ClinicalTrials.gov record NCT05731050
Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase II, Open Label, Multi Dose Study of NM8074 in Soliris-Treated Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
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 will evaluate the safety and efficacy of NM8074 in adults with PNH who have been receiving Soliris. Participants will receive NM8074 by intravenous infusion every two weeks for six doses during a 12-week treatment period; total participation is up to 22 weeks including screening and a short washout. The study collects blood tests and other measures to assess hemolysis, anemia, transfusion needs, and complement activity. Enrolment is small (planned at least 6, up to 10 participants).
What it is studying. The study is testing whether giving NM8074 by IV infusion on a schedule of every two weeks (six doses total) is safe and how it affects markers of hemolysis and anemia in adults with PNH who have been treated with Soliris. The protocol specifies that Soliris should not be given on the first day of NM8074 dosing or at any time during the study.
What the researchers hope to learn. Scientific goals: to evaluate the safety profile of NM8074 in this population and to assess measures of hemolysis and anemia. Key outcomes include normalization of lactate dehydrogenase (LDH), changes in total hemoglobin, number and units of packed red blood cell transfusions, and comparison of membrane attack complex (MAC) activity via the alternative versus classical complement pathways. Secondary goals include changes in reticulocyte count, bilirubin levels, and C3b deposition on PNH red blood cells.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.
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
- Male or female patients ≥ 18 years at the time of consent
- Confirmation of PNH diagnosis by flow cytometry evaluation of red blood cells (RBCs) and white blood cells (WBCs), with granulocyte or monocyte clone size of ~5%
- Presence of one or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia: hemoglobin < 10 g/dL, history of a major adverse vascular event (including thrombosis), dysphagia, erectile dysfunction, PNH-mediated pRBC transfusions
- PNH patients must be undergoing treatment with Soliris for at least 3 months prior to screening, and must have a lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (ULN) during Screening
- 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.
- Female subjects of childbearing potential must have a negative pregnancy test at Screening, and must not be planning pregnancy throughout the extent of the study term
- Female subjects of child-bearing potential and all male subjects must agree to use of effective contraception during study
- Soliris treated individuals must be able to provide documentation of vaccination against meningococcal infections.
Exclusion criteria
- Platelet count < 30,000/µL at Screening
- Absolute neutrophil count (ANC) < 500 cells/µL at Screening
- Body weight < 85 lbs. (38 kg) at Screening
- Estimated glomerular filtration rate of < 30 mL/min/1.73m2 based on modification of diet in renal disease (MDRD) equation, creatinine clearance, or CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) at Screening
- Elevation of liver function tests: alanine aminotransferase (ALT) > 2xULN or direct bilirubin and alkaline phosphatase (ALP) both > 2xULN
- Has a known history of meningococcal disease or N. meningitidis infection
- Has an immunological disorder, such as, but not limited to, human immunodeficiency virus (HIV) infection (as evident by HIV-1 or HIV-2 antibody titer) or any acute or chronic infection including, but not limited to, hepatitis B virus (HBV) or hepatitis C virus (HCV)
- 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 bone marrow or solid organ transplantation
- Pregnant, planning to become pregnant, or nursing female subjects
- Recent surgery requiring general anesthesia within the 2 weeks prior to Screening, or expected to have surgery requiring general anesthesia during the 12-week treatment period
- Active malignancy requiring surgery, chemotherapy, or radiation within the prior 12- months (subjects with a history of malignancy who have undergone curative resection or otherwise not requiring treatment for at least 12-months prior to screening with no detectable recurrences are allowed)
- History of any significant major medical conditions (cardiac, pulmonary, renal, e endocrine, or hepatic), or psychiatric disorder that, in the opinion of the Investigator, would make the subject unsuitable for participation in the study
- PNH patients currently under complement blocker treatments other than Soliris
- Concomitant use of anticoagulants is prohibited, if not on a stable regimen for at least 2 weeks prior to Day 1
- 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)
- Known or suspected history of illegal recreational drug or alcohol abuse within 1 year prior to start of screening
- Hypersensitivity or history of allergy to excipients in NM8074 formulation
- Unable or unwilling to comply with the requirements 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-101, will evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects. The drug will be administered by intravenous (IV) infusion to adult patients who qualify based on the inclusion/exclusion criteria. A total of at least 6 patients (maximum of 10) with documented PNH and at least 3 months of Soliris treatment prior to Screening will be enrolled at the study site. The total duration of study term participation for all subjects will be up to 22 weeks, including a screening period of up to 8 weeks, a 12-week treatment period, and 2 weeks of a washout period. All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
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.
After screening (up to 8 weeks) participants receive IV infusions of NM8074 at 15 mg/kg every two weeks for a 12-week treatment period (six doses total), followed by a 2-week washout (total participation up to 22 weeks). Dosing on Day 1 is scheduled to coincide with the participant’s next Soliris dose; Soliris must not be given on Day 1 or at any time thereafter during the study. Each dosing visit includes blood work (performed before dosing) and safety monitoring; there will be multiple site visits for infusions and assessments over the 12 weeks.
How the study is structured. Phase II, open-label (no placebo, no masking), single-group sequential dosing. Planned enrollment is a minimum of 6 and up to 10 adult participants. Timeline per participant: up to 8 weeks screening, a 12-week treatment period with NM8074 infusions every two weeks (Day 1 to Day 84, six doses), and a 2-week washout. Dosing on Day 1 is scheduled to align with the participant's next scheduled Soliris dose, and Soliris is not administered during the study.
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
- June 2026 (estimated)
- Primary completion
- September 2027 (estimated)
- Study completion
- March 2028 (estimated)
- Target enrollment
- 6 participants (estimated)
- Last updated on registry
- Mar 30, 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, PhD 📞 2164402696 ✉ 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.
- childbearing potential
- 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.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- Estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- stable regimen
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- observational
- A study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
- registry
- An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
- 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.
- washout period
- A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
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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