[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100491824":3},{"organization":4,"outcomesModule":7,"designInfo":59,"detailedDescription":66,"studyPopulation":11,"armGroups":67,"interventions":78,"overallOfficials":11,"centralContacts":84,"locations":11,"responsibleParty":90,"collaborators":11,"id":92,"slug":11,"hasResults":93,"nctId":94,"briefTitle":95,"officialTitle":96,"acronym":11,"eligibilityCriteria":97,"healthyVolunteers":93,"sex":98,"minAge":99,"maxAge":100,"enrollmentInfo":101,"targetDuration":11,"studyType":104,"phases":105,"briefSummary":107,"conditions":108,"keywords":11,"overallStatus":110,"whyStopped":11,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":115,"completionDateStruct":117,"leadSponsor":119,"locationsCount":11},{"fullName":5,"class":6},"NovelMed Therapeutics","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":26,"otherOutcomes":46},[9,13,15,17,20,22,24],{"measure":10,"description":11,"timeFrame":12},"Normalization of platelet count (≥150 x 10^9\u002FL)",null,"Up to Study Day 120",{"measure":14,"description":11,"timeFrame":12},"Normalization of LDH levels to below ULN",{"measure":16,"description":11,"timeFrame":12},"Normalization of Schistocyte levels (\u003C1%)",{"measure":18,"description":19,"timeFrame":12},"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.",{"measure":21,"description":11,"timeFrame":12},"Change from Baseline or Percent Change from Baseline in Haptoglobin",{"measure":23,"description":11,"timeFrame":12},"Change from Baseline or Percent Change from Baseline in Hemoglobin",{"measure":25,"description":11,"timeFrame":12},"Change from Baseline or Percent Change from Baseline in proteinuria\u002Fcreatininuria",[27,30,32,34,36,38,40,43],{"measure":28,"description":11,"timeFrame":29},"Time to achieve complete TMA response","Baseline through Study Day 120",{"measure":31,"description":11,"timeFrame":29},"Time to achieve higher hemoglobin from baseline",{"measure":33,"description":11,"timeFrame":12},"Change from Baseline or Percent Change from Baseline in blood clots",{"measure":35,"description":11,"timeFrame":29},"Change from Baseline or Percent Change from Baseline in the total number of plasma infusions or exchanges",{"measure":37,"description":11,"timeFrame":29},"Change from Baseline or Percent Change from Baseline in eGFR (estimated glomerular filtration rate)",{"measure":39,"description":11,"timeFrame":29},"Change from Baseline or Percent Change from Baseline in dialysis requirement",{"measure":41,"description":42,"timeFrame":29},"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.",{"measure":44,"description":45,"timeFrame":29},"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.",[47,49,51,53,55,57],{"measure":48,"description":11,"timeFrame":29},"Change from Baseline or Percent Change from Baseline in CP modulation",{"measure":50,"description":11,"timeFrame":29},"Change from Baseline or Percent Change from Baseline in Factor B levels",{"measure":52,"description":11,"timeFrame":29},"Change from Baseline or Percent Change from Baseline in plasma concentration of NM8074",{"measure":54,"description":11,"timeFrame":29},"Maximum plasma concentration (Cmax)",{"measure":56,"description":11,"timeFrame":29},"Time corresponding to Cmax (tmax)",{"measure":58,"description":11,"timeFrame":29},"Area under the drug concentration-time curves (AUC0-t)",{"allocation":60,"interventionModel":61,"interventionModelDescription":62,"primaryPurpose":63,"observationalModel":11,"timePerspective":11,"maskingInfo":64},"NON_RANDOMIZED","SEQUENTIAL","The study will enroll a planned total of 12 patients as subjects for the trial, with extra enrollment if needed, at the discretion of the Investigator. All subjects will be assigned to either Cohort 1 or Cohort 2. Enrollment in Cohort 2 will occur after at least three patients in Cohort 1 have been evaluated for safety for 3 days after the first dose. No more than three patients will be dosed in a day. Safety data will be assessed and reviewed by the Sponsor and Study Investigators for dosed subjects prior to dosing of the rest of the study subjects. In Cohort 1, all 6 subjects will be administered 20 mg\u002Fkg of NM8074 intravenously every two weeks for a total of 7 doses. All 6 patients in Cohort 2 will receive weekly doses of 10 mg\u002Fkg for a total of 4 doses followed by biweekly doses at 20 mg\u002Fkg for a total of 5 doses.","TREATMENT",{"masking":65,"maskingDescription":11,"whoMasked":11},"NONE","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\u002Fkg) followed by the biweekly dose (20 mg\u002Fkg) 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.",[68,74],{"label":69,"type":70,"description":71,"interventionNames":72},"Cohort 1","EXPERIMENTAL","6 subjects will receive an intravenous (IV) infusion of NM8074 at every two weeks for a total of 7 doses",[73],"Drug: NM8074",{"label":75,"type":70,"description":76,"interventionNames":77},"Cohort 2","6 subjects will receive weekly doses of 10 mg\u002Fkg for a total of 4 doses followed by biweekly doses at 20 mg\u002Fkg for a total of 5 doses",[73],[79],{"type":80,"name":81,"description":82,"armGroupLabels":83,"otherNames":11},"DRUG","NM8074","NM8074 will be administered as an intravenous infusion. In Cohort 1, all subjects will be administered 20 mg\u002Fkg 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\u002Fkg for a total of 4 doses from Day 1 to Day 22 followed by biweekly doses at 20 mg\u002Fkg for a total of 5 doses from Day 29 to Day 85.",[69,75],[85],{"name":86,"role":87,"phone":88,"phoneExt":11,"email":89},"Rekha Bansal, PhD","CONTACT","2164402696","clinicalsae@novelmed.com",{"type":91,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100491824",false,"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)","Inclusion Criteria:\n\n* Patients ≥ 18 years at the time of consent\n* Patients with evidence of resistant or relapsed complement-mediated aHUS with symptoms of Thrombocytopenia, hemolysis, ongoing Thrombotic Microangiopathy and acute kidney injury.\n* Evidence of ongoing Thrombotic Microangiopathy which includes Haptoglobin \\\u003CLLN or undetectable and\u002For presence of schistocytes\n* Acute kidney injury (proteinuria\u002Fcreatinuria \\> ULN and\u002For reduced eGFR)\n* Platelets less than 150,000 per microliter (Thrombocytopenia)\n* Anemia (Hemoglobin ≤10 g\u002FdL) due to hemolysis\n* Lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during Screening\n* 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\n* 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.\n* 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.\n* 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.\n\nExclusion Criteria:\n\n* History of bone marrow, hematopoietic stem cell, or solid organ transplantation\n* Treatment with complement blockers\n* Patients with infections\n* HUS due to ADAMTS-13 deficiency (\\\u003C5%)\n* Kidney disease other than aHUS\n* Chronic dialysis (hemo or peritoneal)\n* Liver disease or other major autoimmune diseases\n* Typical HUS (Shiga toxin +)\n* Known Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, or antiphospholipid antibody positivity or syndrome\n* History of currently active primary or secondary immunodeficiency\n* 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\n* Has a currently active or known history of meningococcal disease or N. meningitidis infection\n* Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, chronic dialysis)\n* Females who have a positive pregnancy test result at Screening or on Day 1.\n* Pregnant, planning to become pregnant, or nursing female subjects.","ALL","18 Years","65 Years",{"count":102,"type":103},12,"ESTIMATED","INTERVENTIONAL",[106],"PHASE2","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.",[109],"aHUS - Atypical Hemolytic Uremic Syndrome","NOT_YET_RECRUITING","2026-04-07",{"date":113,"type":114},"2026-04-13","ACTUAL",{"date":116,"type":103},"2027-11",{"date":118,"type":103},"2031-02",{"name":5,"class":6}]