Trial · NCT00607373
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
NCT00607373, Phase 3, Completed, Kastle Therapeutics, LLC; results first posted 2013-03-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia. Trials, record NCT00607373. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00607373
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
- NCT number
- NCT00607373
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 51 participants, actual
- Lead sponsor
- Kastle Therapeutics, LLC
- Conditions
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
- Interventions
- mipomersen, Placebo
- First posted
- 2008-02-05
- Last updated
- 2016-09-09
- Other registries' IDs
- eudract:2005-003449-15
- Results first posted
- 2013-03-21
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | 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. |
| Primary | LDL-C at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
| Secondary | Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | 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. |
| Secondary | Apo-B at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 ) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
| Secondary | Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | 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. |
| Secondary | Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
| Secondary | Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | 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. |
| Secondary | Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
| Other | Percentage Change From Baseline in Triglycerides at Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | 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. |
| Other | Triglycerides at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set (FAS). The FAS, which represents the practically-feasible intent-to-treat (ITT) population as delineated in ICH Guideline E9, consists of treated participants with a valid baseline and at least one post-baseline LDL-C measure. | percentage of baseline | MEAN |
| SECONDARY | Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | percentage of baseline | MEAN |
| SECONDARY | Apo-B at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 ) | Full analysis set | mg/dL | MEAN |
| SECONDARY | Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | percentage of baseline | MEAN |
| SECONDARY | Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | mg/dL | MEAN |
| PRIMARY | LDL-C at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | mg/dL | MEAN |
| SECONDARY | Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | percentage of baseline | MEAN |
| SECONDARY | Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | mg/dL | MEAN |
| OTHER_PRE_SPECIFIED | Percentage Change From Baseline in Triglycerides at Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | percentage of baseline | MEDIAN |
| OTHER_PRE_SPECIFIED | Triglycerides at Baseline and the Primary Efficacy Time Point (PET) | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 | Full analysis set | mg/dL | MEDIAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | Placebo | -3.31 | Standard Deviation 17.06 | percentage of baseline | |
| Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | Mipomersen | -24.66 | Standard Deviation 19.85 | percentage of baseline | |
| Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Placebo | -2.54 | Standard Deviation 12.56 | percentage of baseline | |
| Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Mipomersen | -26.77 | Standard Deviation 17.04 | percentage of baseline | |
| Apo-B at Baseline and the Primary Efficacy Time Point (PET) | Placebo | Baseline | 259.2 | Standard Deviation 84.4 | mg/dL |
| Apo-B at Baseline and the Primary Efficacy Time Point (PET) | Mipomersen | Baseline | 283.1 | Standard Deviation 78.4 | mg/dL |
| Apo-B at Baseline and the Primary Efficacy Time Point (PET) | Placebo | PET | 252.6 | Standard Deviation 85.0 | mg/dL |
| Apo-B at Baseline and the Primary Efficacy Time Point (PET) | Mipomersen | PET | 205.4 | Standard Deviation 70.0 | mg/dL |
| Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Placebo | -1.98 | Standard Deviation 14.82 | percentage of baseline | |
| Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Mipomersen | -21.20 | Standard Deviation 17.69 | percentage of baseline |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period | STARTED | Placebo | 17 | |
| Treatment Period | STARTED | Mipomersen | 34 | |
| Treatment Period | COMPLETED | Placebo | 17 | |
| Treatment Period | COMPLETED | Mipomersen | 28 | |
| Treatment Period | NOT COMPLETED | Placebo | 0 | |
| Treatment Period | NOT COMPLETED | Mipomersen | 6 | |
| Follow-up Period | STARTED | Placebo | 1 | |
| Follow-up Period | STARTED | Mipomersen | 11 | |
| Follow-up Period | COMPLETED | Placebo | 0 | |
| Follow-up Period | COMPLETED | Mipomersen | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events192
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 1 | 17 | |
| other | Anaemia | Blood and lymphatic system disorders | Mipomersen | 2 | 34 | |
| other | Angina pectoris | Cardiac disorders | Placebo | 0 | 17 | |
| other | Angina pectoris | Cardiac disorders | Mipomersen | 1 | 34 | |
| other | Aortic valve disease | Cardiac disorders | Placebo | 0 | 17 | |
| other | Aortic valve disease | Cardiac disorders | Mipomersen | 1 | 34 | |
| other | Cardiac discomfort | Cardiac disorders | Placebo | 0 | 17 | |
| other | Cardiac discomfort | Cardiac disorders | Mipomersen | 1 | 34 | |
| other | Palpitations | Cardiac disorders | Placebo | 0 | 17 | |
| other | Palpitations | Cardiac disorders | Mipomersen | 1 | 34 |
1 to 10 of 192Source ClinicalTrials.gov
See all 192 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | ||
| Total, all-cause mortality | Mipomersen | ||
| Total, other adverse events | Placebo | 13 | 17 |
| Total, other adverse events | Mipomersen | 30 | 34 |
| Total, serious adverse events | Placebo | 1 | 17 |
| Total, serious adverse events | Mipomersen | 2 | 34 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Mipomersen vs Placebo: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00607373
GET https://conductscience.org/api/trials/v1/NCT00607373/results?page=2