Alpha-1 Anti-trypsin Deficiency (DBCOND0066043)

Identifiers

Synonyms
Alpha-1 AT Deficiency / Alpha₁-Antitrypsin Deficiency / Alpha 1-Antitrypsin (AAT) Deficiency / Alpha 1-antitrypsin Deficiency (AATD) / Alpha-1 Antitrypsin Deficiency (AATD) / Alpha1-antitrypsin Deficiency / Alpha1 Anti-Trypsin Deficiency / Alpha-1 Antitrypsin Deficiency / Alpha 1 Antitrypsin Deficiency / Alpha-1-Antitrypsin Deficiency / Alpha-1-antitrypsin-deficiency / Alpha 1-Antitrypsin Deficiency / Alpha-1 anti-trypsin deficiency / Alpha-1-Antitrypsin deficiency (disorder) / Alpha-1 proteinase inhibitor deficiency

Associated Data

Indicated Drugs and Targets
DrugDescriptionTargets
Recombinant alpha 1-antitrypsin
A medication indicated to treat patients with chronic alpha1-proteinase inhibitor deficiency.
Clinical Trials
IdentifierTitleDrug(s)PurposePhaseStatus
NCT01455298
Liver Disease in Adults With Alpha-1 AT DeficiencyNo drug interventionsscreeningNot Availablecompleted
NCT02282527
A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiencytreatment2 / 3completed
NCT01213043
Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiencytreatment2completed
NCT03679598
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiencytreatment2completed
NCT05899673
An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Diseasetreatment3active_not_recruiting
NCT06165341
Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)treatment3recruiting
NCT06512454
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver ProblemsNo drug interventionsNot AvailableNot Availablerecruiting
NCT05677971
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Proteintreatment3recruiting
NCT05466747
A Study of RYMPHYSIA for Alpha1-Proteinase Inhibitor (A1PI) Therapy in Adults With A1PI Deficiency and Chronic Obstructive Pulmonary Disease (COPD)-Emphysematreatment4withdrawn
NCT04474197
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ GenotypeNo drug interventionstreatment2completed
NCT02525861
GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Studytreatment3completed
NCT02870309
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiencytreatment1 / 2completed
NCT02870348
Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)treatment1 / 2completed
NCT00313144
Aralast alpha1-proteinase Inhibitor Surveillance Studytreatment4terminated
NCT02722304
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiencytreatment3terminated
NCT04722887
A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiencytreatment1 / 2recruiting
NCT01651351
GLASSIA Infusion Rate Studytreatment4completed
NCT04440488
ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Studytreatment4withdrawn
NCT00161707
Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiencytreatment1 / 2completed
NCT00157092
Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiencytreatment1 / 2completed
NCT02929940
Liver Disease in Patients With alpha1-antitrypsin DeficiencyNo drug interventionsNot AvailableNot Availableunknown_status
NCT02922751
FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)No drug interventionsNot AvailableNot Availablecompleted
NCT04443192
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ SubjectsNo drug interventionstreatment1terminated
NCT03823443
COPD Exacerbation Blood and Urine Biomarkers StudyNo drug interventionsNot AvailableNot Availableterminated
NCT01974830
AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient RegistryNo drug interventionsNot AvailableNot Availablecompleted
NCT06622668
A Phase 1/2 Study of NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung DiseaseNo drug interventionstreatment1 / 2not_yet_recruiting
NCT05643495
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ GenotypeNo drug interventionstreatment2completed
NCT01615484
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant SuitabilityNo drug interventionstreatmentNot Availablecompleted
NCT06405633
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATDNo drug interventionstreatment1 / 2recruiting
NCT02900183
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin LevelsNo drug interventionstreatment2withdrawn
NCT01810458
Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)Not AvailableNot Availablecompleted
NCT01419158
Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD)No drug interventionsNot AvailableNot Availablecompleted
NCT01054339
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiencytreatment2completed
NCT00188825
Study Comparing Simulect Plus Standard Immunosuppression to Standard Immunosuppression Alone for the Prevention of Acute Rejection and Bronchiolitis Obliterans in Lung Transplanttreatment3completed
NCT00005292
Alpha1-antitrypsin Deficiency RegistryNo drug interventionsNot AvailableNot Availablecompleted
NCT03804021
Long-Term Follow-up Study of ADVM-043No drug interventionsNot AvailableNot Availablecompleted
NCT04262284
Respreeza® Self-administration and Learning Program (AmAREtTI Study)No drug interventionsNot AvailableNot Availableactive_not_recruiting
NCT00532805
The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin DeficiencyNo drug interventionsNot AvailableNot Availablecompleted
NCT02001688
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"No drug interventionstreatment2completed
NCT02014415
Alpha-1 Antitrypsin Deficiency Adult Liver StudyNo drug interventionsNot AvailableNot Availableactive_not_recruiting
NCT03008915
Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiencyother2completed
NCT02547532
Microbioma in Sputa From COPD With Alpha-1 Antitrypsin DeficiencyNo drug interventionsNot AvailableNot Availablecompleted
NCT05727800
A Phase 1, First-in-human Study of VX-668No drug interventionstreatment1completed
NCT01851642
Lung Disease and Its Affect on the Work of White Blood Cells in the LungsNot AvailableNot Availablerecruiting
NCT01241942
Phase 2 Study of Ex-vivo Perfusion and Ventilation of Lungs to Assess Transplant SuitabilityNo drug interventionsdevice_feasibilityNot Availableterminated
NCT03815396
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiencytreatment1completed
NCT02810327
Alpha-1 Carrier Genomics StudyNo drug interventionsNot AvailableNot Availablecompleted
NCT06186492
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)No drug interventionstreatment1recruiting
NCT02502201
Environment Effect on Six-Minute Walk Test PerformanceNo drug interventionsdiagnosticNot Availableterminated
NCT02363946
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)treatment1terminated
NCT00500123
The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) StudyNo drug interventionsNot AvailableNot Availablerecruiting
NCT01669421
Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.treatment2completed
NCT01832220
Genomic Research in Alpha-1 Antitrypsin DeficiencyNo drug interventionsNot AvailableNot Availablecompleted
NCT00884455
Alpha-1 Foundation DNA and Tissue BankNo drug interventionsNot AvailableNot Availableenrolling_by_invitation
NCT02273349
Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin DeficiencyNo drug interventionstreatmentNot Availablewithdrawn
NCT05178277
Czech AATD RegistryNo drug interventionsNot AvailableNot Availablerecruiting
NCT01379469
Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiencytreatment2terminated
NCT01380626
Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patientsNo drug interventionsbasic_scienceNot Availablecompleted
NCT04764448
A Study of Belcesiran in Patients With AATLDother2terminated
NCT05146882
An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)other2withdrawn
NCT06505603
PiMZ Longitudinal Cohort (PiMZ Logic)No drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT06389877
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)No drug interventionstreatment1 / 2recruiting
NCT04167345
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ GenotypeNo drug interventionstreatment2terminated
NCT00242385
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)treatment1completed
NCT03285100
The Effects of Discontinuation of Vitamin K Antagonists on the Rate of Elastin DegradationNo drug interventionsNot AvailableNot Availableunknown_status
NCT03362242
Study of ARO-AAT in Normal Adult Volunteerstreatment1completed
NCT00396006
Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)treatment4completed
NCT03946449
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)treatment2active_not_recruiting
NCT04180319
EARCO REGISTRY. History Of Patients With Alpha-1 AntitrypsinNo drug interventionsNot AvailableNot Availablerecruiting
NCT00499941
Alpha-1 Foundation Research RegistryNo drug interventionsNot AvailableNot Availablecompleted
NCT00460096
Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin DeficiencyNo drug interventionstreatment2 / 3completed
NCT02168686
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) DeficiencyNo drug interventionstreatment1 / 2completed
NCT00301366
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiencytreatment3completed
NCT03114020
Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiencytreatment2completed
NCT06049082
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin DeficiencyNo drug interventionstreatment1recruiting
NCT03945292
Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)treatment2completed
NCT00571272
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)No drug interventionsNot AvailableNot Availablesuspended
NCT00430768
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiencytreatment1completed
NCT03385395
Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiencytreatment2withdrawn
NCT04966221
Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary DiseaseNo drug interventionsNot AvailableNot Availableunknown_status
NCT00263887
Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)treatment2completed
NCT03636347
A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.treatment2completed
NCT00005098
Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin DeficiencyNo drug interventionsNot AvailableNot Availableterminated
NCT00295061
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adultstreatment3completed
NCT02691611
Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin DeficiencyNo drug interventionsNot AvailableNot Availableunknown_status
NCT01676688
Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at HomeNo drug interventionsNot AvailableNot Availablecompleted
NCT00067756
4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?treatment2completed
NCT04204252
Evaluate Efficacy and Safety of "Kamada-AAT for Inhalation" in Patients With AATDtreatment3recruiting
NCT04157049
Alpha-1 Research RegistryNo drug interventionsNot AvailableNot Availablerecruiting
NCT04174118
Study of DCR-A1AT in Healthy Adult Volunteersother1completed
NCT00700934
Management of Patients With Alpha-1 Antitrypsin Deficiency Associated EmphysemaNo drug interventionsNot AvailableNot Availableunknown_status
NCT05897424
Open Label Extension Study of INBRX-101 in Adults With AATD Emphysematreatment2recruiting
NCT00377416
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin DeficiencyNo drug interventionstreatment0completed
NCT03039335
The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin DeficiencyNo drug interventionsNot AvailableNot Availablecompleted
NCT03455686
Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung DiseaseNo drug interventionsbasic_scienceNot Availablerecruiting
NCT03802357
Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency PatientsNo drug interventionstreatmentNot Availableunknown_status
NCT05687474
Baby Detect : Genomic Newborn ScreeningNo drug interventionsNot AvailableNot Availablerecruiting
NCT05579431
A Phase 1, First-in-human Study of VX-634No drug interventionstreatment1completed
NCT05856331
Study of INBRX-101 Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysematreatment2recruiting
NCT00001462
Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient IndividualsNo drug interventionsNot AvailableNot Availablecompleted
NCT05297812
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of DiseaseNo drug interventionsNot AvailableNot Availableactive_not_recruiting
NCT03172455
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)Not AvailableNot Availableno_longer_available