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	NCT02900183 (ClinicalTrials.gov)	October 2016	9/9/2016	Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels	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	Drug: ARC-AAT Injection	Arrowhead Pharmaceuticals	NULL	Withdrawn	18 Years	75 Years	All	0	Phase 2	Canada;Ireland;Italy;Sweden
2	EUCTR2016-000917-59-SE (EUCTR)	04/08/2016	08/06/2016	A 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: 19.0;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]	Product Name: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API -AAT Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance Product Name: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API -AAT Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance Product Name: ARC-AAT Injection Product Code: ARC-AAT INN or Proposed INN: API -AAT Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance	Arrowhead Pharmaceuticals Inc	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	Canada;Ireland;Sweden
3	EUCTR2016-000917-59-IE (EUCTR)	29/06/2016	13/04/2016	A 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 INN or Proposed INN: API -AAT Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance	Arrowhead Pharmaceuticals, Inc	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	Canada;Ireland;Sweden
4	NCT02363946 (ClinicalTrials.gov)	February 2015	2/2/2015	A 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 Deficiency	Drug: ARC-AAT Injection;Other: Placebo	Arrowhead Pharmaceuticals	NULL	Terminated	18 Years	70 Years	All	65	Phase 1	Australia;Germany;Netherlands;United Kingdom

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

NCT02900183
(ClinicalTrials.gov)

October 2016

9/9/2016

Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels

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

Drug: ARC-AAT Injection

Arrowhead Pharmaceuticals

NULL

Withdrawn

18 Years

75 Years

All

Phase 2

Canada;Ireland;Italy;Sweden

EUCTR2016-000917-59-SE
(EUCTR)

04/08/2016

08/06/2016

A 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: 19.0;Level: PT;Classification code 10001806;Term: Alpha-1 anti-trypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Product Name: ARC-AAT Injection
Product Code: ARC-AAT
INN or Proposed INN: API -AAT
Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance
Product Name: ARC-AAT Injection
Product Code: ARC-AAT
INN or Proposed INN: API -AAT
Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance
Product Name: ARC-AAT Injection
Product Code: ARC-AAT
INN or Proposed INN: API -AAT
Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance

Arrowhead Pharmaceuticals Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Canada;Ireland;Sweden

EUCTR2016-000917-59-IE
(EUCTR)

29/06/2016

13/04/2016

A 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)

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
INN or Proposed INN: API -AAT
Other descriptive name: API -AAT , AD00370, ARC-AAT, API drug Substance

Arrowhead Pharmaceuticals, Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Canada;Ireland;Sweden

NCT02363946
(ClinicalTrials.gov)

February 2015

2/2/2015

A 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 Deficiency

Drug: ARC-AAT Injection;Other: Placebo

Arrowhead Pharmaceuticals

NULL

Terminated

18 Years

70 Years

All

Phase 1

Australia;Germany;Netherlands;United Kingdom