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	NCT04174118 (ClinicalTrials.gov)	October 24, 2019	6/11/2019	A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease	A Phase 1/2 Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered DCR-A1AT in Healthy Adult Volunteers and Patients With Alpha1-Antitrypsin-Deficiency-Associated Liver Disease	Alpha 1-Antitrypsin Deficiency	Drug: DCR-A1AT;Drug: Placebo	Dicerna Pharmaceuticals, Inc.	NULL	Recruiting	18 Years	70 Years	All	60	Phase 1;Phase 2	Sweden

No.

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Public_title

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Condition

Intervention

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Inclusion_
agemin

Inclusion_
agemax

Inclusion_
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NCT04174118
(ClinicalTrials.gov)

October 24, 2019

6/11/2019

A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease

A Phase 1/2 Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered DCR-A1AT in Healthy Adult Volunteers and Patients With Alpha1-Antitrypsin-Deficiency-Associated Liver Disease

Alpha 1-Antitrypsin Deficiency

Drug: DCR-A1AT;Drug: Placebo

Dicerna Pharmaceuticals, Inc.

NULL

Recruiting

18 Years

70 Years

All

Phase 1;Phase 2

Sweden