DDrare: Database of Drug Development for Rare Diseases

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	1

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.

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

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