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

NCT00607373 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 51

Last updated 2016-09-09

Study results available
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Summary

The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.

This study consisted of a 26-week treatment period and a 24-week post-treatment follow-up period. Following treatment and Week 28 evaluations, participants could elect to enroll in an open-label extension study (301012-CS6; NCT00694109). Participants who were not eligible or elected not to enroll in the open-label extension study or who discontinued during the 28-week treatment period were followed in this study for 24 weeks from administration of the last dose of study drug.

Conditions

  • Lipid Metabolism, Inborn Errors
  • Hypercholesterolemia, Autosomal Dominant
  • Hyperlipidemias
  • Metabolic Diseases
  • Hyperlipoproteinemia Type II
  • Metabolism, Inborn Errors
  • Genetic Diseases, Inborn
  • Infant, Newborn, Diseases
  • Metabolic Disorder
  • Congenital Abnormalities
  • Hypercholesterolemia
  • Hyperlipoproteinemias
  • Dyslipidemias
  • Lipid 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.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • Kastle Therapeutics, LLC

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-07-31
Primary Completion
2009-03-31
Completion
2009-03-31

Countries

  • United States
  • Brazil
  • Canada
  • Singapore
  • South Africa
  • Taiwan
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00607373 on ClinicalTrials.gov