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Trial record 10 of 19 for:    MIPOMERSEN

A Study to Evaluate 3 Different Dosing Regimens of Mipomersen Administered Via Subcutaneous Injections to Healthy Volunteers

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01061814
Recruitment Status : Completed
First Posted : February 3, 2010
Last Update Posted : August 3, 2016
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Kastle Therapeutics, LLC

Brief Summary:

Hypercholesterolemia is characterized by markedly elevated low density lipoproteins (LDL). Elevated LDL is a major risk factor for coronary heart disease (CHD). Mipomersen is an antisense drug that reduces a protein in the liver cells called apolipoprotein B-100 (apoB-100). ApoB-100 plays a role in producing low density lipoprotein cholesterol (LDL-C) (the 'bad' cholesterol) and moving it from the liver to one's bloodstream. High LDL-C is an independent risk factor for the development of coronary heart disease (CHD) or other diseases of blood vessels. It has been shown that lowering LDL-C reduces the risk of heart attacks and other major adverse cardiovascular events.

Mipomersen is an investigational product being studied to determine if it is safe and effective in lowering LDL-C in specific populations of patients with hypercholesterolemia.

This phase 1 study is being conducted to evaluate 3 different dosing regimens (daily, 3 times per week, or weekly) in healthy volunteers for a total of 3 weeks of dosing. Study procedures will include blood testing and physical examinations to assess the safety and tolerability of the different regimens. Tests will also be done to determine how much of the drug is present in the circulation (blood flow in the body). Specific pharmacokinetic (PK) tests on the blood samples will determine what the body does to the investigational product after it is injected, including how it is absorbed, distributed, the rate at which drug action begins and the duration of the effect.

Eligible subjects will receive study injections of either mipomersen or placebo over a 3 week period followed by a 12 week safety follow-up period.

Condition or disease Intervention/treatment Phase
Healthy Volunteer Drug: mipomersen Drug: Placebo Phase 1

Detailed Description:

This is a prospective, randomized, double blind placebo-controlled, parallel-group, single center Phase 1 study to investigate the relative bioavailability, PK, safety, and tolerability of different sc dosing regimens of mipomersen in healthy volunteers. The bioavailability of 2 test regimens (Cohorts A and B) will be assessed relative to that of the reference treatment regimen (Cohort C). Approximately 84 subjects will be randomized equally to 1 of the 3 treatment regimens and then further randomized in a 3:1 ratio to mipomersen vs. placebo:

Cohort A/Test Treatment Regimen 1: up to 28 subjects will receive a 30 mg sc dose of study drug or matching volume of placebo daily for 3 weeks (21 doses; 630 mg total) Cohort B/Test Treatment Regimen 2: up to 28 subjects will receive a 70 mg sc dose of study drug or matching volume of placebo 3 times a week for 3 weeks (9 doses; 630 mg total) Cohort C/Reference Treatment Regimen: up to 28 subjects will receive a 200 mg sc dose of study drug or matching volume of placebo once a week for 3 weeks (3 doses; 600 mg total) Each subject will participate in a ≤ 6-week screening period, a 3-week treatment period, and a 12-week safety follow-up period.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 84 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Prospective, Randomized, Double-Blind, Placebo-Controlled, Phase 1 Study to Evaluate the Relative Bioavailability, Pharmacokinetics, Safety, and Tolerability of Daily, Thrice Weekly, and Weekly Dosing Regimens of Mipomersen Administered Subcutaneously to Healthy Volunteers
Study Start Date : January 2010
Actual Primary Completion Date : March 2010
Actual Study Completion Date : June 2010

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: mipomersen
30 mg (cohort A), 70mg (cohort B) or 200mg (cohort C) SC daily
Drug: mipomersen
30 mg (cohort A), 70mg (cohort B) or 200mg (cohort C) subcutaneous (SC) dose of study drug daily for 3 weeks
Other Name: ISIS 301012

Placebo Comparator: Placebo
30 mg (cohort A), 70mg (cohort B), or 200mg (cohort C) SC daily
Drug: Placebo
30 mg (cohort A), 70mg (cohort B), or 200mg (cohort C) subcutaneous (SC) dose of study drug daily for 3 weeks

Primary Outcome Measures :
  1. Incidence of treatment-emergent AEs and SAEs [ Time Frame: Assessed at each study visit through 21 weeks ]
  2. Maximum plasma concentration (Cmax) [ Time Frame: variable up to 105 days ]
  3. time to maximal concentration (Tmax) [ Time Frame: variable up to 105 days ]
  4. area under the curve (AUC) based on PK profiles following the first and last dose [ Time Frame: variable up to 105 days ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  • Age 18 to 75, inclusive
  • On acceptable birth control and/or partner compliant with acceptable contraceptive for 4 weeks prior to, during, and 12 weeks after the last study drug dose.
  • In good overall health
  • Body weight > 50 kg and body mass index (BMI) < 32 kg/m2
  • Skin Type I-III based on Fitzpatrick scale

Exclusion Criteria:

  • Clinically significant (CS) abnormalities in medical history, physical examination or laboratory assessments
  • Positive test for human immunodeficiency virus (HIV), hepatitis B or C.
  • Malignancy (with the exception of basal or squamous cell carcinoma of the skin if adequately treated and no recurrence for > 1 year)
  • History of rash, impetigo, or drug allergies
  • Alcohol and/or drug abuse
  • Receiving prescription medications within 30 days, with the exception of contraceptives; Vaccinations are not allowed beginning 3 weeks prior to the first dose of study drug until completion of the Day 28 visit

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01061814

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Canada, Quebec
Anapharm, Inc.
Montreal,, Quebec, Canada, H3X 2H9
Sponsors and Collaborators
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.
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Study Director: Medical Monitor Genzyme, a Sanofi Company
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Kastle Therapeutics, LLC Identifier: NCT01061814    
Other Study ID Numbers: MIPO3200309
First Posted: February 3, 2010    Key Record Dates
Last Update Posted: August 3, 2016
Last Verified: August 2016
Keywords provided by Kastle Therapeutics, LLC:
ApoB (Apolipoprotein B)
LDL (low density lipoprotein)
Additional relevant MeSH terms:
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Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents