Back to resultsDose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
Primary Purpose
Hypercholesterolemia, Familial
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
ISIS 301012
ISIS 301012
ISIS 301012
ISIS 301012
Sponsored by
About this trial
This is an interventional treatment trial for Hypercholesterolemia, Familial focused on measuring LDL-cholesterol, apoB-100, Homozygous Familial Hypercholesterolemia
Eligibility 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.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
Cohort A
Cohort B
Cohort C
Cohort D
Arm Description
Loading doses followed by weekly maintenance doses
Loading doses followed by weekly maintenance doses
Loading doses followed by weekly maintenance doses
Loading doses followed by extended weekly maintenance doses
Outcomes
Primary Outcome Measures
Percent reduction in LDL-cholesterol from baseline
Secondary Outcome Measures
Full Information
NCT ID
NCT00280995
First Posted
January 20, 2006
Last Updated
August 1, 2016
Sponsor
Kastle Therapeutics, LLC
Collaborators
Ionis Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT00280995
Brief Title
Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2016
Overall Recruitment Status
Completed
Study Start Date
January 2006 (undefined)
Primary Completion Date
April 2007 (Actual)
Study Completion Date
August 2007 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Kastle Therapeutics, LLC
Collaborators
Ionis Pharmaceuticals, Inc.
4. Oversight
5. Study Description
Brief Summary
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypercholesterolemia, Familial
Keywords
LDL-cholesterol, apoB-100, Homozygous Familial Hypercholesterolemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort A
Arm Type
Experimental
Arm Description
Loading doses followed by weekly maintenance doses
Arm Title
Cohort B
Arm Type
Experimental
Arm Description
Loading doses followed by weekly maintenance doses
Arm Title
Cohort C
Arm Type
Experimental
Arm Description
Loading doses followed by weekly maintenance doses
Arm Title
Cohort D
Arm Type
Experimental
Arm Description
Loading doses followed by extended weekly maintenance doses
Intervention Type
Drug
Intervention Name(s)
ISIS 301012
Intervention Description
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Intervention Type
Drug
Intervention Name(s)
ISIS 301012
Intervention Description
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Intervention Type
Drug
Intervention Name(s)
ISIS 301012
Intervention Description
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
Intervention Name(s)
ISIS 301012
Intervention Description
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Primary Outcome Measure Information:
Title
Percent reduction in LDL-cholesterol from baseline
Time Frame
Week 7 (Cohorts A-C), Week 15 (Cohort D)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60610
Country
United States
City
New York City
State/Province
New York
ZIP/Postal Code
10021
Country
United States
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27103
Country
United States
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
12. IPD Sharing Statement
Learn more about this trial
Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
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