Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
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
2 other identifiers
interventional
44
2 countries
8
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Feb 2006
8 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 20, 2006
CompletedFirst Posted
Study publicly available on registry
January 24, 2006
CompletedStudy Start
First participant enrolled
February 1, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2007
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2007
CompletedAugust 3, 2016
August 1, 2016
1.4 years
January 20, 2006
August 1, 2016
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percent reduction in LDL-cholesterol from baseline
Week 7 (Cohorts A-C), Week 15 (Cohort D)
Study Arms (4)
Cohort A
EXPERIMENTALLoading doses followed by weekly maintenance doses
Cohort B
EXPERIMENTALLoading doses followed by weekly maintenance doses
Cohort C
EXPERIMENTALLoading doses followed by weekly maintenance doses
Cohort D
EXPERIMENTALLoading doses followed by extended weekly maintenance doses
Interventions
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Kastle Therapeutics, LLClead
- Ionis Pharmaceuticals, Inc.collaborator
Study Sites (8)
Unknown Facility
Chicago, Illinois, 60610, United States
Unknown Facility
Auburn, Maine, 04210, United States
Unknown Facility
Scarborough, Maine, 04074, United States
Unknown Facility
New York, New York, 10021, United States
Unknown Facility
Winston-Salem, North Carolina, 27103, United States
Unknown Facility
Cincinnati, Ohio, 45229, United States
Unknown Facility
Houston, Texas, 77030, United States
Unknown Facility
Amsterdam, 1105 AZ, Netherlands
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Monitor
Genzyme, a Sanofi Company
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
January 20, 2006
First Posted
January 24, 2006
Study Start
February 1, 2006
Primary Completion
July 1, 2007
Study Completion
December 1, 2007
Last Updated
August 3, 2016
Record last verified: 2016-08