DDrare: Database of Drug Development for Rare Diseases

Akcea-angptl3-lrx (DrugBank: -)

2 diseases

ID	Disease name (Link within this page)	Number of trials
79	Homozygous familial hypercholesterolemia	1
265	Lipodystrophy	1

79. Homozygous familial hypercholesterolemia [ 128 clinical trials, 105 drugs, (DrugBank: 29 drugs), 8 drug target genes, 15 drug target pathways]

Searched query = "Homozygous familial hypercholesterolemia"
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.

1 / 128 trial 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	NCT03455777 (ClinicalTrials.gov)	April 12, 2018	22/2/2018	Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)	A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)	Homozygous Familial Hypercholesterolemia	Drug: AKCEA-ANGPTL3-LRX	Akcea Therapeutics	Ionis Pharmaceuticals, Inc.	Withdrawn	18 Years	N/A	All	0	Phase 2	Canada

265. Lipodystrophy [ 109 clinical trials, 164 drugs, (DrugBank: 59 drugs), 26 drug target genes, 94 drug target pathways]

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons syndrome"
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.

1 / 109 trial 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	NCT03514420 (ClinicalTrials.gov)	May 30, 2018	21/4/2018	Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)	An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy	Familial Partial Lipodystrophy	Drug: AKCEA-ANGPTL3-LRX	Akcea Therapeutics	Ionis Pharmaceuticals, Inc.	Completed	18 Years	N/A	All	4	Phase 2	United States