Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

44 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-02-28

Study Completion Date

2007-12-31

Brief Summary

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The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 (mipomersen) as add-on therapy in subjects with Heterozygous Familial Hypercholesterolemia

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Detailed Description

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Conditions

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Hypercholesterolemia, Familial

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Cohort A

Loading doses followed by weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012 or Placebo

Intervention Type DRUG

50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

Cohort B

Loading doses followed by weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012 or Placebo

Intervention Type DRUG

100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

Cohort C

Loading doses followed by weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012 or Placebo

Intervention Type DRUG

200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

Cohort D

Loading doses followed by extended weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012 or Placebo

Intervention Type DRUG

300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78, and 85

Interventions

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ISIS 301012 or Placebo

50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

Intervention Type DRUG

ISIS 301012 or Placebo

100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

Intervention Type DRUG

ISIS 301012 or Placebo

200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

Intervention Type DRUG

ISIS 301012 or Placebo

300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78, and 85

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Weight ≥ 50 kg
* Diagnosis of Heterozygous Familial Hypercholesterolemia.
* Females must be non-pregnant and non-lactating.
* On stable lipid lowering therapy for at least 4 weeks.
* Lipid levels meet the pre-specified criteria.

Exclusion Criteria

* Subject had heart problems in the prior 6 months.
* Subject has elevated ALT, AST, or CPK.
* History of renal disease, liver disease, or malignancy.
* Use of oral anticoagulants, unless the dose has been stable for 4 weeks
* Have any other conditions, which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No