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 231. α1-アンチトリプシン欠乏症 [臨床試験数:38,薬物数:56(DrugBank:17),標的遺伝子数:43,標的パスウェイ数:49] 

Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
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1NCT03946449November 15, 201911 November 2019Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AATA Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)Alpha 1-Antitrypsin DeficiencyDrug: ARO-AAT InjectionArrowhead PharmaceuticalsRecruiting18 Years75 YearsAll12Phase 2Austria;Germany;United Kingdom
2NCT03945292August 7, 201922 October 2019Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AATA Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]Alpha 1-Antitrypsin DeficiencyDrug: ARO-AAT Injection;Other: PlaceboArrowhead PharmaceuticalsRecruiting18 Years75 YearsAll120Phase 2/Phase 3United States
3NCT03815396July 19, 20199 September 2019Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin DeficiencyAn Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD)Alpha-1 Antitrypsin Deficiency;AATDDrug: INBRX-101/rhAAT-FcInhibrx, Inc.Recruiting18 Years70 YearsAll30Phase 1United States
4NCT03679598April 8, 201924 June 2019Alvelestat (MPH996) for the Treatment of ALpha-1 ANTitrypsin DeficiencyA First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan DiseaseAlpha-1 Antitrypsin Deficiency (AATD);Pi*ZZ, Pi*SZ, or Pi*Null Phenotype;Emphysema or COPDDrug: Alvelestat (MPH996);Other: PlaceboUniversity of Alabama at BirminghamNational Institutes of Health (NIH);Mereo BioPharmaRecruiting25 Years80 YearsAll66Phase 2United States
5NCT03767829December 5, 201811 November 2019A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseA Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseDrug: ALN-AAT02;Drug: PlaceboAlnylam PharmaceuticalsNot recruiting18 Years65 YearsAll96Phase 1/Phase 2United Kingdom
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6NCT03636347October 29, 201828 October 2019A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 (PiZZ or Null) Antitrypsin DeficiencyAlpha 1-Antitrypsin Deficiency;Emphysema;COPDDrug: Placebo Oral Tablet;Drug: Alvelestat oral tablet - dose 1;Drug: Alvelestat oral tablet - dose 2Mereo BioPharmaSyneos HealthRecruiting18 Years75 YearsAll165Phase 2United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom
7NCT03385395July 201811 June 2018Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin DeficiencyA Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intravenous Administration of OctaAlpha1 (Octapharma) With a Marketed Preparation Glassia® (Kamada Ltd.) in Subjects With Alpha-1-antitrypsin DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: OctaAlpha1;Drug: GlassiaOctapharmaNot recruiting18 YearsN/AAll0Phase 2
8EUCTR2015-004110-23-PL19/04/201830 April 2019A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin DeficiencyAn Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLEPulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 20.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Prolastin-C
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human)
CAS Number: 9041-92-3
Current Sponsor code: TAL6004
Other descriptive name: ALPHA-1-ANTITRYPSIN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Grifols Therapeutics LLCAuthorised Female: yes
Male: yes
250Phase 3United States;Estonia;Canada;Poland;Denmark;Australia;New Zealand;Sweden
9NCT03362242March 12, 201813 May 2019Study of ARO-AAT in Normal Adult VolunteersA Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult VolunteersAlpha 1-Antitrypsin DeficiencyDrug: ARO-AAT Injection;Other: Sterile Normal Saline (0.9% NaCl)Arrowhead PharmaceuticalsNot recruiting18 Years55 YearsAll45Phase 1New Zealand
10NCT03172455July 25, 201726 November 2018Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)Acute-graft-versus-host Disease;Steroid Refractory Acute Graft Versus Host Disease;Graft-versus-host-disease;Graft Vs Host Disease;Alpha 1-Antitrypsin Deficiency;Alpha-1 Proteinase Inhibitor;Alpha-1 Protease Inhibitor Deficiency;Acute Graft-Versus-Host Reaction Following Bone Marrow TransplantDrug: GlassiaImpatients N.V. trading as myTomorrowsKamada, Ltd.Not recruitingN/AN/AAllPhase 2/Phase 3
No.TrialIDDate_
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11NCT03114020March 22, 20174 November 2019Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin DeficiencyPhase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of EmphysemaEmphysema;Alpha 1-Antitrypsin DeficiencyDrug: Hyaluronic Acid Inhalation Solution;Drug: Placebo Inhalation SolutionGerard TurinoRecruiting18 Years80 YearsAll40Phase 2United States;Canada
12NCT03008915January 201723 April 2019Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin DeficiencyTargeting Pulmonary Perfusion in Alpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency;Emphysema;Chronic Obstructive Pulmonary DiseaseDrug: Placebo;Drug: AspirinColumbia UniversityAlpha-1 Foundation;Stony Wold-Herbert Fund, Inc.Not recruiting40 YearsN/AAll15Phase 2United States
13NCT02796937July 20169 September 2019Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin DeficiencyAn Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin DeficiencyPulmonary Emphysema in Alpha-1 Antitrypsin DeficiencyBiological: Alpha-1 MPGrifols Therapeutics LLCRecruiting20 Years72 YearsAll250Phase 3United States;Australia;Canada;Denmark;Estonia;New Zealand;Poland;Sweden
14EUCTR2016-000917-59-IE29/06/201616 January 2017A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD)An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as evidenced by changes in liver biopsy in Patients with Alpha-1 Antitrypsin Deficiency (AATD)Alpha-1 Antitrypsin Deficiency related liver disease
MedDRA version: 19.1 Level: PT Classification code 10001806 Term: Alpha-1 anti-trypsin deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Product Name: ARC-AAT Injection
Product Code: ARC-AAT
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: API-AAT
Current Sponsor code: API-AAT
Other descriptive name: API-AAT, AD00370, ARC-AAT, API drug Substance
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 52-
Arrowhead Pharmaceuticals, IncNot RecruitingFemale: yes
Male: yes
12Phase 2Canada;Ireland;Sweden
15EUCTR2015-004110-23-DK29/03/201617 October 2016A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin DeficiencyAn Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alphal-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLEPulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 19.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Prolastin-C
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human)
CAS Number: 9041-92-3
Current Sponsor code: GTI1201-OLE
Other descriptive name: ALPHA-1-ANTITRYPSIN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Grifols Therapeutics Inc.AuthorisedFemale: yes
Male: yes
250Phase 3United States;Estonia;Spain;Russian Federation;France;Canada;Argentina;Brazil;Poland;Romania;Denmark;Australia;Germany;New Zealand;Sweden
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16EUCTR2015-001297-18-GB29/06/201528 February 2019The First-in-Human Study of an Investigational Drug, ALN-AAT, in Healthy Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver DiseaseA Phase 1/2, Randomized, Single-Blind, Placebo-Controlled, Single-Ascending, and Multiple-Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver DiseaseZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
MedDRA version: 19.1 Level: PT Classification code 10001806 Term: Alpha-1 anti-trypsin deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ALN-AAT
Product Code: ALN-AAT
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ALN-61444
Current Sponsor code: ALN-61444
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
Alnylam Pharmaceuticals, Inc.Not Recruiting Female: yes
Male: yes
66Phase 1;Phase 2United States;United Kingdom
17NCT02363946February 201516 December 2017A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect of Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients With Alpha-1 Antitrypsin Deficiency (AATD)Alpha-1 Antitrypsin DeficiencyDrug: ARC-AAT Injection;Other: PlaceboArrowhead PharmaceuticalsNot recruiting18 Years70 YearsAll65Phase 1Australia;Germany;Netherlands;United Kingdom
18NCT02001688April 201427 June 2016Phase II, Safety and ELF Study of Kamada-API for InhalationPhase II, Double-blind, Placebo-controlled Study to Explore the ELF and Plasma Concentration as Well as Safety of Inhaled Alpha-1 Antitrypsin in Alpha-1 Antitrypsin Deficiency SubjectsAlpha-1 Antitrypsin DeficiencyBiological: Kamada-API for Inhalation, 80mg;Drug: Placebo;Biological: Kamada-API for Inhalation, 160mgKamada, Ltd.Not recruiting18 Years65 YearsBoth24Phase 2United States
19EUCTR2013-001870-38-SE09/01/201427 January 2014A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin DeficiencyA Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTAPulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 16.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Prolastin-C
Product Name: Alpha1-Proteinase Inhibitor (Human)
Product Code: Prolastin-C
Pharmaceutical Form: Powder for infusion
INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human)
CAS Number: 9041-92-3
Current Sponsor code: TAL6004
Other descriptive name: ALPHA-1-ANTITRYPSIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: not less then
Concentration number: 47-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use
Grifols Therapeutics Inc.AuthorisedFemale: yes
Male: yes
339United States;Estonia;Slovakia;Finland;Spain;Ireland;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;New Zealand;Sweden
20NCT01983241November 20139 September 2019Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin DeficiencyPulmonary Emphysema in Alpha-1 PI DeficiencyBiological: Alpha-1 MP;Other: 0.9% Sodium Chloride for Injection, USPGrifols Therapeutics LLCRecruiting18 Years70 YearsAll339Phase 3United States;Argentina;Australia;Brazil;Canada;Denmark;Estonia;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden
No.TrialIDDate_
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21NCT01810458October 201313 May 2019Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin DeficiencyLiver Fibrosis;Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATDDevice: Abdominal ultrasound;Procedure: History and physical;Procedure: Intravenous catheter;Procedure: Blood draw;Other: Liver questionnaire;Procedure: Liver Biopsy;Drug: Midazolam;Drug: Fentanyl;Drug: Lidocaine;Drug: Acetaminophen;Drug: Lorazepam;Drug: Oxycodone/Acetaminophen;Drug: OndansetronUniversity of FloridaNot recruiting18 Years70 YearsAll109Phase 3United States
22NCT01669421July 201213 June 2016Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.Effect of a Higher Dose of Alpha-1 Antitrypsin Augmentation Therapy on Lung Inflammation in Subjects With Alpha-1 Antitrypsin Deficiency.Alpha 1 Antitrypsin DeficiencyDrug: Alpha-1 Antitrypsin (human)Michael Campos, MDCSL BehringNot recruiting18 Years75 YearsBoth10Phase 2United States
23NCT01379469January 201215 July 2019Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin DeficiencyA Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin DeficiencyAlpha-1-antitrypsin Deficiency;Liver CirrhosisDrug: Drug-Carbamazepine (Tegretol XR);Drug: Carbamazepine (Tegretol XR) PlaceboWashington University School of MedicineNovartis;National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);University of PittsburghRecruiting14 Years80 YearsAll30Phase 2United States
24NCT01280994January 201123 April 2019Hyperpolarized 129Xe MRI for Imaging Pulmonary FunctionHyperpolarized 129Xe MR Imaging of the Lung Function in Healthy Volunteers and Subjects With Pulmonary DiseaseInterstitial Lung Disease;Cystic Fibrosis;Pulmonary Hypertension;NSIP;Alpha 1-Antitrypsin DeficiencyDrug: XenonBastiaan DriehuysRecruiting18 Years80 YearsAll445Phase 1United States
25NCT01213043November 201019 October 2017Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin DeficiencyA Randomized Double-blind Crossover Study to Assess the Safety and Pharmacokinetics of Two Different Doses of Weekly Intravenous Administration of Alpha1-Proteinase Inhibitor (Human) Prolastin®-C in Subjects With Alpha1-Antitrypsin DeficiencyEmphysema;Alpha 1-antitrypsin Deficiency (AATD)Biological: Prolastin-C, 60 mg/kg;Biological: Prolastin-C, 120 mg/kgGrifols Therapeutics Inc.Not recruiting18 Years70 YearsAll30Phase 2United States
No.TrialIDDate_
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26NCT01054339June 20101 April 2019Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin DeficiencyA Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha-1 Antitrypsin (rAAV1-CB-hAAT) in Patients With Alpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin DeficiencyDrug: rAAV1-CB-hAATApplied Genetic Technologies CorpNational Heart, Lung, and Blood Institute (NHLBI)Not recruiting18 Years75 YearsAll9Phase 2United States;Ireland
27EUCTR2008-005326-36-GB02/11/200914 July 2014A clinical trial looking at the safety and effectiveness of a new drug for inhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients with Emphysema.A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with EmphysemaAlpha-1 Antitrypsin deficiency in patients with emphysema.
MedDRA version: 17.0 Level: PT Classification code 10001806 Term: Alpha-1 anti-trypsin deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Aerosolized human Alpha-1 Antitrypsin
Pharmaceutical Form: Nebuliser solution
CAS Number: 8000047054
Other descriptive name: ALPHA-1-ANTITRYPSIN
Concentration unit: g/l gram(s)/litre
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Nebuliser solution
Route of administration of the placebo: Inhalation use
Kamada LimitedAuthorisedFemale: yes
Male: yes
200Phase 2/3Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden
28EUCTR2007-004869-18-GB03/07/200810 July 2012Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATDEvaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATDThe medical condition is emphysema and will be investigated in subjects with alpha 1-antitrypsin deficiency.
MedDRA version: 9.1 Level: LLT Classification code 10001811 Term: Alpha-1 proteinase inhibitor deficiency
MedDRA version: 9.1 Level: LLT Classification code 10014563 Term: Emphysema pulmonary
MedDRA version: 9.1 Level: LLT Classification code 10010952 Term: COPD
MedDRA version: 9.1 Level: LLT Classification code 10006458 Term: Bronchitis chronic
Trade Name: Prolastin
Product Name: Prolastin
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: alpha 1-proteinase inhibitor (human)
CAS Number: 8000047054
Other descriptive name: ALPHA-1-ANTITRYPSIN
Concentration unit: mg/l milligram(s)/litre
Concentration type: equal
UHB NHS Foundation TrustAuthorisedFemale: yes
Male: yes
United Kingdom
29NCT01851642September 200726 August 2019Lung Disease and Its Affect on the Work of White Blood Cells in the LungsThe Role of Conformational Diseases on Macrophage FunctionAlpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD;Cystic Fibrosis (CF)Procedure: History and physical exam.;Procedure: Blood draw.;Procedure: Pulmonary function testing.;Drug: Albuterol inhaler.University of FloridaRecruiting18 YearsN/AAll220Phase 1/Phase 2United States
30NCT00460096March 200719 February 2015Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin DeficiencyRandomized Double-Blind Comparison of an Alpha-1 Proteinase Inhibitor (Kamada API) With a Currently Marketed API Product in Individuals With Alpha-1 Antitrypsin DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: Kamada-APIKamada, Ltd.Not recruiting18 YearsN/ABoth50Phase 2/Phase 3United States
No.TrialIDDate_
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31NCT00396006October 200616 December 2017Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)The Effect of Augmentation Therapy With ARALAST Fraction IV-1 (ARALAST) Alpha1-Proteinase Inhibitor (a1-PI) on the Level of a1-PI and Other Analytes in the Bronchoalveolar (BAL) Epithelial Lining Fluid (ELF)Alpha 1-Antitrypsin DeficiencyBiological: Alpha1-Proteinase InhibitorBaxalta now part of ShireNot recruiting18 YearsN/AAll21Phase 4Australia;New Zealand
32NCT00301366June 200619 October 2017The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) DeficiencyMulti-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) DeficiencyAlpha 1-Antitrypsin DeficiencyDrug: alpha-1 proteinase inhibitor (human)Grifols Therapeutics Inc.Not recruiting18 YearsN/AAll38Phase 3United States;United Kingdom;Netherlands;Poland
33NCT00295061May 200619 October 2017Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient AdultsMulti-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency.Alpha 1-Antitrypsin DeficiencyDrug: Alpha-1 MP;Drug: alpha-1 proteinase inhibitor (human)Grifols Therapeutics Inc.Not recruiting18 YearsN/AAll24Phase 3United States
34NCT00430768February 200619 October 2017Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) DeficiencyPreclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient AdultsAlpha 1-Antitrypsin DeficiencyBiological: rAAV1-CB-hAATUniversity of Massachusetts, WorcesterNational Heart, Lung, and Blood Institute (NHLBI);Applied Genetic Technologies Corp;Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR)Not recruiting18 YearsN/AAll9Phase 1United States
35NCT00242385December 200516 December 2017Pharmacokinetic Study of ARALAST (Human Alpha1- PI)Single-Dose, Double-Blind, Crossover Study to Evaluate the Pharmacokinetic Comparability of ARALAST Fraction IV-1 Alpha1-Proteinase Inhibitor (ARALAST Fr. IV-1) and ARALASTAlpha 1-Antitrypsin DeficiencyBiological: Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor;Biological: Dose of 60 mg/kg alpha1-proteinase inhibitorBaxalta now part of ShireBaxter Healthcare CorporationNot recruiting18 YearsN/AAll25Phase 1Australia;New Zealand
No.TrialIDDate_
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36EUCTR2004-001688-23-ES27/10/200419 March 2012A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiencyA multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiencysymptomatic emphysema secondary to alpha-1-antitrypsin deficiencyProduct Name: Not applicable
Product Code: RO3300074
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: None assigned
Other descriptive name: Retinoic Acid Receptor gamma Agonist
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.5-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use
F. Hoffmann-La Roche LtdAuthorisedFemale: yes
Male: yes
300United Kingdom;Denmark;Spain
37NCT00263887December 200319 October 2017Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)Multi-center, Randomized Trial With I.V. Prolastin® to Evaluate Frequency of Exacerbations and Progression of Emphysema by Means of Multi-slice CT Scans in Patients With Congenital Alpha-1-antitrypsin Deficiency.Alpha 1-Antitrypsin DeficiencyDrug: Alpha1-Proteinase Inhibitor (Human);Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)Grifols Therapeutics Inc.Not recruiting18 YearsN/AAll77Phase 2Denmark;Sweden;United Kingdom
38NCT00067756November 200119 February 20154-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient IndividualsAlpha 1-Antitrypsin DeficiencyDrug: 4-PBAUniversity of FloridaAlpha-1 Foundation;Brantly, Mark L., M.D.Not recruiting18 Years65 YearsBoth12Phase 2United States

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