Hypercholesterolemia Clinical Trial
— RADICHOL 1Official title:
A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012)
in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
This study consisted of a 26-week treatment period and a 24-week post-treatment follow-up
period. Following treatment and Week 28 evaluations, participants could elect to enroll in
an open-label extension study (301012-CS6; NCT00694109). Participants who were not eligible
or elected not to enroll in the open-label extension study or who discontinued during the
28-week treatment period were followed in this study for 24 weeks from administration of the
last dose of study drug.
Status | Completed |
Enrollment | 51 |
Est. completion date | March 2009 |
Est. primary completion date | March 2009 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 12 Years and older |
Eligibility |
Inclusion Criteria: - Diagnosis of Homozygous Familial Hypercholesterolemia (HoFH) - Stable lipid-lowering therapy for 12 weeks - Stable weight for 6 weeks - Stable low fat diet for 8 weeks Exclusion Criteria: - Significant health problems in the recent past including heart attack, stroke, blood disorders, cancer, or digestive problems |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Genzyme, a Sanofi Company | Ionis Pharmaceuticals, Inc. |
United States, Brazil, Canada, Singapore, South Africa, Taiwan, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Percentage Change From Baseline in Triglycerides at Primary Efficacy Time Point (PET) | Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Triglycerides at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Percentage Change From Baseline in Lipoprotein(a) at Primary Efficacy Time Point (PET) | Lipoprotein(a) was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Lipoprotein(a) at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Percentage Change From Baseline in Very-Low-Density Lipoprotein Cholesterol (VLDL-C) at Primary Efficacy Time Point (PET) | VLDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | VLDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Change From Baseline in Ratio of Low-density Lipoprotein Cholesterol (LDL-C) to High-density Lipoprotein Cholesterol (HDL-C) at Primary Efficacy Time Point (PET) | LDL-C and HDL-C were measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Ratio of LDL-C to HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Percent Change From Baseline in Apolipoprotein A1 (Apo-A1) at Primary Efficacy Time Point (PET) | Apo-A1 was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Apo-A1 at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | Percentage Change From Baseline in High-Density Lipoprotein Cholesterol (HDL-C) at Primary Efficacy Time Point (PET) | HDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Other | HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Primary | Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | LDL-C was measured in mg/dL. Samples were taken following an overnight fast. For patients with triglycerides <400 mg/dL, LDL-C was obtained using Friedewald's calculation; and for patients with triglycerides >=400 mg/dL, LDL-C was directly measured by the central laboratory using ultracentrifugation. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. If the Study Day 1 and screening LDL-C values were >12% different (relative to the maximum value), then the screening value was not used, because the Study Day 1 value represents the best estimate of the patient's condition at the beginning of study drug administration. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Primary | LDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Secondary | Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Apo-B was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Secondary | Apo-B at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 ) | No |
Secondary | Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Secondary | Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Secondary | Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET) | Non-HDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
Secondary | Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | No |
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