Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
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
2 other identifiers
interventional
12
2 countries
6
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jan 2006
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2006
CompletedFirst Submitted
Initial submission to the registry
January 20, 2006
CompletedFirst Posted
Study publicly available on registry
January 24, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2007
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2007
CompletedAugust 3, 2016
August 1, 2016
1.2 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
Eligibility Criteria
You may qualify if:
- 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.
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 (6)
Unknown Facility
Chicago, Illinois, 60610, 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
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
January 20, 2006
First Posted
January 24, 2006
Study Start
January 1, 2006
Primary Completion
April 1, 2007
Study Completion
August 1, 2007
Last Updated
August 3, 2016
Record last verified: 2016-08