ClinicalTrials.gov record NCT05646524
Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
A Phase II, Open-Label Study of NM8074 in 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 gives intravenous NM8074 to adults with PNH who are treatment‑naïve to complement inhibitors. Two dosing schedules are compared across two small parallel cohorts. The study will collect safety data and blood markers of hemolysis and complement activity over a roughly 13-week treatment period.
What it is studying. The study is testing the safety of IV NM8074 and its effects on hemolysis (for example, hemoglobin and LDH levels), need for blood transfusions, markers of complement activity (including alternative versus classical pathway measures), pharmacokinetics, and whether participants develop antidrug antibodies.
What the researchers hope to learn. To describe the safety profile of NM8074 in adults with PNH. To measure its effects on blood markers of hemolysis and transfusion needs. To assess NM8074 pharmacokinetics and immunogenicity (antidrug antibodies). To evaluate whether NM8074 affects complement alternative pathway activity compared with the classical pathway.
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 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- NM8074
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 of white blood cells (WBCs), with neutrophil, granulocyte and/or monocyte clone size of ≥10%
- Evidence of ongoing hemolysis
- ≥1 packed red blood cell (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
- 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
- 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
- Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1
- 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 (chronic kidney disease (CKD) stage 4, dialysis)
- Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1
- 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 refrain from donating 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 will enroll a planned number of 12 treatment naïve PNH patients with amaximum of 18 PNH patients who have been diagnosed with hemolytic anemia and meet the inclusion criteria. There will be 2 cohorts with 6 to 9 patients each. Patients in Cohort 1 will be administered NM8074 at 20 mg/kg intravenously (IV) every 2 weeks over the treatment period. Cohort 2 patients will be administered a dose of 10 mg/kg NM8074 weekly for 4 weeks followed by a 20 mg/kg dose of NM8074 administered via IV every 2 weeks for the remainder of the treatment period. This study will determine if NM8074 will provide the desired inhibition of the alternative pathway (AP).
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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 receive intravenous (IV) infusions of NM8074 over a 13‑week treatment period (either 20 mg/kg every 2 weeks, or 10 mg/kg weekly for 4 weeks then 20 mg/kg every 2 weeks). They will have regular safety monitoring and blood draws for lab tests (hemoglobin, LDH, complement measures), antidrug antibody testing, and drug concentration (PK) sampling, plus questionnaires about fatigue and quality of life. Vaccination against meningococcus is required before dosing (or prophylactic antibiotics if vaccination timing is short); pregnancy testing and contraception requirements apply. Participants are monitored for adverse events and followed through outcome assessments up to Study Day 105.
How the study is structured. Phase II, interventional (treatment) study that is open-label and not randomized. Participants are assigned to one of two parallel cohorts: Cohort 1 receives 20 mg/kg IV every two weeks; Cohort 2 receives 10 mg/kg IV weekly for four weeks then 20 mg/kg IV every two weeks. Treatments are given by IV infusion over a 13-week treatment period with safety and lab monitoring through Day 105. Planned enrollment is small (about 12, up to 18 patients) and the study measures blood tests, drug levels, and patient-reported quality-of-life questionnaires.
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
- April 2026 (estimated)
- Primary completion
- August 2027 (estimated)
- Study completion
- September 2028 (estimated)
- Target enrollment
- 12 participants (estimated)
- Last updated on registry
- Mar 11, 2025
- 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.
- Cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
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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