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

NCT ID: NCT00280995

Last Updated: 2016-08-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-01-31

Study Completion Date

2007-08-31

Brief Summary

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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

Detailed Description

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Conditions

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Hypercholesterolemia, Familial

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort A

Loading doses followed by weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012

Intervention Type DRUG

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

Cohort B

Loading doses followed by weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012

Intervention Type DRUG

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

Cohort C

Loading doses followed by weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012

Intervention Type DRUG

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

Cohort D

Loading doses followed by extended weekly maintenance doses

Group Type EXPERIMENTAL

ISIS 301012

Intervention Type DRUG

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

Interventions

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ISIS 301012

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

Intervention Type DRUG

ISIS 301012

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

Intervention Type DRUG

ISIS 301012

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

Intervention Type DRUG

ISIS 301012

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

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role collaborator

Kastle Therapeutics, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Genzyme Coporation

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

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Chicago, Illinois, United States

Site Status

New York, New York, United States

Site Status

Winston-Salem, North Carolina, United States

Site Status

Cincinnati, Ohio, United States

Site Status

Houston, Texas, United States

Site Status

Amsterdam, , Netherlands

Site Status

Countries

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United States Netherlands

Other Identifiers

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EudraCT No.: 2005-004796-38

Identifier Type: -

Identifier Source: secondary_id

301012CS8

Identifier Type: -

Identifier Source: org_study_id

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