Not Yet RecruitingPhase 2

Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy

12 participantsStarted 2027-11

Plain-language summary

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.

Who can participate

Age range

18 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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,…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Normalization of platelet count (≥150 x 10^9/L)

Timeframe: Up to Study Day 120

Normalization of LDH levels to below ULN

Timeframe: Up to Study Day 120

Normalization of Schistocyte levels (<1%)

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in renal function

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in Haptoglobin

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in Hemoglobin

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in proteinuria/creatininuria

Timeframe: Up to Study Day 120

Trial details

NCT IDNCT05684159

SponsorNovelMed Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-04

Contact for this trial

Rekha Bansal, PhD

2164402696 clinicalsae@novelmed.com

View on ClinicalTrials.gov More aHUS - Atypical Hemolytic Uremic Syndrome trials

Who can participate

Age range

18 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Normalization of platelet count (≥150 x 10^9/L)

Timeframe: Up to Study Day 120

Normalization of LDH levels to below ULN

Timeframe: Up to Study Day 120

Normalization of Schistocyte levels (<1%)

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in renal function

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in Haptoglobin

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in Hemoglobin

Timeframe: Up to Study Day 120

Change from Baseline or Percent Change from Baseline in proteinuria/creatininuria

Timeframe: Up to Study Day 120