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CompletedNCT00280995Updated Aug 3, 2016

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

A Phase 2 interventional study of ISIS 301012 and ISIS 301012 in Hypercholesterolemia, Familial, sponsored by Kastle Therapeutics, LLC. Completed at 6 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-08-03.

Sponsored by Kastle Therapeutics, LLC · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
12
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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

02

Conditions studied

  • Hypercholesterolemia, Familial

Keywords

  • LDL-cholesterol
  • apoB-100
  • Homozygous Familial Hypercholesterolemia
03

In context

Hyperlipoproteinemia Type II

245 studies on the registry are indexed under Hyperlipoproteinemia Type II; 52 are open to participants now.

This study's enrollment of 12 is below the median of 86 across 170 interventional studies indexed under Hyperlipoproteinemia Type II.

Browse Hyperlipoproteinemia Type II studies →

Lead sponsor

Kastle Therapeutics, LLC is the lead sponsor of 17 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
12 participants (actual)

Study arms

  • Experimental
    Cohort A

    Loading doses followed by weekly maintenance doses

    Drug: ISIS 301012

  • Experimental
    Cohort B

    Loading doses followed by weekly maintenance doses

    Drug: ISIS 301012

  • Experimental
    Cohort C

    Loading doses followed by weekly maintenance doses

    Drug: ISIS 301012

  • Experimental
    Cohort D

    Loading doses followed by extended weekly maintenance doses

    Drug: ISIS 301012

Interventions

  • DrugISIS 301012

    100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

  • DrugISIS 301012

    200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

  • DrugISIS 301012

    300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64,71, 78, and 85

  • DrugISIS 301012

    50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36

06

What researchers measure

Primary outcomes

  1. Percent reduction in LDL-cholesterol from baseline

    Time frame: Week 7 (Cohorts A-C), Week 15 (Cohort D)

07

Study locations

6 sites
  • Chicago, Illinois 60610, United States
  • New York City, New York 10021, United States
  • Winston-Salem, North Carolina 27103, United States
  • Cincinnati, Ohio 45229, United States
  • Houston, Texas 77030, United States
  • Amsterdam, 1105 AZ, Netherlands
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 3, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00280995
Lead sponsor
Kastle Therapeutics, LLC
Collaborators
Ionis Pharmaceuticals, Inc.
First posted
Jan 24, 2006
Start date
Jan 2006
Primary completion
Apr 2007
Completion
Aug 2007
Last update
Aug 3, 2016

Study contacts

Medical Monitor
study director · Genzyme, a Sanofi Company
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2016. You cannot join it, but the record below documents what was studied.

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