Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

This study has been completed.
Sponsor:
Collaborator:
Isis Pharmaceuticals
Information provided by:
Sanofi
ClinicalTrials.gov Identifier:
NCT00280995
First received: January 20, 2006
Last updated: December 2, 2013
Last verified: December 2013
  Purpose

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


Condition Intervention Phase
Hypercholesterolemia, Familial
Drug: ISIS 301012
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Percent reduction in LDL-cholesterol from baseline [ Time Frame: Week 7 (Cohorts A-C), Week 15 (Cohort D) ] [ Designated as safety issue: No ]

Enrollment: 12
Study Start Date: January 2006
Study Completion Date: August 2007
Primary Completion Date: April 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort A
Loading doses followed by weekly maintenance doses
Drug: ISIS 301012
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Experimental: Cohort B
Loading doses followed by weekly maintenance doses
Drug: ISIS 301012
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Experimental: Cohort C
Loading doses followed by weekly maintenance doses
Drug: ISIS 301012
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Experimental: Cohort D
Loading doses followed by extended weekly maintenance doses
Drug: ISIS 301012
300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64,71, 78, and 85

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Weight ≥ 40 kg for Cohorts A, B, & C; Weight ≥ 50 kg for Cohort D
  • Diagnosis of Homozygous Familial Hypercholesterolemia.
  • Female must be non-pregnant and non-lactating.
  • On stable lipid lowering therapy for at least 4 weeks.
  • Lipid values that 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.
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00280995

Locations
United States, Illinois
Chicago, Illinois, United States, 60610
United States, New York
New York City, New York, United States, 10021
United States, North Carolina
Winston-Salem, North Carolina, United States, 27103
United States, Ohio
Cincinnati, Ohio, United States, 45229
United States, Texas
Houston, Texas, United States, 77030
Netherlands
Amsterdam, Netherlands, 1105 AZ
Sponsors and Collaborators
Genzyme, a Sanofi Company
Isis Pharmaceuticals
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Medical Monitor, Genzyme Coporation
ClinicalTrials.gov Identifier: NCT00280995     History of Changes
Other Study ID Numbers: 301012CS8, EudraCT No.: 2005-004796-38
Study First Received: January 20, 2006
Last Updated: December 2, 2013
Health Authority: United States: Food and Drug Administration
Netherlands: Central Committee on Research Involving Human Subjects

Keywords provided by Sanofi:
LDL-cholesterol
apoB-100
Homozygous Familial Hypercholesterolemia

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias

ClinicalTrials.gov processed this record on September 16, 2014