH patients receive IV NM8074 over a 13-week treatment period: initial 10 mg/kg weekly for 4 weeks, then 20 mg/kg every 2 weeks. Cohort 1 discontinues Soliris after the initial period and continues NM8074 alone; Cohort 2 continues Soliris while receiving NM8074 as combination therapy. Participants are followed for safety and laboratory outcomes through Day 105.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- January 2027 (estimated)
- Primary completion
- July 2029 (estimated)
- Study completion
- August 2030 (estimated)
- Target enrollment
- 12 participants (estimated)
- Last updated on registry
- Apr 13, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- NovelMed Therapeutics
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Rekha Bansal 📞 216-440-2696 ✉ clinicalsae@novelmed.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- investigational drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- corticosteroids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- WOCBP
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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