231. Alpha-1-antitrypsin deficiency Clinical trials / Disease details

Clinical trials : 89 / Drugs : 89 - (DrugBank : 16) / Drug target genes : 35 - Drug target pathways : 47

3 trials found

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	NCT05146882 (ClinicalTrials.gov)	December 2021	9/11/2021	An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)	A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease	Alpha 1-Antitrypsin Deficiency	Drug: Belcesiran	Dicerna Pharmaceuticals, Inc.	NULL	Enrolling by invitation	18 Years	75 Years	All	54	Phase 2	New Zealand
2	NCT04764448 (ClinicalTrials.gov)	February 12, 2021	27/1/2021	A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease	A Phase 2a, Randomized, Double-blind, Placebo Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency	Alpha 1-Antitrypsin Deficiency	Drug: belcesiran;Other: Placebo	Dicerna Pharmaceuticals, Inc.	NULL	Recruiting	18 Years	70 Years	All	54	Phase 2	Australia;New Zealand;Spain;United Kingdom
3	NCT04174118 (ClinicalTrials.gov)	October 24, 2019	6/11/2019	Study of DCR-A1AT in Healthy Adult Volunteers	A Phase 1 Single Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered Belcesiran in Healthy Adult Volunteers	Alpha 1-Antitrypsin Deficiency	Drug: belcesiran;Drug: Placebo	Dicerna Pharmaceuticals, Inc.	NULL	Recruiting	18 Years	55 Years	All	36	Phase 1	New Zealand;Sweden