[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"alpha-1-antitrypsin-deficiency\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:alpha-1-antitrypsin-deficiency":119},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,62,75,105,130,157,185,214,238,265,288,318],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":54,"completionDateStruct":56,"leadSponsor":58,"locationsCount":61},"100053904","phase-3-a-non-inferiority-pharmacokinetic-and-safetytolerability-study-of-two-different-doses-of-weekly-sc-alpha1-pi-15-compared-with-corresponding-standard-iv-alpha1-pi-in-participants-with-alpha1-antitrypsin-deficiency-aatd-100053904",false,"NCT07555483","A Non-inferiority Pharmacokinetic and Safety\u002FTolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)","An Open-Label, Multicenter, Randomized, Non-Inferiority Pharmacokinetic and Safety\u002FTolerability Study of Two Different Weekly Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Patients With Alpha1-Antitrypsin Deficiency Compared to Corresponding Standard 60 mg\u002Fkg\u002FWeek and 120 mg\u002Fkg\u002FWeek Doses of Intravenous Alpha1-Proteinase Inhibitor (5%)","SWIFT-SC","Inclusion Criteria:\n\n* Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or \"at-risk\" alleles (patients with \"at-risk\" alleles must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a blood draw for genotyping (i.e., allelic discrimination) and phenotyping will be obtained at the Screening Visit.\n* Participants may be naïve to alpha1-PI augmentation therapy or may be currently receiving alpha1-PI augmentation therapy or received alpha1-PI augmentation therapy in the past. If the total alpha1-PI serum (alpha-1 antitrypsin \\[AAT\\]) level has yet to be documented as in a treatment-naïve patient, a blood draw for total alpha1-PI serum level will be obtained at the Screening Visit. For participants currently receiving alpha1-PI augmentation, a pre-alpha1-PI augmentation AAT level must be documented in the participant's medical history\u002Frecords.\n* All participants must have a documented total alpha1-PI serum level \\\u003C11 μM (80mg\u002FdL if measured by radial immunodiffusion or 50 mg\u002FdL if measured by nephelometry) which is documented pre-alpha1-PI augmentation for participants receiving AAT augmentation.\n* At the Screening Visit, have post-bronchodilator Forced Expiratory Volume in 1 second (FEV₁) ≥25% and \\\u003C80% predicted of predicted FEV₁\u002FForced Vital Capacity (FVC) \\\u003C70% (Global Initiative for Chronic Obstructive Lung Disease \\[GOLD\\] stage II-III, and some individuals are GOLD stage IV).\n* If the participant has received alpha1-PI augmentation therapy of any kind, he\u002Fshe must be willing to discontinue that treatment at the Week 1 (Baseline) Visit and remain off any kind of alpha1-PI treatment, other than the IPs of this study, while participating in the study.\n* Willing and able to provide written informed consent indicating that they understand the purpose of, and procedures required for the study and are willing to participate in it.\n\nExclusion Criteria:\n\n* Have had a moderate or severe chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.\n* Have history of lung or liver transplant or on transplantation waiting list.\n* Have any lung surgery during the past 1 year (excluding lung biopsy).\n* At screening, have elevated liver enzymes (aspartate aminotransferase \\[AST\\], alanine aminotransferase \\[ALT\\], and alkaline phosphatase \\[ALP\\]) ≥ 2.5 times the upper limit of normal (ULN).\n* Have severe concomitant disease (e.g., congestive heart failure, clinically significant pulmonary fibrosis, malignant disease \\[except for skin cancers other than melanoma\\], history of acute hypersensitivity pneumonitis reaction, or current chronic hypersensitivity pneumonitis).\n* Females who are pregnant, breastfeeding or, if of child-bearing potential†, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam\u002Fgel\u002Ffilm\u002Fcream\u002Fsuppository, male sterilization, or true abstinence\\*) throughout the study. †Women of childbearing potential are defined as premenopausal and not surgically sterile, post tubal ligation, nor documented as infertile due to a concurrent medical condition.\n\n  \\*True abstinence: When this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence \\[e.g., calendar, ovulation, symptothermal, post-ovulation methods\\], declaration of abstinence for the duration of a study, and withdrawal are not acceptable methods of contraception.)\n* Have known previous infection with or clinical signs and symptoms consistent with current Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), or Human Immunodeficiency Virus (HIV) infection.\n* Have smoked during the past 6 months (this includes electronic\u002Fvapor cigarettes) or a positive urine cotinine test at the Screening Visit that is due to smoking.\n* Received IP in another study within 30 days prior to the Week 1 (Baseline) Visit or received any recombinant human AAT-Fc fusion protein (e.g., INBRX-101) or other extended half-life AAT products within 5 half-lives of the product relative to the Screening Visit date.\n* Have history of anaphylaxis or severe systemic response to any plasma-derived alpha1- PI preparation or other blood product(s).\n* Use systemic steroids above a stable dose equivalent to 5 mg\u002Fday prednisone (i.e., 10 mg every 2 days) within the 4 weeks prior to the Week 1 (Baseline) Visit (Note: inhaled steroids are not considered systemic steroids). It is recommended to maintain the same dose throughout the study.\n* Use systemic or aerosolized antibiotics for a COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.\n* Have known selective or severe Immunoglobulin A (IgA) deficiency based on prior medical records.\n* In the opinion of the Investigator, the participant may have compliance problems or any medical condition that may place them at safety risk with the protocol and the procedures of the protocol, or because of unstable health be unable to come to the study site for in-person clinic visits required by the protocol.","ALL","18 Years","80 Years",{"count":21,"type":22},40,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses of the same medicine given through a vein.\n\nAdults with Alpha-1 Antitrypsin Deficiency will take part. Participants will be randomly assigned to one of the treatment groups, and both the study doctors and participants will know which treatment is being given.\n\nThe main goals of the study are to understand how the body processes the medicine (pharmacokinetics) and to assess how safe and well tolerated the different weekly doses are.",[28],"Alpha 1 Antitrypsin Deficiency",[30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48],"Phase 3","Clinical Trial","Alpha1-Proteinase Inhibitor (Human)","Alpha 1-Antitrypsin","Subcutaneous","Intravenous","Pulmonary Emphysema","Emphysema","Pathologic Processes","Pulmonary Disease, Chronic Obstructive","Lung Diseases, Obstructive","Lung Diseases","Respiratory Tract Diseases","Genetic Diseases, Inborn","Subcutaneous Emphysema","Serine Proteinase Inhibitors","Protease Inhibitors","Enzyme Inhibition","Molecular Mechanisms of Pharmacological Action","RECRUITING","2026-07-10",{"date":52,"type":53},"2026-07-13","ACTUAL",{"date":55,"type":53},"2026-05-05",{"date":57,"type":22},"2027-09-30",{"name":59,"class":60},"Grifols Therapeutics LLC","INDUSTRY",22,{"id":63,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":64,"targetDuration":4,"studyType":23,"phases":65,"briefSummary":26,"conditions":66,"keywords":67,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":72,"leadSponsor":73,"locationsCount":74},"100635653",{"count":21,"type":22},[25],[28],[30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48],"2026-05-28",{"date":70,"type":53},"2026-06-01",{"date":55,"type":53},{"date":57,"type":22},{"name":59,"class":60},19,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":81,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":83,"enrollmentInfo":84,"targetDuration":4,"studyType":23,"phases":86,"briefSummary":88,"conditions":89,"keywords":90,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":104},"100626090","phase-1-a-study-of-air-001-in-adults-with-alpha-1-antitrypsin-deficiency-aatd-100626090","NCT07431112","A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)","Phase 1, Open-Label, Single Ascending Dose and Multiple Dose Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered AIR-001 in Adults With AATD Due to PiZZ Genotype","RepAIR1","Inclusion Criteria:\n\n1. Male or female participants \\>18 years and \\\u003C75 years of age at the time of signing informed consent\n2. Total serum AAT levels \\\u003C 11µM (57 mg\u002FdL)\n3. Pi\\*ZZ genotype confirmed by DNA sequencing within the SERPINA1 gene with no known co-occurring SERPINA1 null variants\n4. Spirometry: Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted\n5. Non-smoker, including vaping, for at least 6 months prior to screening\n6. Body mass index between 18-33.0 kg\u002Fm²\n7. Body weight ≥ 45 kg and ≤110 kg\n8. Willing and able to give written informed consent prior to the initiation of any study procedure by the participant\n9. Negative beta human chorionic gonadotropin (β-hCG) at enrolment for women of childbearing potential (WOCBP) only.\n10. Participants who are either a WOCBP or male participant who is heterosexually active with a WOCBP must consent to use a highly effective method of contraception from screening visit until at least 4 weeks after the last dose of investigational medicinal product (IMP).\n11. Willing and able to comply with the study design schedule, all study procedures, and other requirements\n\nExclusion Criteria:\n\n1. Female participants who are nursing or lactating\n2. Participant has received AAT augmentation therapy within 30 days prior to Screening Visit or plans to receive AAT augmentation therapy at any time during study participation.\n3. Known or suspected allergy or intolerance to AIR-001 or its components\n4. Acute respiratory tract infection or clinically-diagnosed chronic obstructive pulmonary disease (COPD) exacerbation that required antibiotic treatment and\u002For systemic corticosteroids within the 8 weeks prior to dosing.\n5. Positive screening test for COVID-19 and\u002For Influenza.\n6. Lung disease that requires use of continuous oral corticosteroids, continuous supplemental oxygen, day-time ventilatory support, or any participant who is on a lung transplant waiting list.\n7. Liver Fibrosis score \\> 10 kPa defined by screening liver elastography, historical liver biopsy showing ≥ F3 fibrosis (METAVIR or comparable scoring system), or established diagnosis of hepatic cirrhosis.\n8. Any of the following screening laboratory abnormalities:\n\n   1. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) \\> 3 x upper limit of normal (ULN)\n   2. Total bilirubin \\> ULN (note: for participants with documented Gilbert's syndrome and direct bilirubin ≤ ULN , exclusion criterion is total bilirubin is \\> 2.5 mg\u002FdL)\n   3. INR \\> ULN (for participants taking stable doses of anticoagulants, the exclusion criterion is INR \\> 3.0)\n   4. Platelet count ≤ 150 k\u002FμL\n   5. Estimated glomerular filtration rate (eGFR) ≤ 60 mL\u002Fmin\u002F1.73m² by Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) equation\n   6. Urine Albumin-to-Creatinine Ratio \\> 300 mg\u002Fg\n   7. Urine Protein-to-Creatinine Ratio \\> 500 mg\u002Fg\n9. Prolonged QT interval on electrocardiogram (ECG), defined as QTcF ≥ 450ms (men) or ≥ 470ms (women)\n10. ECG findings at screening that render measurements of QT interval imprecise.\n11. History of congestive heart failure, serious cardiac arrythmias requiring anti-arrhythmic medications or unexplained black-outs or fainting episodes with a suspected cardiac origin\n12. Positive screening test or known chronic infection with Hepatitis B, Hepatitis C, or HIV.\n13. Known history of coagulopathy or bleeding diathesis\n14. History or intolerance to subcutaneous (SC) injection including relevant dermatological conditions affecting standard injection sites\n15. History or presence of any medical condition, behavioral or psychiatric disorder, or planned surgical procedure or surgical history that may interfere with participation in the study or interpretation of study results, and\u002For put the participant at significant risk (in the opinion of the investigator) if he\u002Fshe participates in the study.\n16. History of any lung-volume reduction procedure in the 6 months prior to screening.\n17. Laboratory value(s) outside the laboratory reference range that is (are) considered to be clinically significant and may affect the safety, efficacy, PK, or PD assessments or interpretation by the Investigator, at screening\n18. History of alcohol or drug abuse within the past three months\n19. Current or previous participation in any other clinical study where the participant has received a dose of an IMP within 3 months or 5 half-lives of the IMP, whichever is longest, prior to Screening Visit\n20. Any previous gene replacement or DNA-editing therapy\n21. Any previous use of an RNA-based therapeutic (except for AIR-001 or RNA-based vaccines) within the 6 months prior to the Screening Visit or at any time if stopped due to drug-related adverse event.\n22. Use of any new prescription, vaccine, herbal remedy, over-the-counter medication, or supplement, or changes in chronic therapies within the 28 days prior to dosing unless approved by study Medical Monitor.","74 Years",{"count":85,"type":22},54,[87],"PHASE1","This is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype.",[28],[41,91,42,92,93,44,37,38,94,28],"Liver Diseases","Genetic Disease","Inborn Congenital, Hereditary, Neonatal Diseases and Abnormalities","Pathological Conditions, Signs and Symptoms","2026-04-27",{"date":97,"type":53},"2026-04-28",{"date":99,"type":53},"2026-03-17",{"date":101,"type":22},"2029-01",{"name":103,"class":60},"AIRNA Corporation",4,{"id":106,"slug":107,"hasResults":11,"nctId":108,"briefTitle":109,"officialTitle":110,"acronym":4,"eligibilityCriteria":111,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":112,"enrollmentInfo":113,"targetDuration":4,"studyType":23,"phases":115,"briefSummary":117,"conditions":118,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":120,"lastUpdatePostDateStruct":121,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":129},"100546049","phase-1-a-study-to-evaluate-the-safety-and-efficacy-of-beam-302-in-adult-patients-with-alpha-1-antitrypsin-deficiency-aatd-100546049","NCT06389877","A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)","A Phase 1\u002F2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and\u002For Liver Disease","Part A:\n\nInclusion Criteria:\n\n* Males or females 18 - 70 years of age inclusive at the time of consent.\n* Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).\n* Blood total AAT level \\\u003C11 μM or equivalent protein in mg\u002FdL.\n* Patients receiving augmentation therapy in regions where augmentation is not SoC must be willing to washout augmentation therapy for at least 6 weeks prior to signing the ICF and for the length of the study (unless clinically indicated)\n* A postbronchodilator FEV1 ≥40% of predicted and an FEV1\u002FFVC \\\u003C70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n* Evidence of emphysema on a historic CT scan or a DLCO ≤70% of the predicted value (corrected for hemoglobin) at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n\nExclusion Criteria:\n\n* Body mass index \\>30\n* Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.\n* Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\]).\n* Liver disease with any of the following:\n\n  * FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI \\>0.5 can be used as a surrogate exclusion criterion \\[Yilmaz, 2011\\].\n  * Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).\n  * Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.\n  * Have ALT or AST \\> upper limit of normal (ULN).\n  * Total bilirubin levels \\> ULN; if documented Gilbert's Syndrome, total bilirubin \\>2 × ULN.\n  * INR ≥1.2 at screening. If deemed appropriate by the investigator and\u002For prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within \\\u003C1.2 would be acceptable.\n  * Seropositive for hepatitis B (positive surface Ag).\n  * Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative.\n\nPart B:\n\nInclusion Criteria:\n\n* Males or females 18 - 70 years of age inclusive at the time of consent.\n* Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).\n* Evidence of METAVIR F1, F2, or F3 liver fibrosis based on a central read of a baseline liver biopsy during the screening period or a histological diagnosis made no more than 6 months before enrollment and stage confirmed by central read.\n* A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n\nExclusion Criteria:\n\n* Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.\n* Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\])\n* Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).","70 Years",{"count":114,"type":22},106,[87,116],"PHASE2","This is a Phase 1\u002F2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK\u002FPD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and\u002For liver disease and to determine the optimal biological dose (OBD).",[119],"Alpha 1-Antitrypsin Deficiency","2026-03-18",{"date":122,"type":53},"2026-03-20",{"date":124,"type":53},"2024-06-19",{"date":126,"type":22},"2030-05",{"name":128,"class":60},"Beam Therapeutics Inc.",11,{"id":131,"slug":132,"hasResults":11,"nctId":133,"briefTitle":134,"officialTitle":134,"acronym":4,"eligibilityCriteria":135,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":112,"enrollmentInfo":136,"targetDuration":4,"studyType":23,"phases":138,"briefSummary":139,"conditions":140,"keywords":141,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":156},"100592694","phase-1-gene-therapy-for-alpha-1--antitrypsin-deficiency-100592694","NCT06996756","Gene Therapy for Alpha 1- Antitrypsin Deficiency","Inclusion Criteria:\n\n* AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \\\u003C11 μM\n* Emphysema as assessed by chest high resolution computational tomography (HRCT)\n* Lung function parameters consistent with mild to moderate loss of lung function and the presence of emphysema.\n* Troponin T within normal limits\n* Normal liver ultrasound and serum alpha fetoprotein\n* Normal kidney function\n* No contraindications to receiving corticosteroid immunosuppression\n\nExclusion Criteria:\n\n* Individuals receiving systemic corticosteroids or other immunosuppressive medications for pre-existing conditions.\n* Inability to tolerate immunosuppression with corticosteroids (e.g., uncontrolled diabetes)\n* Individuals with an immunodeficiency disease, or evidence of active infection of any type, including human immunodeficiency virus\n* Evidence of major central nervous system, major psychiatric, musculoskeletal or immune disorder\n* Prior history of myocardial infarction or cancer within the past 5 years (other than basal cell carcinoma of the skin)\n* Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment)\n* Abnormal ECG at screening with findings consistent with cardiac disease\n* Females who are currently pregnant or lactating\n* Any history of allergies to drugs used for bronchoscopy, including xylocaine, lidocaine, versed, valium, atropine, pilocarpine, isoproterenol, terbutaline, aminophylline, or any local anesthetic\n* Individuals receiving experimental medications or participating in another experimental protocol for at least 3 months prior to entry to the study\n* Use of oxygen supplementation\n* Risk for thromboembolic disease\n* History of significant cardiovascular disease, hypertension, prior myocardial infarction and\u002For cerebrovascular event\n* Individuals who are currently on beta-blockers, or other cardiac therapy related drugs\n* Prior history of hypersensitivity or anaphylaxis associated with the administration of any AAT product",{"count":137,"type":22},16,[87],"This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis. We hope to learn the safety\u002Ftoxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin deficient individuals.",[119],[142,37,143,144,145],"AAT","Gene therapy","DLCO","Augmentation therapy","2026-03-11",{"date":148,"type":53},"2026-03-13",{"date":150,"type":53},"2025-02-26",{"date":152,"type":22},"2032-08-01",{"name":154,"class":155},"Weill Medical College of Cornell University","OTHER",1,{"id":158,"slug":159,"hasResults":11,"nctId":160,"briefTitle":161,"officialTitle":162,"acronym":4,"eligibilityCriteria":163,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":164,"targetDuration":166,"studyType":167,"phases":4,"briefSummary":168,"conditions":169,"keywords":171,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":175,"lastUpdatePostDateStruct":176,"startDateStruct":178,"completionDateStruct":180,"leadSponsor":182,"locationsCount":184},"100554939","pimz-longitudinal-cohort-pimz-logic-100554939","NCT06505603","PiMZ Longitudinal Cohort (PiMZ Logic)","Alpha-1 Antitrypsin PiMZ Longitudinal Cohort (PiMZ Logic)","Inclusion Criteria:\n\n1. Males and females aged 18 years and older\n2. Understand the study procedures, risks, benefits, purpose\n3. Able and willing to comply with the study procedures\n4. Have PiMZ alpha-1 antitrypsin deficiency\n5. Post bronchodilator FEV1 \\\u003C 80% predicted AND post bronchodilator FEV1\u002FFVC \\\u003C 70%\n6. Be an existing member of the Alpha-1 Foundation Clinical Cohort (also known as the Alpha-1 Foundation Research Registry)\n7. Agree to have the data collected in this study be shared with the Alpha-1 Foundation Research Registry\n\nExclusion Criteria:\n\n1. AATD non-PiMZ status, including carriers\n2. Current lung, hematologic, or solid organ malignancy other than skin or cervical Stage 1 cancers within the past 3 years\n3. COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit\n4. Pregnancy at the time of the screening visit\n5. Inability to lie still in a supine position for 15 minutes during CT acquisition\n6. Inability to perform quality-controlled lung function testing\n7. Allergy to albuterol\n8. Currently receiving intravenous or subcutaneous immunoglobulin for any disease state\n9. Past or present major surgery on the lungs including pneumonectomy or lobectomy. Wedge resections, past segmentectomy, and pleurodesis surgeries are allowed.\n10. Previous lung or liver transplantation or currently on the transplant list\n11. Decompensated cirrhosis\n12. Current presence of endobronchial coils or valves in the lung\n13. Clinically significant bronchiectasis as defined by the investigator. In general, this would exclude patients with chronic infection of the lungs requiring treatment within the past 6 months including non-tuberculous mycobacterial disease, chronic fungal disease, allergic bronchopulmonary aspergillosis, or known colonization of bronchiectasis with pseudomonas or stenotrophomonas species.\n14. Participation in the active treatment arm of a therapeutic clinical trial at baseline visit unless using one of the Alpha-1 augmentation therapies in alternative doses.\n15. Patient with Automatic Implantable Cardioverter Defibrillator (AICD) and permanent pacemakers (PPM)\n16. Patient receiving biologic immunomodulators that will affect the assessment of the serum biomarkers (as determined by the site PI)\n17. Patient with pleural catheters\n18. Any condition that in the opinion of the investigator might adversely influence the study outcome",{"count":165,"type":22},80,"3 Years","OBSERVATIONAL","Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. Presentations are variable in the heterozygous population, the most predominant genotype being PiMZ. The purpose of this study in PiMZ heterozygous patients is to examine the density of the lung as measured by chest computed tomography (CT) and determine if existing emphysema predicts changes in the rate of subsequent emphysema or changes in CT, serum or plasma biomarkers of interest. The overarching goal is to develop biomarkers pertinent to the PiMZ patient that can be used in interventional trials since lung function changes do not typically inform disease progression in AATD.",[119,170],"Emphysema or COPD",[172,173,174],"Alpha-1","Biomarker","PiMZ","2026-01-15",{"date":177,"type":53},"2026-01-16",{"date":179,"type":53},"2025-05-30",{"date":181,"type":22},"2029-02",{"name":183,"class":155},"Columbia University",5,{"id":186,"slug":187,"hasResults":11,"nctId":188,"briefTitle":189,"officialTitle":189,"acronym":190,"eligibilityCriteria":191,"healthyVolunteers":192,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":193,"targetDuration":195,"studyType":167,"phases":4,"briefSummary":196,"conditions":197,"keywords":199,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":205,"lastUpdatePostDateStruct":206,"startDateStruct":208,"completionDateStruct":210,"leadSponsor":212,"locationsCount":156},"100095205","the-alpha-1-foundations-and-university-of-floridas-alpha-1-coded-testing-act-study-100095205","NCT00500123","The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study","ACT","Inclusion Criteria:\n\n* Individuals of any age at risk for alpha-1 antitrypsin deficiency on the basis of symptoms or family genetic risk.\n\nExclusion Criteria:\n\n* Any person who has already had genotype and AAT level testing completed and has a qualified result.",true,{"count":194,"type":22},50000,"50 Years","The Alpha-1 Coded Testing (ACT) Study was established to study genetic testing and outcomes of individuals at risk for alpha-1 antitrypsin deficiency.",[198],"Alpha-1 Antitrypsin Deficiency",[200,201,142,202,203,204],"alpha-1","antitrypsin","genetic testing","alpha-1 antitrypsin deficiency","AATD","2025-11-21",{"date":207,"type":53},"2025-11-24",{"date":209,"type":53},"2001-01",{"date":211,"type":22},"2050-01",{"name":213,"class":155},"University of Florida",{"id":215,"slug":216,"hasResults":11,"nctId":217,"briefTitle":218,"officialTitle":219,"acronym":4,"eligibilityCriteria":220,"healthyVolunteers":192,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":221,"targetDuration":4,"studyType":167,"phases":4,"briefSummary":223,"conditions":224,"keywords":227,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":230,"lastUpdatePostDateStruct":231,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":156},"100197847","lung-disease-and-its-affect-on-the-work-of-white-blood-cells-in-the-lungs-100197847","NCT01851642","Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs","The Role of Conformational Diseases on Macrophage Function","Inclusion Criteria:\n\n* Signed informed consent\n* Male or female 18 years of age or older\n* Negative pregnancy test for women of childbearing potential\n* Hemoglobin \\>12.5 g\u002Fdl measured on the day of participation\n* Negative urine nicotine test\n\nExclusion Criteria:\n\n* Pregnancy or breastfeeding\n* Weight \\\u003C 50 kg\n* History of anemia requiring blood transfusions, erythropoietin supplementation, or iron supplementation within the past 36 months\n* Known hemoglobin \\\u003C12.5 g\u002Fdl within the past 90 days\n* Systolic blood pressure \\> 180 mmHg and\u002For diastolic blood pressure \\>100 mmHg\n* Poor venous access\n* Large volume blood donation (\\>200 ml or 7 ounces) within the previous 56 days (e.g. blood donation for the purposes of blood banking)\n* Clinically significant cardiac, hemostatic or neurological impairment or any other significant medical condition that, in the opinion of the investigator would affect subject safety (e.g., recent myocardial infarction, history of prolonged bleeding time, cerebral vascular accident, advanced cancer or uncontrolled medical condition)\n* Psychiatric or cognitive disturbance or illness that would affect subject safety\n* Current smoker",{"count":222,"type":22},220,"The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macrophages, and their ability to work.",[198,225,204,226],"AAT Deficiency","Cystic Fibrosis (CF)",[228,198,229],"Macrophage","Cystic Fibrosis","2025-11-05",{"date":232,"type":53},"2025-11-06",{"date":234,"type":53},"2007-08-09",{"date":236,"type":22},"2033-07-20",{"name":213,"class":155},{"id":239,"slug":240,"hasResults":11,"nctId":241,"briefTitle":242,"officialTitle":243,"acronym":244,"eligibilityCriteria":245,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":246,"enrollmentInfo":247,"targetDuration":4,"studyType":23,"phases":248,"briefSummary":249,"conditions":250,"keywords":251,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":255,"lastUpdatePostDateStruct":256,"startDateStruct":258,"completionDateStruct":260,"leadSponsor":262,"locationsCount":264},"100378184","phase-3-phase-iii-efficacy-and-safety-of-kamada-aat-for-inhalation-100378184","NCT04204252","Phase III, Efficacy and Safety of \"Kamada-AAT for Inhalation\"","Prospective Phase 3 Multi-center 2-Year Placebo Controlled Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation 80 mg\u002FDay in Alpha-1 Antitrypsin Deficiency With Moderate and Severe Airflow Limitation Followed by a 2-Year Open Label Extension","InnovAATe","Inclusion Criteria\n\n1. Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z\u002FNull), or Pi(Null\u002FNull) genotypes.\n2. Serum AAT levels ≤ 11 µM at screening.\n3. Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1\u002FSVC≤70%) at screening.\n4. 40% ≤ FEV1 ≤ 80% of predicted post-bronchodilator at screening.\n5. Patients who are either naïve or washed out of any AAT treatment for at least 8 weeks prior to randomization.\n6. Age between 18 to 65 years inclusive at screening.\n7. Able to read and sign informed consent and willing to participate in the study.\n8. Males or non-pregnant, non-lactating females whose screening pregnancy test is negative, who are willing to use contraceptive methods for the duration of the study, or who are postmenopausal, or surgically sterilized.\n9. Study medication use for at least 20 out of the 28 days of run-in, as recorded in the study nebulization PARI Track data.\n10. Demonstrated ability to complete eDiary for at least 20 out of the first 28 days of run-in.\n\nExclusion Criteria\n\n1. Immunoglobulin A (IgA) absolute deficiency defined as serum IgA levels \\\u003C 0.05 g\u002FL.\n2. History of life-threatening transfusion reaction(s), allergy, anaphylactic reaction, or systemic response to human plasma-derived products.\n3. Two or more moderate or any severe exacerbation(s) within the year prior to baseline.\n4. A moderate exacerbation within 6 weeks prior to baseline.\n5. Use of oral or parenteral glucocorticoids in doses above 10 mg of prednisone daily or equivalent generics (substance and dose).\n6. Clinically significant inter-current illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal, or other. Patients might be included after consultation with the treating physician and the sponsor if, in the opinion of the Investigator, their condition will not interfere with the safety, compliance or other aspects of this study.\n7. Hospitalization for any cause during the 6 weeks prior to screening.\n8. History of lung or liver transplant.\n9. On any thoracic or hepatic surgery waiting list.\n10. Any lung surgery within the past two years (including bronchoscopic lung volume reduction).\n11. Any smoking within the year prior to screening.\n12. Evidence of alcohol abuse or history of alcohol abuse, or use of illegal drugs and\u002For abuse of legally prescribed drugs in the last 5 years prior to screening.\n13. Acute or chronic hepatitis (hepatitis A, hepatitis B, hepatitis C), or positive human immunodeficiency virus (HIV) serology.\n14. Signs of significant abnormalities in serum hematology, serum chemistry, serum inflammatory \u002F immunogenic markers and urinalysis per investigator judgment, taking into considerations the potential effects of the AAT deficiency.\n15. Signs of significant abnormalities in ECG per investigator judgment at screening.\n16. Presence of psychiatric\u002F mental disorder or any other medical disorder that might impair the patient's ability to give informed consent or to comply with the requirements of the study protocol. If, in the opinion of the Investigator, the condition will not interfere with the compliance or other aspects of this study, the patient might be included after consultation with the treating physician and the sponsor.\n17. Participation in another clinical trial involving investigational medication or interventional treatment within 30 days and\u002For last dose 5 half-lives prior to screening visit.\n18. Inability to attend scheduled clinic visits and\u002For comply with study protocol.\n19. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol.\n\nAdditional eligibility criteria apply for the open label extension","65 Years",{"count":222,"type":22},[25],"The goal of this clinical trial is to learn if AAT for inhalation, at a dose of 80 mg\u002Fday can slow the progression of lung disease in people who have lung disease caused by severe genetic deficiency in Alpha 1 Antitrypsin (AATD).\n\nThe main question it aims to answer is:\n\n• Can daily treatment with Kamada AAT for inhalation at a dose of 80 mg\u002Fday prevent or slow lung function worsening ? Lung function will be measured by spirometry.\n\nOther questions it aims to answer are:\n\n* Can daily treatment with Kamada AAT for inhalation at a dose of 80 mg\u002Fday prevent or slow lung density loss ? Lung density will be measured by a CT scan.\n* Can daily treatment with Kamada AAT for inhalation at a dose of 80 mg\u002Fday prevent or slow lung disease from worsening ? Lung disease will be measured using spirometry, lung volume, gas diffusion, six minute walk test, quality of life questionaires and biomarkers.\n* What medical problems do participants have when taking AAT for inhalation 80 mg\u002Fday daily ? Researchers will compare AAT for inhalation to a placebo (a look-alike substance that contains no drug) to see if AAT for inhalation works to treat AAT-deficiency related lung disease. Study participants will receive either AAT for inhalation or placebo for the first two years of the study. During the third and fourth years of the study all participants will receive AAT for inhalation regardless of which drug they received during the first two years.\n\nParticipants will:\n\n* Inhale the study drug every day\n* Clean and disinfect the nebulizer every day\n* Document daily symptoms and study drug use in an electronic diary\n* Visit the clinic for tests and assessments. There are 11 clinic visits during the first two years of the study and 5-6 clinic visits during the third and fourth year, combined. After treatment ends, participants will visit the clinic 3 times in half a year.",[119],[252,253,254],"Alpha 1 Antitrypsin","Inhaled Alpha 1 Antitrypsin","Nebulizer","2025-08-27",{"date":257,"type":53},"2025-09-04",{"date":259,"type":53},"2019-11-25",{"date":261,"type":22},"2031-06",{"name":263,"class":60},"Kamada, Ltd.",9,{"id":266,"slug":267,"hasResults":11,"nctId":268,"briefTitle":269,"officialTitle":270,"acronym":271,"eligibilityCriteria":272,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":112,"enrollmentInfo":273,"targetDuration":4,"studyType":23,"phases":275,"briefSummary":276,"conditions":277,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":278,"lastUpdatePostDateStruct":279,"startDateStruct":281,"completionDateStruct":283,"leadSponsor":285,"locationsCount":287},"100519863","phase-1-a-study-of-kb408-for-the-treatment-of-alpha-1-antitrypsin-deficiency-100519863","NCT06049082","A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency","A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency","Serpentine-1","Inclusion Criteria:\n\n1. The subject or legally authorized representative must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and instructions.\n2. Subject is aged ≥18 to ≤70 years, at the time of informed consent.\n3. Subject has a genetically confirmed diagnosis of AATD with a PI\\*ZZ or PI\\*ZNull genotype.\n4. Cohort 2b and Cohort 3: Subjects receiving AAT augmentation therapy must be willing to washout for at least 10 days prior to Screening and be willing to remain off augmentation therapy for the duration of the study.\n5. Cohort 2b and Cohort 3: Serum AAT level \\\u003C11 μM at Screening.\n6. Willing to remain on a stable regimen of treatment during the study.\n7. Resting oxygen saturation ≥92% on room air at Screening.\n8. Clinically stable and in good general health, except for AATD, as determined by the Investigator.\n\nExclusion Criteria:\n\n1. Pulmonary function test with percent predicted forced expired volume in 1 second (ppFEV1) after inhalation of a bronchodilator is \\\u003C40% at Screening.\n2. Diffusing capacity of the lungs for carbon monoxide (DLCO) \\\u003C30 percent predicted (historical DLCO within 2 years prior to Screening without any intervening change in clinical status since the measurement was taken, or as measured at Screening).\n3. Known ongoing or history of clinically significant pulmonary impairment other than AATD.\n4. A pulmonary exacerbation within six weeks (42 days) of first dose.\n5. Initiation of any new chronic therapy or any change in ongoing therapy routine within 28 days of first dose.\n6. Participation in another interventional clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, of first dose. Previous treatment with a genetic therapy for AATD, where the investigational product was demonstrated to be non-efficacious, is not exclusionary.\n7. History of or listed for solid organ transplantation or has undergone major lung surgery (e.g., lobectomy) within 6 months of first dose.\n8. Any clinical condition or illness (including a history or current evidence of substance abuse or dependence) that, in the opinion of the Investigator, would impact a subject's ability to complete all study-related procedures and\u002For poses an additional risk to the assessment of safety of KB408.\n9. An active oral herpes infection 30 days prior to the first dose.\n10. Clinically significant hepatic dysfunction defined as any one of the following:\n\n    1. AST and ALT ≥3× upper limit of normal (ULN) at Screening\n    2. Total bilirubin ≥2× ULN at Screening (unless associated with Gilbert's syndrome)\n    3. Evidence of liver cirrhosis with clinical manifestations of portal hypertension (e.g., ascites, encephalopathy, variceal hemorrhage)\n11. History of cigarette smoking or any other tobacco use, or use of e-cigarettes or other recreational inhalant, within 6 months of Screening.\n12. Unwilling to refrain from smoking, e-cigarette use, or vaping throughout the duration of the study.\n13. A positive urine cotinine result that is consistent with active smoking at Screening. (A positive cotinine test due to nicotine replacement therapy for the purpose of smoking cessation, as attested by the Investigator, is allowed.)\n14. Abnormal hematology or chemistry testing at Screening as defined below, or any other clinically significant abnormalities that the Investigator believes may interfere with the assessment of safety of the study treatment.\n\n    * Platelet count \\\u003C100×10\\^9\u002FL\n    * Hemoglobin \\\u003C9 g\u002FdL\n    * White blood cell count \\\u003C3 or \\>15×10\\^9\u002FL\n    * Sodium \\\u003C130 or \\>150 mmol\u002FL\n    * Potassium \\\u003C3 or \\>5.5 mmol\u002FL\n    * Carbon dioxide \\\u003C16 mmol\u002FL\n    * Creatinine \\>2 mg\u002FdL\n15. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.\n16. Females who are pregnant or nursing.\n17. Subject who is unwilling to comply with contraception requirements per protocol",{"count":274,"type":22},15,[87],"The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI\\*ZZ or PI\\*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Repeat dosing will be evaluated at the mid dose level. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. In the repeat dose and the high dose cohorts, subjects must wash out from IV AAT for at least 10 days, as applicable.",[119],"2025-07-17",{"date":280,"type":53},"2025-07-22",{"date":282,"type":53},"2024-02-15",{"date":284,"type":22},"2026-12",{"name":286,"class":60},"Krystal Biotech, Inc.",3,{"id":289,"slug":290,"hasResults":11,"nctId":291,"briefTitle":292,"officialTitle":293,"acronym":4,"eligibilityCriteria":294,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":295,"targetDuration":297,"studyType":167,"phases":4,"briefSummary":298,"conditions":299,"keywords":300,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":309,"lastUpdatePostDateStruct":310,"startDateStruct":312,"completionDateStruct":314,"leadSponsor":316,"locationsCount":156},"100374560","alpha-1-research-registry-100374560","NCT04157049","Alpha-1 Research Registry","Alpha-1 Research Registry Protocol","Inclusion Criteria:\n\n* Patients diagnosed with Alpha-1 Antitrypsin Deficiency (PiZZ, PiZNull, PiSZ etc.)\n* Alpha-1 carriers (PiMZ, PiMS etc.)\n\nExclusion Criteria:\n\n* Failure to provide informed consent\n* Normal healthy individuals (MM)",{"count":296,"type":22},4000,"2 Years","The Alpha-1 Research Registry is a confidential database made up of individuals diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and individuals identified as Alpha-1 carriers. The Registry was established to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1.",[119],[204,301,302,142,303,304,305,306,307,308],"ALPHA 1","COPD","ZZ","MZ","CARRIER","MM","MS","PiZZ","2024-01-29",{"date":311,"type":53},"2024-01-30",{"date":313,"type":53},"2019-06-20",{"date":315,"type":22},"2029-06-20",{"name":317,"class":155},"Alpha-1 Foundation",{"id":319,"slug":320,"hasResults":11,"nctId":321,"briefTitle":322,"officialTitle":323,"acronym":4,"eligibilityCriteria":324,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":325,"targetDuration":327,"studyType":167,"phases":4,"briefSummary":328,"conditions":329,"keywords":331,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":334,"lastUpdatePostDateStruct":335,"startDateStruct":337,"completionDateStruct":339,"leadSponsor":341,"locationsCount":156},"100452958","czech-aatd-registry-100452958","NCT05178277","Czech AATD Registry","Czech Alpha-1 Antitrypsin Deficiency Registry, the National Observational Study.","Inclusion Criteria:\n\n* Patients with alpha-1-antitrypsin deficiency\n\nExclusion Criteria:\n\n* Patient disagreement with inclusion in the study",{"count":326,"type":22},300,"20 Years","Alpha-1-antitrypsin deficiency is the most common congenital disease of the respiratory system, leading to early pulmonary emphysema or bronchiectasis. Pulmonary involvement significantly accelerates active cigarette smoking. Patients with alpha-1-antitrypsin deficiency may also have liver cirrhosis, vasculitis, skin or intestinal disorders.\n\nThe AATD Registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency.\n\nThe aim of the AATD National Registry is to collect and analyze clinical data in patients with alpha-1 antitrypsin deficiency.",[330],"Alpha-1-antitrypsin Deficiency",[332,204,333],"Alpha-1-antitrypsin deficiency","national registry","2021-12-16",{"date":336,"type":53},"2022-01-05",{"date":338,"type":53},"2018-01-01",{"date":340,"type":22},"2035-12-31",{"name":342,"class":155},"Thomayer University Hospital"]