ALN-PCSSC ( DrugBank: ALN-PCSsc )

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1 disease

ID	Disease name (Link within this page)	Number of trials
79	Homozygous familial hypercholesterolemia	2

79. Homozygous familial hypercholesterolemia

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Clinical trials : 145 /  Drugs : 114 - (DrugBank : 26) / Drug target genes : 8 - Drug target pathways : 17

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	EUCTR2016-003376-49-NL (EUCTR)	20/06/2017	18/01/2017	A study to evaluate the effect of ALN-PCSSC treatment in patients with Homozygous Familial Hypercholesterolemia	An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia - ORION-2	Homozygous Familial Hypercholesterolemia MedDRA version: 19.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]	Product Name: ALN-PCSSC Product Code: ALN-PCSSC INN or Proposed INN: ALN-60212 Other descriptive name: ALN-60212	The Medicines Company	NULL	Not Recruiting			Female: yes Male: yes	10	Phase 2	United States;South Africa;Netherlands
2	NCT02963311 (ClinicalTrials.gov)	December 13, 2016	10/11/2016	A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)	An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)	Homozygous Familial Hypercholesterolemia	Drug: ALN-PCSSC;Drug: Standard of Care	The Medicines Company	NULL	Completed	12 Years	N/A	All	9	Phase 2	United States;Netherlands;South Africa