Status:

COMPLETED

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

Lead Sponsor:

Kastle Therapeutics, LLC

Collaborating Sponsors:

Ionis Pharmaceuticals, Inc.

Conditions:

Hypercholesterolemia, Familial

Eligibility:

All Genders

18+ years

Phase:

PHASE2

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

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

January 1 2006

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

August 1 2007

Estimated Enrollment :

12 Patients enrolled

Trial Details

Trial ID

NCT00280995

Start Date

January 1 2006

End Date

August 1 2007

Last Update

August 3 2016

Active Locations (6)

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Page 1 of 2 (6 locations)

1

Chicago, Illinois, United States, 60610

2

New York, New York, United States, 10021

3

Winston-Salem, North Carolina, United States, 27103

4

Cincinnati, Ohio, United States, 45229