ClinicalTrials.gov record NCT05684159
Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy
A Phase II, Open-Label Study of NM8074 in Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
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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 aHUS who have evidence of active thrombotic microangiopathy (TMA). Two small, sequential dose cohorts (about six patients each) receive different NM8074 schedules over roughly 3 months. The study measures blood and kidney markers and quality-of-life to assess remission from TMA.
What it is studying. The study is testing whether intravenous NM8074 leads to remission of thrombotic microangiopathy (TMA) in adults with atypical hemolytic uremic syndrome (aHUS) who have ongoing TMA, using blood and kidney measures and symptom/quality-of-life questionnaires.
What the researchers hope to learn. To determine whether NM8074 can induce remission from TMA in aHUS, assessed by normalization of platelet count, lactate dehydrogenase (LDH), schistocyte levels, changes in kidney function (serum creatinine and eGFR), haptoglobin and hemoglobin, proteinuria, time to TMA response, dialysis requirement, and patient-reported quality-of-life measures.
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 determine if intravenously administered NM8074 results in remission from TMA in treatment-naïve aHUS patients.
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 at the time of consent
- Patients with evidence of resistant or relapsed complement-mediated aHUS with symptoms of Thrombocytopenia, hemolysis, ongoing Thrombotic Microangiopathy and acute kidney injury.
- Evidence of ongoing Thrombotic Microangiopathy which includes Haptoglobin <LLN or undetectable and/or presence of schistocytes
- Acute kidney injury (proteinuria/creatinuria > ULN and/or reduced eGFR)
- Platelets less than 150,000 per microliter (Thrombocytopenia)
- Anemia (Hemoglobin ≤10 g/dL) due to hemolysis
- 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.
- 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.
- 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 month after stopping the investigational drug.
Exclusion criteria
- History of bone marrow, hematopoietic stem cell, or solid organ transplantation
- Treatment with complement blockers
- Patients with infections
- HUS due to ADAMTS-13 deficiency (<5%)
- Kidney disease other than aHUS
- Chronic dialysis (hemo or peritoneal)
- Liver disease or other major autoimmune diseases
- Typical HUS (Shiga toxin +)
- Known Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, or antiphospholipid antibody positivity or syndrome
- 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 currently active or known history of meningococcal disease or N. meningitidis infection
- Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, chronic dialysis)
- Females who have a positive pregnancy test result at Screening or on Day 1.
- Pregnant, planning to become pregnant, or nursing female subjects.
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-aHUS-401,will initially assign six (6) patients per cohort in a 2-cohort trial. In the first cohort, we will evaluate a biweekly dosing regimen whereas in the second cohort, we will evaluate a weekly dose (10 mg/kg) followed by the biweekly dose (20 mg/kg) over a 3-month period. These studies will determine if NM8074 results in remission from TMA in aHUS patients. If the study shows efficacy in aHUS, additional patients may be added per cohort.
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 and required vaccinations, participants are assigned to one of two IV dosing schedules of NM8074 over about 12 weeks: Cohort 1 receives 20 mg/kg every two weeks for seven infusions; Cohort 2 receives 10 mg/kg weekly for four doses then 20 mg/kg every two weeks for five doses. Participants will attend clinic visits for infusions, safety monitoring and blood and urine tests (including platelet count, LDH, kidney function, haptoglobin, and schistocyte checks) with follow-up assessments through about Day 120. They will complete quality-of-life questionnaires (FACIT‑Fatigue and EORTC QLQ‑C30) and pregnancy testing as required. Participation also involves agreeing to the study contraception requirements and, for male participants, refraining from donating sperm for the duration of the study.
How the study is structured. Phase II, open-label, non-randomized interventional trial with two sequential cohorts. About six patients start in each cohort. Participants receive NM8074 by IV infusion on one of two predefined dosing schedules over the treatment period (about 85 days). There is no placebo or masking.
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 atypical hemolytic uremic syndrome
Important dates
- Study start
- November 2027 (estimated)
- Primary completion
- April 2030 (estimated)
- Study completion
- February 2031 (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, 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.
- treatment-naïve
- Has not previously received the treatment, or the type of treatment, that the study is about.
- relapsed
- A condition that improved with treatment and has since returned or worsened.
- ULN
- 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.
- eGFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- during Screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- 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.
- 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.
- 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.
- 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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