[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pulmonary-alveolar-proteinosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pulmonary-alveolar-proteinosis":23},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,41,68],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":16,"targetDuration":19,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":24,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100244533","a-national-registry-for-pulmonary-alveolar-proteinosis-100244533",false,"NCT02461615","A National Registry For Pulmonary Alveolar Proteinosis","Inclusion Criteria for Part A and Part B:\n\n* Written informed consent and assent, if applicable\n\nInclusion Criteria for Part A (Cross Sectional Study of PAP Syndrome)\n\n* History of chest computed tomogram or chest radiograph findings compatible with PAP\n* History of diagnosis of PAP made by at least one of the following methods:\n\n  * Positive (Abnormal) serum GMAb test -OR-\n  * Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-\n  * Bronchoalveolar lavage cytology compatible with PAP -OR-\n  * Recessive or compound mutations in genes known to cause PAP, i.e. GM-CSF receptor α or β chain, GM-CSF, surfactant protein B or C or ABCA3, ABCG1, ABCA1, TTF1\n\nInclusion Criteria For Part B (Longitudinal \\& PRO Survey Study of autoimmune PAP Patients)\n\n* Diagnosis of autoimmune PAP as indicated by:\n\n  * Positive (Abnormal) Serum GMAb Test -AND-\n  * History of chest CT or x-rays findings compatible with PAP -OR-\n  * Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-\n  * Bronchoalveolar lavage cytology compatible with PAP\n\nExclusion Criteria or Part A and Part B:\n\n* Individuals who have a serious medical illness that, in the opinion of the investigator, is likely to interfere with completion of the study will be excluded.\n\nFor Part A (Cross-sectional Study of PAP Syndrome)\n\n* Individuals that do not have a diagnosis of PAP\n\nFor Part B (Longitudinal \\& PRO Survey Study of autoimmune PAP Patients)\n\n* Individuals that do not have a diagnosis of autoimmune PAP","ALL",{"count":17,"type":18},500,"ESTIMATED","5 Years","OBSERVATIONAL","The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the diagnosis of PAP, increase awareness and knowledge of PAP, and give patients a 'seat at the table' in planning and conducting PAP research including the clinical testing of several new potential therapies. The major goal of Part B of this study is to define the natural history of autoimmune PAP (aPAP), develop a disease severity score that reflects how aPAP patients feel and function, and to develop and test novel tools to measure the severity of aPAP lung disease. Funding Source - FDA OOPD",[23],"Pulmonary Alveolar Proteinosis",[25,26,27],"Pulmonary Surfactant","Rare Lung Disease","Registry","RECRUITING","2026-03-16",{"date":31,"type":32},"2026-03-17","ACTUAL",{"date":34,"type":4},"2015-04",{"date":36,"type":18},"2030-12",{"name":38,"class":39},"Children's Hospital Medical Center, Cincinnati","OTHER",1,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":15,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":54,"briefSummary":56,"conditions":57,"keywords":4,"overallStatus":58,"whyStopped":4,"lastUpdateSubmitDate":59,"lastUpdatePostDateStruct":60,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":4},"100592123","phase-2-local-spraying-of-gm-csf-via-bronchoscopy-in-the-treatment-of-autoimmune-pulmonary-alveolar-proteinosis-100592123","NCT06989333","Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis","A Single-arm Clinical Study of Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis","aPAP and GMCSF","Inclusion Criteria:\n\n1. Age: 18 to 60 years old;\n2. A clear diagnosis of aPAP must meet at least one of the following diagnostic criteria:\n\n1\\) BALF appears \"milky white\"; Or cytological examination reveals a large amount of PAS-positive protein deposition; 2) HRCT shows typical \"paving stone-like changes\"; 3) Positive for serum GM-CSF antibody. 3. There are more than one of the following treatment indications: Symptoms such as progressive breathing difficulties, coughing, and shortness of breath after activity occur; 2) Without oxygen inhalation, PaO2 \\> 65 mmHg 3) Pulmonary function DLCO accounts for % of the predicted value, ranging from 60% to 80%, including the critical value.\n\n4\\. No other PAP specific treatments (such as WLL, inhaled GM-CSF, biological agents, etc.) have been received recently (for more than 4 weeks).\n\n5\\. Agree to participate in this study and sign the informed consent form.\n\nExclusion Criteria:\n\n1. Secondary PAP (such as secondary to blood diseases, etc.);\n2. Patients with obvious pulmonary fibrosis, emphysema or irreversible lung function impairment;\n3. Patients in the acute exacerbation stage;\n4. Patients with other lung diseases (such as active pulmonary tuberculosis, bronchiectasis with purulent infection or other chronic infections; Have severe asthma, chronic bronchospasm, etc.\n5. Have a history of allergy to GM-CSF antibodies or related drug components;\n6. Patients who have participated in other clinical drug trials within the past three months;\n7. Patients who have experienced severe complications related to bronchoscopy or are intolerant to bronchoscopy;\n8. Concurrent with other serious cardiovascular and cerebrovascular diseases, hematological disorders, malignant tumors, etc.\n9. Pregnant or lactating women.","18 Years","80 Years",{"count":52,"type":18},10,"INTERVENTIONAL",[55],"PHASE2","This study aims to explore a new therapeutic approach: the feasibility, safety and preliminary efficacy of directly spraying GM-CSF into the airway through bronchoscopy for the treatment of aPAP.",[23],"NOT_YET_RECRUITING","2025-05-17",{"date":61,"type":32},"2025-05-25",{"date":63,"type":18},"2025-07-01",{"date":65,"type":18},"2027-06-30",{"name":67,"class":39},"Jiuwu Bai",{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":74,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":76,"targetDuration":78,"studyType":20,"phases":4,"briefSummary":79,"conditions":80,"keywords":86,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":5},"100274490","european-management-platform-for-childhood-interstitial-lung-diseases---child-eu-register-and-biobank-100274490","NCT02852928","European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank","Orphans Unite: chILD Better Together - European Management Platform for Childhood Interstitial Lung Diseases \u002F chILD-EU - International Register and Biobank for Children´s Interstital Lung Disease","chILD-EU","Inclusion Criteria:\n\n* all children with suspected or\n* verified diagnosis of ILD or\n* masquerading as ILD and\n* those with rare localized parenchymal lung diseases\n\nExclusion Criteria:\n\n* other indication as inclusion criteria",{"count":77,"type":18},1000,"10 Years","Generation of a common European database and biobank Continous assessment and implementation of guidelines and treatment protocols Establishment of a large observational cohort of chILD patients Determination the value of outcomes used in child Assess treatment variations used, deliver data from defined protocols and linked outcomes",[81,23,82,83,84,85],"Lung Diseases, Interstitial","Pulmonary Eosinophilia","Pulmonary Fibrosis","Respiratory Distress Syndrome, Newborn","Child",[87],"ILD; chILD","2022-09-18",{"date":90,"type":32},"2022-09-21",{"date":92,"type":32},"2013-12",{"date":94,"type":18},"2029-12",{"name":96,"class":39},"Matthias Griese"]