[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"acquired-pure-red-cell-aplasia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:acquired-pure-red-cell-aplasia":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,50],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":30,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100547786","phase-2-mt2023-20-hematopoietic-cell-transplant-with-reduced-intensity-conditioning-and-post-transplant-cyclophosphamide-for-severe-aplastic-anemia-and-other-forms-of-acquired-bone-marrow-failure-100547786",false,"NCT06412497","MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.","Inclusion Criteria:\n\n* Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL AND bone marrow cellularity \\\u003C50% (with \\\u003C 30% residual hematopoietic cells)\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%), without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic SAA with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Paroxysmal Nocturnal Hemoglobinuria (PNH), including AA-PNH overlap syndrome, acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone \\>\u002F= 10%\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%) without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic PNH, aPRCA, or aAT with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Adequate organ function within 30 days of conditioning regimen\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment\n* Uncontrolled infection\n* Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy\n* Known allergy to any of the study components\n* Prior radiation therapy deemed excessive by radiation therapist for proposed low dose TBI exposure on this protocol\n* Diagnosis of an inherited bone marrow failure disorder such as Fanconi anemia, Telomere biology disorder, or Schwachman-Diamond syndrome, unless reviewed by the principal investigator and deemed appropriate for this approach (e.g. GATA2 deficiency)\n* Advanced myelodysplastic syndrome (MDS; BM blasts \\>5%) or acute myeloid leukemia\n* Psychiatric illness\u002Fsocial situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements\n* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study","ALL","0 Years","75 Years",{"count":19,"type":20},60,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and\u002For clonal hematopoiesis.",[26,27,28,29],"Severe Aplastic Anemia","Acquired Amegakaryocytic Thrombocytopenia","Acquired Pure Red Cell Aplasia","Paroxysmal Nocturnal Hemoglobinuria",[31,32,33,34,35,36],"HCT","RIC","SAA","PTCy","aAT","aPRCA","RECRUITING","2026-06-02",{"date":40,"type":41},"2026-06-03","ACTUAL",{"date":43,"type":41},"2024-06-05",{"date":45,"type":20},"2036-05-01",{"name":47,"class":48},"Masonic Cancer Center, University of Minnesota","OTHER",1,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":4,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":15,"minAge":57,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":21,"phases":60,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":66,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":49},"100595335","phase-2-linperlisib-in-the-treatment-of-aprca-100595335","NCT07031115","Linperlisib in the Treatment of aPRCA","Linperlisib in Acquired Pure Red Cell Aplasia: a Single-arm,Cphase II Prospective Clinical Study","Inclusion Criteria:\n\n* Age ≥ 18 years;\n* Patients meeting the diagnostic criteria for acquired PRCA;\n* Patients who are relapsed or refractory after at least two lines of prior therapy. Refractory is defined as failure to achieve partial remission after 3 months of treatment at a stable dose of immunosuppressive agents.\n* Hb ≤ 100 g\u002FL;\n* Patients with complete clinical data, demonstrated good treatment compliance, and who have signed the informed consent form;\n* If taking glucocorticoids, must have discontinued them or been on a stable low maintenance dose (prednisone ≤ 15 mg\u002Fday) for at least 2 weeks prior to enrollment, and continue this dose;\n* If taking immunosuppressive agents such as cyclosporine or sirolimus, must have been on a stable dose for at least 3 months, and discontinue them upon enrollment.\n\nExclusion Criteria:\n\n* Patients with organ dysfunction (e.g., heart, liver, or lung) or acute renal insufficiency;\n* Patients who have used PI3Kδ inhibitors within the past 6 months;\n* Patients with severe infectious diseases;\n* Patients with malignant tumors;\n* Patients with psychiatric disorders or cognitive impairment;\n* Pregnant or lactating women;\n* Patients who have participated in other clinical trials within the past 3 months.","18 Years",{"count":59,"type":20},15,[23],"Pure red cell aplasia (PRCA) is a syndrome characterized by normocytic normochromic anemia, reticulocytopenia, and reduced erythroid precursors in an otherwise normocellular bone marrow. It primarily affects erythropoiesis, while granulocytic and megakaryocytic lineages typically remain unaffected. First-line therapies for PRCA include corticosteroids (CS) and cyclosporine A（CsA). Although CS demonstrates high response rates, relapse frequently occurs upon dose reduction or discontinuation. CsA achieves response rates of 65%-87%, yet exhibits a delayed onset of action, often requiring 2-3 months to achieve transfusion independence. Sirolimus constitutes a second-line option, with additional therapeutic agents including methotrexate and cyclophosphamide.\n\nPhosphatidylinositol 3-kinases (PI3Ks) represent a family of lipid kinases. The δ and γ isoforms are predominantly expressed in leukocytes and are frequently activated in various B-cell lymphomas, serving as the primary therapeutic targets for currently approved PI3K inhibitors in hematological malignancies. PI3K also plays a critical role in modulating cells of both the adaptive and innate immune systems. Studies indicate that engagement of multiple immune receptors on leukocytes triggers PI3K activation. Consequently, isoform-selective (δ or γ) or dual δ\u002Fγ inhibitors are being investigated for autoimmune conditions such as COPD, asthma, allergies, and Sjögren's syndrome. Leniolisib, the first oral PI3Kδ inhibitor approved by the FDA for immunodeficiency, exemplifies this therapeutic strategy. Several other PI3K-targeting agents are under clinical evaluation, including Parsaclisib (Phase II trial in relapsed\u002Frefractory autoimmune hemolytic anemia) and Linperlisib (Phase I trial in relapsed\u002Frefractory AIHA).\n\nT-lymphocyte dysfunction is a pivotal factor in PRCA pathogenesis. RNA sequencing analyses have revealed significant upregulation of genes associated with the PI3K\u002FAKT\u002FmTOR pathway in bone marrow CD8+ T lymphocytes of patients with acquired PRCA, suggesting that targeting this pathway may represent a novel therapeutic strategy. Linperlisib, a highly selective PI3Kδ inhibitor approved for relapsed\u002Frefractory follicular lymphoma, suppresses PI3Kδ protein expression and reduces AKT phosphorylation, thereby inducing apoptosis and inhibiting lymphocyte proliferation. In 2024, a seminal report documented rapid responses and manageable tolerability with Linperlisib in four patients with acquired PRCA.\n\nCurrently, no cohort studies have been conducted on Linperlisib for PRCA treatment. This study seeks to characterize the dosing regimen, efficacy, and safety profile of Linperlisib in relapsed\u002Frefractory pure red cell aplasia.",[28],[64,28,65],"Linperlisib","Treatment","NOT_YET_RECRUITING","2025-06-12",{"date":69,"type":41},"2025-06-22",{"date":71,"type":20},"2025-09-01",{"date":73,"type":20},"2026-12-31",{"name":75,"class":48},"Peking Union Medical College Hospital"]