Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia

PHASE3CompletedINTERVENTIONAL
Enrollment

51

Participants

Timeline

Start Date

July 31, 2007

Primary Completion Date

March 31, 2009

Study Completion Date

March 31, 2009

Conditions
Lipid Metabolism, Inborn ErrorsHypercholesterolemia, Autosomal DominantHyperlipidemiasMetabolic DiseasesHyperlipoproteinemia Type IIMetabolism, Inborn ErrorsGenetic Diseases, InbornInfant, Newborn, DiseasesMetabolic DisorderCongenital AbnormalitiesHypercholesterolemiaHyperlipoproteinemiasDyslipidemiasLipid Metabolism Disorders
Interventions
DRUG

mipomersen

200 mg mipomersen administered once a week for 26 weeks as a 1 mL subcutaneous injection. Subjects weighing less than 50 kg received a lower dose of 160 mg (0.8mL) mipomersen.

DRUG

Placebo

1 mL subcutaneous injection once a week for 26 weeks. Subjects weighing less than 50 kg received 0.8 mL subcutaneous injection.

Trial Locations (10)

2193

Parktown

7925

Observatory

11217

Taipei

28204

Charlotte

45212

Cincinnati

168752

Mistri Wing

05403-000

São Paulo

G7H 5H6

Chicoutimi

G1V 4G2

Ste Foy

WC1N 3BG

London

Sponsors
All Listed Sponsors
collaborator

Ionis Pharmaceuticals, Inc.

INDUSTRY

lead

Kastle Therapeutics, LLC

INDUSTRY