Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Hypercholesterolemia, Familial Clinical Trial

Official title:

A Phase 2, Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Safety, Efficacy, and Pharmacokinetics of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00281008
• Other study ID #: 301012CS9
• Secondary ID: 2005-004797-24
• Status: Completed
• Phase: Phase 2
• First received: January 20, 2006
• Last updated: August 1, 2016
• Start date: February 2006
• Est. completion date: December 2007

Study information

• Verified date: August 2016
• Source: Kastle Therapeutics, LLC
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationNetherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial Summary

The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 (mipomersen) as add-on therapy in subjects with Heterozygous Familial Hypercholesterolemia

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: December 2007
• Est. primary completion date: July 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Weight = 50 kg

• Diagnosis of Heterozygous Familial Hypercholesterolemia.

• Females must be non-pregnant and non-lactating.

• On stable lipid lowering therapy for at least 4 weeks.

• Lipid levels 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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hypercholesterolemia
• Hypercholesterolemia, Familial
• Hyperlipoproteinemia Type II

Intervention

Drug:
• ISIS 301012 or Placebo
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
• ISIS 301012 or Placebo
100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
• ISIS 301012 or Placebo
200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
• ISIS 301012 or Placebo
300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78, and 85

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Kastle Therapeutics, LLC	Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent reduction in LDL-cholesterol from baseline		Week 7 (Cohorts A-C), Week 15 (Cohort D)	No