[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pnh---paroxysmal-nocturnal-hemoglobinuria\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pnh---paroxysmal-nocturnal-hemoglobinuria":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,42,66],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100612647","phase-1-a-phase-ib-multicenter-open-label-study-of-multiple-dose-ea5-in-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100612647",false,"NCT07256301","A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)","A Multicenter, Open-Label, Phase Ib Clinical Trial to Evaluate the Safety, Pharmacokinetic, and Pharmacodynamic Profiles of Multiple Doses of the Humanized Monoclonal Antibody EA5 in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","Inclusion Criteria:\n\n* Male or female subjects aged ≥18 years.\n* Body weight between 40 kg and 100 kg (inclusive) at screening.\n* Patients diagnosed with PNH, confirmed by flow cytometry demonstrating a PNH clone size (glycosylphosphatidylinositol-anchored protein-deficient granulocytes or monocytes) of ≥10% in peripheral blood, and meeting one of the following criteria:\n\n  * a) Previously naive to complement inhibitor therapy; or\n  * b) Previously treated with a complement inhibitor, which has been discontinued for ≥5 half-lives prior to screening.\n* Lactate dehydrogenase (LDH) level ≥1.5 times the upper limit of normal (ULN) at screening.\n* Presence of one or more of the following PNH-related signs or symptoms within 3 months prior to screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin \\\u003C10 g\u002FdL), history of major thrombotic event (including thrombosis), dysphagia, or erectile dysfunction; or a history of packed red blood cell (pRBC) transfusion due to PNH.\n* Vaccination against Neisseria meningitidis(serogroups A, C, W, Y) within \\\u003C3 years prior to the initiation of study treatment; OR if not previously vaccinated, receipt of the meningococcal vaccine (MPV-ACYW) at least 14 days prior to the first dose of the investigational product. If the vaccine is administered within 14 days before dosing, antibiotic prophylaxis must be provided until 2 weeks post-vaccination.\n* Vaccination against Streptococcus pneumoniaeaccording to national vaccination recommendations (e.g., ACIP guidelines). OR if not previously vaccinated, receipt of the pneumococcal vaccine at least 14 days prior to the first dose of the investigational product. If the vaccine is administered within 14 days before dosing, antibiotic prophylaxis must be provided until 2 weeks post-vaccination.\n* For patients receiving concomitant therapies (e.g., immunosuppressants, corticosteroids, iron supplements, anticoagulants, erythropoiesis-stimulating agents): the dose must have been stable for ≥28 days prior to the first dose of the investigational product.\n* Platelet count ≥30 × 10\\^9\u002FL at screening (without transfusion support within 7 days), and absolute neutrophil count (ANC) ≥0.5 × 10\\^9\u002FL (without short-acting granulocyte colony-stimulating factor (G-CSF) within 14 days or long-acting G-CSF within 28 days).\n* Adequate liver function, defined as alanine aminotransferase (ALT) ≤3 × ULN, OR both direct bilirubin and alkaline phosphatase (ALP) ≤2 × ULN at screening.\n* Adequate renal function, defined as serum creatinine ≤2.5 × ULN and an estimated creatinine clearance ≥30 mL\u002Fmin as calculated by the Cockcroft-Gault formula.\n* Male subjects must agree to use effective contraception (including vasectomy, abstinence, or condom) from screening until 6 months after the final study intervention. Women of childbearing potential (WOCBP) must have a negative blood pregnancy test at screening and baseline. During the study and for 6 months thereafter, all subjects and their partners must agree to use effective contraceptive measures (Note: contraceptive measures include both pharmacological and non-pharmacological methods).\n* Ability to understand the procedures and methods of the study, willingness to provide written informed consent, and commitment to strictly adhere to the clinical study protocol to complete the study.\n\nExclusion Criteria:\n\n* History of allogeneic bone marrow transplantation.\n* History of Neisseria meningitidisinfection within 6 months prior to screening and before the first dose.\n* Known or suspected immunodeficiency (e.g., history of frequent or recurrent infections).\n* Known or suspected hereditary complement deficiency.\n* Evidence of active tuberculosis (TB) within 6 months prior to screening, or a history of active TB without having completed an appropriate, documented course of treatment; OR chest X-ray (posteroanterior and lateral) or CT scan findings during the 3 months prior to screening or during the screening period that suggest the presence of active TB infection.\n* History of major surgery (Grade 3 or 4 surgery) within 3 months prior to the first dose.\n* Presence of an autoimmune disease, OR use of systemic immunosuppressive\u002Fimmunomodulatory agents (including, but not limited to, methotrexate, cyclosporine, mycophenolate, tacrolimus, penicillamine, sulfasalazine, hydroxychloroquine, azathioprine, cyclophosphamide) for the treatment of inflammatory diseases within 12 weeks or 5 half-lives (whichever is longer) prior to screening.\n* Active systemic bacterial, viral, or fungal infection within 14 days prior to the first dose; OR requirement for hospitalization or intravenous antibiotic therapy for an infection between 28 days prior to screening and the first dose; OR requirement for oral antibiotic therapy for an infection between 14 days prior to screening and the first dose.\n* Occurrence of fever (≥38°C) within 7 days prior to the first drug administration.\n* Administration of any live-attenuated vaccine within 1 month prior to the first dose.\n* History of malignancy within 5 years prior to screening and before the first dose, with the following exceptions: patients with any malignancy that has been treated with curative intent and who have been disease-free and off treatment for \\>5 years prior to the first dose may be enrolled. Patients with a history of basal cell carcinoma, squamous cell carcinoma of the skin, or carcinoma in situof the cervix that has been cured with no evidence of recurrence at any time prior to the first dose may be enrolled. Patients with a history of low-grade, early-stage prostate cancer (Gleason score ≤6, Stage 1 or 2) not requiring treatment at any time prior to the first dose may be enrolled.\n* History of allergy to any component of EA5, including a history of hypersensitivity to human, humanized, or murine monoclonal antibodies, or known hypersensitivity to any excipient of the product.\n* Any contraindication to receiving the meningococcal vaccination and\u002For antibiotic prophylaxis (e.g., beta-lactam antibiotics, ciprofloxacin) as required by the study protocol.\n* Participation in another interventional therapeutic clinical trial involving an investigational drug or receipt of any experimental therapy within 3 months (or within 5 half-lives of the investigational agent, whichever is longer) prior to screening.\n* History of drug abuse within 12 months prior to screening, as judged by the investigator.\n* Alcohol abuse, or regular alcohol consumption exceeding 14 units per week within 6 months prior to screening (1 unit of alcohol ≈ 360 mL of beer, or 45 mL of 40% spirits, or 150 mL of wine).\n* Splenectomy performed within ≤6 months prior to screening.\n* Positive for hepatitis C virus (HCV) antibody at screening (except for those with a negative HCV RNA result), positive for human immunodeficiency virus (HIV) antibody, positive for anti-Treponema pallidumantibody (TP-Ab) (except for those with a negative RPR or TRUST test), OR positive for hepatitis B virus (HBV) surface antigen (HBsAg) (except for those with an HBV DNA level ≤1000 IU\u002FmL).\n* History of or ongoing cryoglobulinemia at the time of screening.\n* History of myelodysplastic syndromes (MDS), with a Revised International Prognostic Scoring System (IPSS-R) risk category of Intermediate, High, or Very High.\n* Any other condition that, in the opinion of the investigator, renders the subject unsuitable for participation in the trial.","ALL","18 Years",{"count":19,"type":20},24,"ESTIMATED","INTERVENTIONAL",[23],"PHASE1","This is a multicenter, open-label, Phase Ib clinical trial designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple doses of the humanized monoclonal antibody EA5 in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).",[26],"PNH - Paroxysmal Nocturnal Hemoglobinuria",[28],"EA5、PNH、Paroxysmal Nocturnal Hemoglobinuria、C5 complement inhibitor","RECRUITING","2026-05-13",{"date":32,"type":33},"2026-05-14","ACTUAL",{"date":35,"type":33},"2024-01-03",{"date":37,"type":20},"2026-10-30",{"name":39,"class":40},"Shanghai Lanyi Therapeutics Co., Ltd.","INDUSTRY",2,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":21,"phases":51,"briefSummary":53,"conditions":54,"keywords":55,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":60,"completionDateStruct":62,"leadSponsor":63,"locationsCount":65},"100591305","phase-2-a-phase-ii-study-evaluating-the-efficacy-and-safety-of-xh-s003-capsules-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100591305","NCT06978699","A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","A Multicenter, Randomized, Single-blind Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","XH-S003-II-101","Inclusion Criteria:\n\n* Male or female with aged ≥18 years old;\n* Weight ≥40 kg and BMI≥18 kg\u002Fm2 ;\n* Diagnosed with PNH: with red blood cell or granulocyte clone levels \\>10% detected by flow cytopy within 6 months prior to screening or during screening;\n* Patients who have not previously received any complement inhibitor therayp;\n* LDH \\> 1.5×ULN detected two times during the screening period (interval of 2 to 8 weeks);\n* Hb meets one of the following conditions: (1) Hb \\\u003C100 g\u002FL at the first screening visit, and subjects receive RBC transfusion because of PNH-related anemia during the screening period; (2) The average Hb of two tests during the screening period \\\u003C100 g\u002FL (interval of 2\\~8 weeks);\n* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae before the first administration. If the subject has not been vaccinated previously or requires booster vaccination (according to local vaccination policies), vaccination must be administered at least 2 weeks before the first administration. If the first administration must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination;\n\nExclusion Criteria:\n\n* Subjects with laboratory evidence of bone marrow failure during the screening period (reticulocyte count \\\u003C100×109\u002FL, platelet count \\\u003C30×109\u002FL, or neutrophil count \\\u003C0.5×109\u002FL);\n* Subjects receiving other therapies prior to screening who have not achieved the following treatment durations:\n\n  • Erythropoietin or immunosuppressants for at least 8 weeks; • Systemic corticosteroids for at least 4 weeks; • Iron supplements, vitamin B12, or folic acid for at least 4 weeks; • Anticoagulants: Vitamin K antagonists for at least 4 weeks with stable international normalized ratio (INR) (as determined by the investigator), low molecular weight heparin for at least 4 weeks; • Hypoxic-inducing factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks; • Androgens for at least 4 weeks;\n* A history of bone marrow\u002Fhematopoietic stem cell or solid organ transplantation;\n* Alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) \\>3×ULN at screening; - Positive HIV antibody, active syphilis infection, positive HBsAg, active HCV infection, or active tuberculosis infection at screening;\n* Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;\n* A history of Neisseria meningitidis infection;\n* Subjects with chronic active or recurrent infections within 1 year prior to screening;\n* Subjects with systemic active bacterial, viral (including COVID-19), or fungal infections within 2 weeks prior to the first administration; subjects with body temperature \\>38°C within 7 days prior to the first administration;",{"count":19,"type":20},[52],"PHASE2","This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally",[26],[56],"Paroxysmal Nocturnal Hemoglobinuria","2025-05-16",{"date":59,"type":33},"2025-05-18",{"date":61,"type":33},"2025-04-30",{"date":37,"type":20},{"name":64,"class":40},"S-INFINITY Pharmaceuticals Co., Ltd",1,{"id":67,"slug":68,"hasResults":11,"nctId":69,"briefTitle":70,"officialTitle":71,"acronym":4,"eligibilityCriteria":72,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":73,"enrollmentInfo":74,"targetDuration":4,"studyType":21,"phases":76,"briefSummary":77,"conditions":78,"keywords":4,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":83,"completionDateStruct":85,"leadSponsor":87,"locationsCount":4},"100495427","phase-2-study-of-nm8074-in-soliris-treated-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100495427","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)","Inclusion Criteria:\n\n* Male or female patients ≥ 18 years at the time of consent\n* 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%\n* 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 \\\u003C 10 g\u002FdL, history of a major adverse vascular event (including thrombosis), dysphagia, erectile dysfunction, PNH-mediated pRBC transfusions\n* 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\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* 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\n* Female subjects of child-bearing potential and all male subjects must agree to use of effective contraception during study\n* Soliris treated individuals must be able to provide documentation of vaccination against meningococcal infections.\n\nExclusion Criteria:\n\n* Platelet count \\\u003C 30,000\u002FµL at Screening\n* Absolute neutrophil count (ANC) \\\u003C 500 cells\u002FµL at Screening\n* Body weight \\\u003C 85 lbs. (38 kg) at Screening\n* Estimated glomerular filtration rate of \\\u003C 30 mL\u002Fmin\u002F1.73m2 based on modification of diet in renal disease (MDRD) equation, creatinine clearance, or CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) at Screening\n* Elevation of liver function tests: alanine aminotransferase (ALT) \\> 2xULN or direct bilirubin and alkaline phosphatase (ALP) both \\> 2xULN\n* Has a known history of meningococcal disease or N. meningitidis infection\n* 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)\n* Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection that requires antibiotic, antifungal, antiparasitic, or antiviral mediations\n* Temperature \\> 38°C for more than two weeks prior to screening\n* History of bone marrow or solid organ transplantation\n* Pregnant, planning to become pregnant, or nursing female subjects\n* 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\n* 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)\n* 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\n* PNH patients currently under complement blocker treatments other than Soliris\n* Concomitant use of anticoagulants is prohibited, if not on a stable regimen for at least 2 weeks prior to Day 1\n* Participation in any experimental small molecule or non-antibody therapy within 60 days prior to dosing on Day 1 (participation in observational studies and\u002For registry studies is permitted)\n* Known or suspected history of illegal recreational drug or alcohol abuse within 1 year prior to start of screening\n* Hypersensitivity or history of allergy to excipients in NM8074 formulation\n* Unable or unwilling to comply with the requirements of the study","65 Years",{"count":75,"type":20},6,[52],"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.",[26],"NOT_YET_RECRUITING","2025-03-28",{"date":82,"type":33},"2025-03-30",{"date":84,"type":20},"2026-06",{"date":86,"type":20},"2028-03",{"name":88,"class":40},"NovelMed Therapeutics"]