Trial · NCT01475825
A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
NCT01475825, Phase 3, Completed, Kastle Therapeutics, LLC; results first posted 2019-03-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol. Trials, record NCT01475825. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01475825
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
- NCT number
- NCT01475825
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 309 participants, actual
- Lead sponsor
- Kastle Therapeutics, LLC
- Conditions
- Hypercholesterolemia, Heterozygous Familial
- Interventions
- mipomersen sodium 200 mg, Placebo, mipomersen sodium 70 mg
- First posted
- 2011-11-21
- Last updated
- 2019-03-26
- Other registries' IDs
- eudract:2011-001480-42
- Results first posted
- 2019-03-14
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | Baseline and Week 61 | The percent change from baseline to PET in LDL-C was measured in Cohort 1, which included participants with severe heterozygous familial hypercholesterolemia (HeFH). Severe HeFH was defined as LDL-C levels ≥200 mg/deciliter (dL) plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents. |
| Secondary | Percent Change From Baseline To PET In LDL-C In Cohort 2 | Baseline, PET (up to 60 weeks) | The percent change from baseline to PET in LDL-C was measured in Cohort 2, which included participants with HeFH with LDL-C levels ≥160 mg/dL and \<200 mg/dL, plus the presence of CHD/risk equivalents. |
| Secondary | Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1 | Baseline and Week 61 | The percent change from baseline to PET in Apo B was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period. |
| Secondary | Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2 | Baseline and Week 61 | The percent change from baseline to PET in Apo B was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period. |
| Secondary | Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1 | Baseline and Week 61 | The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period. |
| Secondary | Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2 | Baseline and Week 61 | The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period. |
Source ClinicalTrials.gov
Outcomes6
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 To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | Baseline and Week 61 | The full analysis set for Cohort 1 included all randomized participants who took at least 1 dose of study drug in Cohort 1 (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Percent | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline To PET In LDL-C In Cohort 2 | Baseline, PET (up to 60 weeks) | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Percent | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1 | Baseline and Week 61 | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Percent | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2 | Baseline and Week 61 | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Percent | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1 | Baseline and Week 61 | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Percent | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2 | Baseline and Week 61 | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Percent | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group24
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 To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | Regimen A: Mipomersen, 200 mg, Once Weekly | -27.17 | Standard Error 5.653 | Percent | |
| Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | Regimen A: Placebo, Once Weekly | -6.77 | Standard Error 6.749 | Percent | |
| Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | Regimen B: Mipomersen, 70 mg, Thrice Weekly | -22.96 | Standard Error 5.362 | Percent | |
| Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | Regimen B: Placebo, Thrice Weekly | -10.62 | Standard Error 5.765 | Percent | |
| Percent Change From Baseline To PET In LDL-C In Cohort 2 | Regimen A: Mipomersen, 200 mg, Once Weekly | -31.20 | Standard Error 8.927 | Percent | |
| Percent Change From Baseline To PET In LDL-C In Cohort 2 | Regimen A: Placebo, Once Weekly | -9.25 | Standard Error 10.621 | Percent | |
| Percent Change From Baseline To PET In LDL-C In Cohort 2 | Regimen B: Mipomersen, 70 mg, Thrice Weekly | -43.60 | Standard Error 8.342 | Percent | |
| Percent Change From Baseline To PET In LDL-C In Cohort 2 | Regimen B: Placebo, Thrice Weekly | -13.57 | Standard Error 9.066 | Percent | |
| Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1 | Regimen A: Mipomersen, 200 mg, Once Weekly | -24.14 | Standard Error 5.058 | Percent | |
| Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1 | Regimen A: Placebo, Once Weekly | -2.83 | Standard Error 6.115 | Percent |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow28
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Regimen A: Mipomersen, 200 mg, Once Weekly | 104 | |
| Overall Study | STARTED | Regimen A: Placebo, Once Weekly | 51 | |
| Overall Study | STARTED | Regimen B: Mipomersen, 70 mg, Thrice Weekly | 102 | |
| Overall Study | STARTED | Regimen B: Placebo, Thrice Weekly | 52 | |
| Overall Study | Randomized and Treated | Regimen A: Mipomersen, 200 mg, Once Weekly | 104 | |
| Overall Study | Randomized and Treated | Regimen A: Placebo, Once Weekly | 51 | |
| Overall Study | Randomized and Treated | Regimen B: Mipomersen, 70 mg, Thrice Weekly | 102 | |
| Overall Study | Randomized and Treated | Regimen B: Placebo, Thrice Weekly | 52 | |
| Overall Study | Completed Blinded Treatment Period | Regimen A: Mipomersen, 200 mg, Once Weekly | 60 | |
| Overall Study | Completed Blinded Treatment Period | Regimen A: Placebo, Once Weekly | 42 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 participant flowAdverse events416
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 | Influenza like illness | General disorders | Regimen A: Mipomersen, 200 mg, Once Weekly | 43 | 104 | |
| other | Influenza like illness | General disorders | Regimen A: Placebo, Once Weekly | 10 | 51 | |
| other | Influenza like illness | General disorders | Regimen B: Mipomersen, 70 mg, Thrice Weekly | 26 | 102 | |
| other | Influenza like illness | General disorders | Regimen B: Placebo, Thrice Weekly | 10 | 52 | |
| other | Injection site erythema | General disorders | Regimen A: Mipomersen, 200 mg, Once Weekly | 8 | 104 | |
| other | Injection site erythema | General disorders | Regimen A: Placebo, Once Weekly | 1 | 51 | |
| other | Injection site erythema | General disorders | Regimen B: Mipomersen, 70 mg, Thrice Weekly | 13 | 102 | |
| other | Injection site erythema | General disorders | Regimen B: Placebo, Thrice Weekly | 1 | 52 | |
| other | Injection site pain | General disorders | Regimen A: Mipomersen, 200 mg, Once Weekly | 13 | 104 | |
| other | Injection site pain | General disorders | Regimen A: Placebo, Once Weekly | 1 | 51 |
1 to 10 of 416Source ClinicalTrials.gov
See all 416 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Regimen A: Mipomersen, 200 mg, Once Weekly | 1 | 104 |
| Total, all-cause mortality | Regimen A: Placebo, Once Weekly | 0 | 51 |
| Total, all-cause mortality | Regimen B: Mipomersen, 70 mg, Thrice Weekly | 1 | 102 |
| Total, all-cause mortality | Regimen B: Placebo, Thrice Weekly | 0 | 52 |
| Total, other adverse events | Regimen A: Mipomersen, 200 mg, Once Weekly | 82 | 104 |
| Total, other adverse events | Regimen A: Placebo, Once Weekly | 27 | 51 |
| Total, other adverse events | Regimen B: Mipomersen, 70 mg, Thrice Weekly | 78 | 102 |
| Total, other adverse events | Regimen B: Placebo, Thrice Weekly | 33 | 52 |
| Total, serious adverse events | Regimen A: Mipomersen, 200 mg, Once Weekly | 17 | 104 |
| Total, serious adverse events | Regimen A: Placebo, Once Weekly | 13 | 51 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26946290 | DERIVED |
Source PubMed
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/NCT01475825
GET https://conductscience.org/api/trials/v1/NCT01475825/results?page=2