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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00280995

Enrollment

Registered

2006-01-24

Start date

2006-01-31

Completion date

2007-08-31

Last updated

2016-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypercholesterolemia, Familial

Keywords

LDL-cholesterol, apoB-100, Homozygous Familial Hypercholesterolemia

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

Interventions

DRUGISIS 301012

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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
Percent reduction in LDL-cholesterol from baseline	Week 7 (Cohorts A-C), Week 15 (Cohort D)

Countries

Netherlands, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026