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Not yet recruitingNCT05684159Updated Apr 13, 2026

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

A Phase 2 interventional study of NM8074 in aHUS - Atypical Hemolytic Uremic Syndrome, sponsored by NovelMed Therapeutics. Not yet recruiting. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-04-13.

Sponsored by NovelMed Therapeutics · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
12
Allocation
Non-randomized
Ages
18 Years to 65 Years
Sex
All
01

Study 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.

Read the detailed description

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.

02

Conditions studied

  • aHUS - Atypical Hemolytic Uremic Syndrome
03

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
12 participants (estimated)

Study arms

  • Experimental
    Cohort 1

    6 subjects will receive an intravenous (IV) infusion of NM8074 at every two weeks for a total of 7 doses

    Drug: NM8074

  • Experimental
    Cohort 2

    6 subjects will receive weekly doses of 10 mg/kg for a total of 4 doses followed by biweekly doses at 20 mg/kg for a total of 5 doses

    Drug: NM8074

Interventions

  • DrugNM8074

    NM8074 will be administered as an intravenous infusion. In Cohort 1, all subjects will be administered 20 mg/kg of NM8074 intravenously every two weeks for a total of 7 doses from Day 1 to Day 85 of the Treatment Period. Patients in Cohort 2 will receive weekly doses of 10 mg/kg for a total of 4 doses from Day 1 to Day 22 followed by biweekly doses at 20 mg/kg for a total of 5 doses from Day 29 to Day 85.

05

What researchers measure

Primary outcomes

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

    Time frame: Up to Study Day 120

  2. Normalization of LDH levels to below ULN

    Time frame: Up to Study Day 120

  3. Normalization of Schistocyte levels (<1%)

    Time frame: Up to Study Day 120

  4. Change from Baseline or Percent Change from Baseline in renal function

    Assessed via the change from baseline or percent change from baseline in serum creatinine level.

    Time frame: Up to Study Day 120

  5. Change from Baseline or Percent Change from Baseline in Haptoglobin

    Time frame: Up to Study Day 120

  6. Change from Baseline or Percent Change from Baseline in Hemoglobin

    Time frame: Up to Study Day 120

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

    Time frame: Up to Study Day 120

Secondary outcomes

  1. Time to achieve complete TMA response

    Time frame: Baseline through Study Day 120

  2. Time to achieve higher hemoglobin from baseline

    Time frame: Baseline through Study Day 120

  3. Change from Baseline or Percent Change from Baseline in blood clots

    Time frame: Up to Study Day 120

  4. Change from Baseline or Percent Change from Baseline in the total number of plasma infusions or exchanges

    Time frame: Baseline through Study Day 120

  5. Change from Baseline or Percent Change from Baseline in eGFR (estimated glomerular filtration rate)

    Time frame: Baseline through Study Day 120

  6. Change from Baseline or Percent Change from Baseline in dialysis requirement

    Time frame: Baseline through Study Day 120

  7. Change from Baseline or Percent Change from Baseline in quality of life (QoL) Assessed via the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale, Version 4.

    The FACIT-fatigue scale is a 13-item patient-reported measure of fatigue with a 7-day recall period. Items are scored on a 0 - 4 response scale ranging from "Not at all" to "Very much so". All items are summed to create a single fatigue score with a range from 0 to 52 with a better quality of life indicated by a higher score.

    Time frame: Baseline through Study Day 120

  8. Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) Assessed via the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale (QLQ- C30), Version 3.0

    All EORTC QLQ-C30 scales and single-item measures range from 0 to 100. This includes 3 symptom scales (fatigue, pain, nausea and vomiting), 5 functional scales (physical, role, cognitive, emotional, and social), single-item questions addressing symptoms like insomnia, dyspnea, loss of appetite, and others that are commonly reported by cancer patients, and the perceived financial impact of the disease. A higher score is associated with a greater quality of life for global health status.

    Time frame: Baseline through Study Day 120

Other outcomes

  1. Change from Baseline or Percent Change from Baseline in CP modulation

    Time frame: Baseline through Study Day 120

  2. Change from Baseline or Percent Change from Baseline in Factor B levels

    Time frame: Baseline through Study Day 120

  3. Change from Baseline or Percent Change from Baseline in plasma concentration of NM8074

    Time frame: Baseline through Study Day 120

  4. Maximum plasma concentration (Cmax)

    Time frame: Baseline through Study Day 120

  5. Time corresponding to Cmax (tmax)

    Time frame: Baseline through Study Day 120

  6. Area under the drug concentration-time curves (AUC0-t)

    Time frame: Baseline through Study Day 120

06

Study locations

No study locations are listed for this record.

07

References and documents

Publications

  • Barbour T, Scully M, Ariceta G, Cataland S, Garlo K, Heyne N, Luque Y, Menne J, Miyakawa Y, Yoon SS, Kavanagh D; 311 Study Group Members. Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults. Kidney Int Rep. 2021 Mar 24;6(6):1603-1613. doi: 10.1016/j.ekir.2021.03.884. eCollection 2021 Jun. PubMed 34169200 ↗
  • Cammett TJ, Garlo K, Millman EE, Rice K, Toste CM, Faas SJ. Exploratory Prognostic Biomarkers of Complement-Mediated Thrombotic Microangiopathy (CM-TMA) in Adults with Atypical Hemolytic Uremic Syndrome (aHUS): Analysis of a Phase III Study of Ravulizumab. Mol Diagn Ther. 2023 Jan;27(1):61-74. doi: 10.1007/s40291-022-00620-3. Epub 2022 Nov 4. PubMed 36329366 ↗
  • Cofiell R, Kukreja A, Bedard K, Yan Y, Mickle AP, Ogawa M, Bedrosian CL, Faas SJ. Eculizumab reduces complement activation, inflammation, endothelial damage, thrombosis, and renal injury markers in aHUS. Blood. 2015 May 21;125(21):3253-62. doi: 10.1182/blood-2014-09-600411. Epub 2015 Apr 1. PubMed 25833956 ↗
  • Pugh D, O'Sullivan ED, Duthie FA, Masson P, Kavanagh D. Interventions for atypical haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Mar 23;3(3):CD012862. doi: 10.1002/14651858.CD012862.pub2. PubMed 33783815 ↗

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT05684159
Lead sponsor
NovelMed Therapeutics
Responsible party
Sponsor
First posted
Jan 13, 2023
Start date
Nov 2027 (estimated)
Primary completion
Apr 2030 (estimated)
Completion
Feb 2031 (estimated)
Last update
Apr 13, 2026

Study contacts

Rekha Bansal, PhD
Contact
clinicalsae@novelmed.com
2164402696

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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