Trial · NCT00694109
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
NCT00694109, Phase 3, Completed, Kastle Therapeutics, LLC; results first posted 2015-12-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia. Trials, record NCT00694109. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00694109
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
- NCT number
- NCT00694109
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 144 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 Sodium
- First posted
- 2008-06-10
- Last updated
- 2016-09-09
- Other registries' IDs
- eudract:2005-003450-10
- Results first posted
- 2015-12-21
Source ClinicalTrials.gov
Registered outcomes21
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) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Primary | Percent Change From Baseline in Apolipoprotein B (Apo B) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Primary | Percent Change From Baseline in Total Cholesterol | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Primary | Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Secondary | Percent Change From Baseline in Triglycerides | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Secondary | Percent Change From Baseline in Lipoprotein (a) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Secondary | Percent Change From Baseline in LDL Particles' Size (Total) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Secondary | Percent Change From Baseline in LDL Particles' Size (Large) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Secondary | Percent Change From Baseline in LDL Particles' Size (Medium) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
| Secondary | Percent Change From Baseline in LDL Particles' Size (Small) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study). |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
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) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. | percent change | MEAN |
| PRIMARY | Percent Change From Baseline in Apolipoprotein B (Apo B) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. | percent change | MEAN |
| PRIMARY | Percent Change From Baseline in Total Cholesterol | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. | percent change | MEAN |
| PRIMARY | Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in Triglycerides | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in Lipoprotein (a) | Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in LDL Particles' Size (Total) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. Number of participants analyzed = participants with available data for LDL Particles' Size (Total). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in LDL Particles' Size (Large) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. Number of participants analyzed = participants with available data for LDL Particles' Size (Large). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in LDL Particles' Size (Medium) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. Number of participants analyzed = participants with available data for LDL Particles' Size (Medium). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in LDL Particles' Size (Small) | Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years) | Analysis was performed on Safety set. Here n=participants with lipid parameter assessment at specified time. Number of participants analyzed = participants with available data for LDL Particles' Size (Small). | percent change | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group159
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) | Mipomersen | week 26 (n = 130) | -28.5 | 95% Confidence Interval -31.9 to -25.1 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 52 (n = 111) | -27 | 95% Confidence Interval -31.2 to -22.8 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 76 (n = 66) | -27.3 | 95% Confidence Interval -33 to -21.6 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 104 (n = 57) | -27.9 | 95% Confidence Interval -33.9 to -21.8 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 130 (n = 42) | -21.9 | 95% Confidence Interval -31.1 to -12.7 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 156 (n = 30) | -21.4 | 95% Confidence Interval -31.2 to -11.7 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 182 (n = 26) | -23.6 | 95% Confidence Interval -36.6 to -10.6 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 208 (n = 27) | -26.3 | 95% Confidence Interval -36.4 to -16.2 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | week 234 (n = 17) | -22.5 | 95% Confidence Interval -34.3 to -10.6 | percent change |
| Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) | Mipomersen | 24 weeks post last dose (n=117) | 1.6 | 95% Confidence Interval -2.6 to 5.9 | percent change |
1 to 10 of 159Source ClinicalTrials.gov
See all 159 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Mipomersen | 142 | |
| Overall Study | Treated | Mipomersen | 141 | |
| Overall Study | Consented 2 Years Additional Treatment | Mipomersen | 42 | |
| Overall Study | Completed Consented Length of Treatment | Mipomersen | 60 | |
| Overall Study | COMPLETED | Mipomersen | 25 | |
| Overall Study | NOT COMPLETED | Mipomersen | 117 |
Source ClinicalTrials.gov
Adverse events553
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 | Mipomersen | 9 | 141 | |
| other | Leukopenia | Blood and lymphatic system disorders | Mipomersen | 1 | 141 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Mipomersen | 3 | 141 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Mipomersen | 5 | 141 | |
| other | Angina pectoris | Cardiac disorders | Mipomersen | 14 | 141 | |
| other | Aortic valve disease | Cardiac disorders | Mipomersen | 1 | 141 | |
| other | Aortic valve incompetence | Cardiac disorders | Mipomersen | 1 | 141 | |
| other | Atrial fibrillation | Cardiac disorders | Mipomersen | 4 | 141 | |
| other | Atrioventricular block second degree | Cardiac disorders | Mipomersen | 1 | 141 | |
| other | Bradycardia | Cardiac disorders | Mipomersen | 1 | 141 |
1 to 10 of 553Source ClinicalTrials.gov
See all 553 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Mipomersen | ||
| Total, other adverse events | Mipomersen | 141 | 141 |
| Total, serious adverse events | Mipomersen | 36 | 141 |
Source ClinicalTrials.gov
Papers2
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00694109
GET https://conductscience.org/api/trials/v1/NCT00694109/registered?page=2