DDrare: Database of Drug Development for Rare Diseases

231. α1-アンチトリプシン欠乏症
［臨床試験数：83，薬物数：89（DrugBank：16），標的遺伝子数：35，標的パスウェイ数：46］

Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = (4S)-4-[4-cyano-2-(methylsulfonyl)phenyl]-3,6-dimethyl-2-oxo-1-[3-(trifluoromethyl)phenyl]-1,2,3,4-tetrahydropyrimidine-5-carbonitrile; 4-PBA; ADS-001; ALN-61444; ALN-77412; ALN-AAT; ALN-AAT02; ALPHA-1-ANTITRYPSIN; ALVELESTAT; API; API-AAT; API-AAT, AD00370, ARC-AAT, API drug Substance; ARC-AAT; ARC-AAT Injection; ARO-AAT; ARO-AAT Injection; Acetaminophen; Aerosolized human Alpha-1 Antitrypsin; Albumin (Human) 20%, United States Pharmacopeia (USP); Albuterol; Albuterol inhaler.; Alpha 1-Antitrypsin; Alpha 1-Proteinase Inhibitor; Alpha 1-antitrypsin; Alpha 1-proteinase inhibitor (human); Alpha-1 Antitrypsin (AAT); Alpha-1 Antitrypsin (human); Alpha-1 MP; Alpha-1 proteinase inhibitor (human); Alpha-1-antitrypsin; Alpha1-Proteinase Inhibitor; Alpha1-Proteinase Inhibitor (Human); Alpha1-proteinase Inhibitor; Alvelestat; Alvelestat (MPH966); Alvelestat oral tablet - dose 1; Alvelestat oral tablet - dose 2; Alvelestat tosylate; Aspirin; Carbamazepine; DCR-A1AT; Device: Abdominal ultrasound; Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor; Dose of 60 mg/kg alpha1-proteinase inhibitor; Drug-Carbamazepine (Tegretol XR); Fentanyl; Glassia; Hyaluronic Acid Inhalation Solution; Hyaluronic acid; INBRX-101/rhAAT-Fc; Kamada -AAT for Inhalation or inhaled AAT (AAT); Kamada-AAT for Inhalation, 160mg; Kamada-AAT for Inhalation, 80mg; Kamada-AAT for inhalation; Kamada-API; Lidocaine; Lorazepam; MPH966; Midazolam; None; None assigned; OctaAlpha1; Ondansetron; Other: Liver questionnaire; Other: Sterile Normal Saline (0.9% NaCl); Other: Withdrawal from alpha1 antitrypsin replacement therapy; Oxycodone; Oxycodone/Acetaminophen; PBA; PHP-303; Procedure: Blood draw; Procedure: Blood draw.; Procedure: History and physical; Procedure: History and physical exam.; Procedure: Intravenous catheter; Procedure: Liver Biopsy; Procedure: Pulmonary function testing.; Prolastin; Prolastin-C; Prolastin-C, 120 mg/kg; Prolastin-C, 60 mg/kg; RAAV1-CB-hAAT; RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING THE HUMAN ALPHA-1-ANTITRYPSIN GENE; RO3300074; Retinoic Acid Receptor gamma Agonist; Retinoic acid; VX-814; VX-864; VX864

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2009-014286-20-IE (EUCTR)	21/12/2010	31/05/2010	A 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 Deficiency - rAAV1-CB-hAAT in Patients with Alpha 1 Antitrypsin Deficiency	A 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 Deficiency - rAAV1-CB-hAAT in Patients with Alpha 1 Antitrypsin Deficiency	Alpha 1 antitrypsin deficiency.This is an inherited, genetic condition characterized by reduced serum levels of alpha-1 antitrypsin (AAT) which increases the risk of developing emphysema and liver disease MedDRA version: 12.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency	Product Name: rAAV1-CB-hAAT Product Code: rAAV1-CB-hAAT	Applied Genetic Technologies Corporation	NULL	Not Recruiting			Female: yes Male: yes	9	Phase 2	Ireland
2	NCT01054339 (ClinicalTrials.gov)	June 2010	21/1/2010	Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency	A 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 Deficiency	Alpha-1 Antitrypsin Deficiency	Drug: rAAV1-CB-hAAT	Applied Genetic Technologies Corp	National Heart, Lung, and Blood Institute (NHLBI)	Completed	18 Years	75 Years	All	9	Phase 2	United States;Ireland
3	NCT00430768 (ClinicalTrials.gov)	February 2006	31/1/2007	Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency	Preclinical & 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 Adults	Alpha 1-Antitrypsin Deficiency	Biological: rAAV1-CB-hAAT	University of Massachusetts, Worcester	National Heart, Lung, and Blood Institute (NHLBI);Applied Genetic Technologies Corp;Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR)	Completed	18 Years	N/A	All	9	Phase 1	United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2009-014286-20-IE
(EUCTR)

21/12/2010

31/05/2010

A 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 Deficiency - rAAV1-CB-hAAT in Patients with Alpha 1 Antitrypsin Deficiency

Alpha 1 antitrypsin deficiency.This is an inherited, genetic condition characterized by reduced serum levels of alpha-1 antitrypsin (AAT) which increases the risk of developing emphysema and liver disease
MedDRA version: 12.1;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency

Product Name: rAAV1-CB-hAAT
Product Code: rAAV1-CB-hAAT

Applied Genetic Technologies Corporation

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Ireland

NCT01054339
(ClinicalTrials.gov)

June 2010

21/1/2010

Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

A 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 Deficiency

Alpha-1 Antitrypsin Deficiency

Drug: rAAV1-CB-hAAT

Applied Genetic Technologies Corp

National Heart, Lung, and Blood Institute (NHLBI)

Completed

18 Years

75 Years

All

Phase 2

United States;Ireland

NCT00430768
(ClinicalTrials.gov)

February 2006

31/1/2007

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

Preclinical & 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 Adults

Alpha 1-Antitrypsin Deficiency

Biological: rAAV1-CB-hAAT

University of Massachusetts, Worcester

National Heart, Lung, and Blood Institute (NHLBI);Applied Genetic Technologies Corp;Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR)

Completed

18 Years

N/A

All

Phase 1

United States