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

August 1, 2016 updated by: Kastle Therapeutics, LLC

A Phase 2, Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Safety, Efficacy, and Pharmacokinetics of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

44

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
  • United States
    • Illinois
      • Chicago, Illinois, United States, 60610
    • Maine
      • Auburn, Maine, United States, 04210
      • Scarborough, Maine, United States, 04074
    • New York
      • New York City, New York, United States, 10021
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27103
    • Ohio
      • Cincinnati, Ohio, United States, 45229
    • Texas
      • Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort A
Loading doses followed by weekly maintenance doses
Drug: ISIS 301012 or Placebo
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
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
Experimental: Cohort B
Loading doses followed by weekly maintenance doses
Drug: ISIS 301012 or Placebo
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
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
Experimental: Cohort C
Loading doses followed by weekly maintenance doses
Drug: ISIS 301012 or Placebo
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
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
Experimental: Cohort D
Loading doses followed by extended weekly maintenance doses
Drug: ISIS 301012 or Placebo
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Drug: ISIS 301012 or Placebo
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percent reduction in LDL-cholesterol from baseline
Time Frame: Week 7 (Cohorts A-C), Week 15 (Cohort D)
Week 7 (Cohorts A-C), Week 15 (Cohort D)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Kastle Therapeutics, LLC

Collaborators

Ionis Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2006

Primary Completion (Actual)

July 1, 2007

Study Completion (Actual)

December 1, 2007

Study Registration Dates

First Submitted

January 20, 2006

First Submitted That Met QC Criteria

January 20, 2006

First Posted (Estimate)

January 24, 2006

Study Record Updates

Last Update Posted (Estimate)

August 3, 2016

Last Update Submitted That Met QC Criteria

August 1, 2016

Last Verified

August 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 301012CS9
  • 2005-004797-24 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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