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Completed

NCT Number: NCT00280995

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

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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Key information

Conditions

Hypercholesterolemia, Familial Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Homozygous Familial Hypercholesterolemia Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Amsterdam, Netherlands

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ISIS 301012

Drug

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

Primary outcomes

  1. Percent reduction in LDL-cholesterol from baseline

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

Sponsors and collaborators

Lead sponsor

Kastle Therapeutics, LLC

Industry

Collaborators

  • Ionis Pharmaceuticals, Inc.

Registry information

Official study title

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

Important dates

Study start
2006
Primary completion
2007
Study completion
2007
First posted
Jan 24, 2006
Registry last updated
Aug 3, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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