DDrare: Database of Drug Development for Rare Diseases

( DrugBank: - / KEGG DRUG: - )

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
231	α1−アンチトリプシン欠乏症	93

231. α1−アンチトリプシン欠乏症

臨床試験数 : 93 / 薬物数 : 77 - (DrugBank : 8) / 標的遺伝子数 : 10 - 標的パスウェイ数 : 36

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Showing 1 to 10 of 93 trials

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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	NCT05643495 (ClinicalTrials.gov)	February 23, 2023	29/11/2022	A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype	A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitr ...	Alpha-1 Antitrypsin Deficiency	Drug: VX-864	Vertex Pharmaceuticals Incorporated	NULL	Recruiting	18 Years	80 Years	All	20	Phase 2	United States
2	NCT05727800 (ClinicalTrials.gov)	February 8, 2023	3/2/2023	A Phase 1, First-in-human Study of VX-668	A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-668	Alpha-1 Antitrypsin Deficiency	Drug: VX-668;Drug: Placebo	Vertex Pharmaceuticals Incorporated	NULL	Recruiting	18 Years	55 Years	All	114	Phase 1	United States
3	NCT05579431 (ClinicalTrials.gov)	October 13, 2022	10/10/2022	A Phase 1, First-in-human Study of VX-634	A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-634	Alpha 1-Antitrypsin Deficiency	Drug: VX-634;Drug: Placebo	Vertex Pharmaceuticals Incorporated	NULL	Recruiting	18 Years	55 Years	All	114	Phase 1	United States
4	NCT05315921 (ClinicalTrials.gov)	March 22, 2022	30/3/2022	Study of OsrhAAT or Placebo in Healthy Volunteers	A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, ...	Emphysema Secondary to Congenital AATD	Drug: OsrhAAT 1 mg/kg IV;Drug: OsrhAAT 3 mg/kg IV;Drug: OsrhAAT 10 mg/kg IV;Drug: OsrhAAT 20 mg/kg I ...	Healthgen Biotechnology Corp.	NULL	Recruiting	18 Years	55 Years	All	48	Phase 1	United States
5	EUCTR2019-000602-30-IE (EUCTR)	25/05/2021	31/03/2020	A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficac ...	A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficac ...	Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limit ...	Product Name: Kamada -AAT for Inhalation or inhaled AAT (AAT) INN or Proposed INN: Alpha-1 Antitrypsi ...	Kamada Ltd.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	220	Phase 3	Finland;Belgium;Ireland;Netherlands;United Kingdom;Sweden
6	NCT04764448 (ClinicalTrials.gov)	February 12, 2021	27/1/2021	A Study of Belcesiran in Patients With AATLD	A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Ph ...	Alpha 1-Antitrypsin Deficiency	Drug: Belcesiran;Other: Placebo	Dicerna Pharmaceuticals, Inc.	NULL	Recruiting	18 Years	75 Years	All	46	Phase 2	Australia;Austria;Belgium;Canada;France;Germany;Ireland;Netherlands;New Zealand;Portugal;Spain;Swede ...
7	EUCTR2018-003385-14-AT (EUCTR)	28/01/2021	14/09/2020	A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of ...	A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect o ...	alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Class ...	Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001	Arrowhead Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	120	Phase 2;Phase 3	Portugal;United States;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Switzerland;I ...
8	EUCTR2018-003385-14-DE (EUCTR)	05/01/2021	25/05/2020	A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of ...	A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacody ...	alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.1;Level: LLT;Class ...	Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001	Arrowhead Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	36	Phase 2	Portugal;United States;Canada;Spain;Ireland;Austria;Netherlands;Germany;United Kingdom;Italy;Sweden
9	EUCTR2019-004881-16-IE (EUCTR)	29/10/2020	09/07/2020	Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype	A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 i ...	Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.1;Level: PT;Cla ...	Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Produc ...	Vertex Pharmaceuticals Incorporated	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 2	United States;Canada;Ireland;Germany;United Kingdom;Sweden
10	EUCTR2018-003385-14-GB (EUCTR)	19/10/2020	28/06/2019	A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of ...	A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect o ...	alpha-1 antitrypsin deficiency (AATD)-associated liver disease MedDRA version: 23.0;Level: LLT;Class ...	Product Name: ARO-AAT Injection Product Code: ARO-AAT INN or Proposed INN: ADS-001	Arrowhead Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	120	Phase 2;Phase 3	United States;Portugal;Canada;Spain;Poland;Ireland;Netherlands;Italy;United Kingdom;Sweden

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