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

NCT00280995 — Phase 2, Completed, 12 participants, Kastle Therapeutics, LLC

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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

Registry	NCT00280995
Status	Completed
Phase	Phase 2
Study type	Interventional; randomized, parallel, masking: none, treatment
Enrollment	12 (Actual)
Sponsor	Kastle Therapeutics, LLC (Industry); collaborators: Ionis Pharmaceuticals, Inc.
Conditions	Hypercholesterolemia, Familial
Interventions	ISIS 301012 (Drug)
Dates	start 2006-01-01, primary completion 2007-04-01, completion 2007-08-01, first posted 2006-01-24, last updated 2016-08-03
Locations	6 sites in United States, Netherlands

-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

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+Arms and interventions

Arm	Type	Interventions	Description
Cohort A	Experimental	Drug: ISIS 301012	Loading doses followed by weekly maintenance doses
Cohort B	Experimental	Drug: ISIS 301012	Loading doses followed by weekly maintenance doses
Cohort C	Experimental	Drug: ISIS 301012	Loading doses followed by weekly maintenance doses
Cohort D	Experimental	Drug: ISIS 301012	Loading doses followed by extended weekly maintenance doses

ISIS 301012 (Drug): 100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
ISIS 301012 (Drug): 200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
ISIS 301012 (Drug): 300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64,71, 78, and 85
ISIS 301012 (Drug): 50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

+Outcome measures

Primary outcomes

Percent reduction in LDL-cholesterol from baseline [Week 7 (Cohorts A-C), Week 15 (Cohort D)]

+Eligibility

Sex: All, ages: 18 Years to any

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.

+Locations (6)

Facility	City	Country	Status
	Chicago, Illinois	United States
	New York, New York	United States
	Winston-Salem, North Carolina	United States
	Cincinnati, Ohio	United States
	Houston, Texas	United States
	Amsterdam	Netherlands

+Investigators

Medical Monitor, Study director, Genzyme, a Sanofi Company