Trial · NCT00943306
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
NCT00943306, Phase 3, Completed, Aegerion Pharmaceuticals, Inc.; results first posted 2018-05-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia. Trials, record NCT00943306. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00943306
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
- NCT number
- NCT00943306
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 19 participants, actual
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Conditions
- Familial Hypercholesterolemia
- Interventions
- lomitapide
- First posted
- 2009-07-22
- Last updated
- 2018-06-13
- Results first posted
- 2018-05-11
Source ClinicalTrials.gov
Registered outcomes54
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 126 | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012). |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 174 | Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012). |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 222 | Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012). |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 246 | Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012). |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 270 | Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012). |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 294 | Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012). |
| Secondary | Percent Change in Total Cholesterol | Baseline and Week 126 | Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012). |
| Secondary | Percent Change in Total Cholesterol | Baseline and Week 174 | Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012). |
| Secondary | Percent Change in Total Cholesterol | Baseline and Week 222 | Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012). |
| Secondary | Percent Change in Total Cholesterol | Baseline and Week 246 | Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012). |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 registered outcomesOutcomes54
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 126 | Week 126 Completers Population | Percent Change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 174 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 222 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 246 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 270 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 294 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Total Cholesterol | Baseline and Week 126 | Week 126 Completers Population | Percent Change | MEAN |
| SECONDARY | Percent Change in Total Cholesterol | Baseline and Week 174 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Total Cholesterol | Baseline and Week 222 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
| SECONDARY | Percent Change in Total Cholesterol | Baseline and Week 246 | Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program. | Percent Change | MEAN |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 outcomesResults by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Lomitapide | -45.5 | Standard Deviation 31.35 | Percent Change | |
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Lomitapide | -51.0 | Standard Deviation 16.03 | Percent Change | |
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Lomitapide | -58.5 | Standard Deviation 24.25 | Percent Change | |
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Lomitapide | -60.1 | Standard Deviation 18.51 | Percent Change | |
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Lomitapide | -74.0 | Standard Deviation 19.10 | Percent Change | |
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Lomitapide | -51.1 | Standard Deviation 10.11 | Percent Change | |
| Percent Change in Total Cholesterol | Lomitapide | -43.2 | Standard Deviation 25.35 | Percent Change | |
| Percent Change in Total Cholesterol | Lomitapide | -46.9 | Standard Deviation 15.21 | Percent Change | |
| Percent Change in Total Cholesterol | Lomitapide | -51.0 | Standard Deviation 21.34 | Percent Change | |
| Percent Change in Total Cholesterol | Lomitapide | -54.1 | Standard Deviation 16.88 | Percent Change |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Lomitapide | 19 | |
| Overall Study | COMPLETED | Lomitapide | 16 | |
| Overall Study | NOT COMPLETED | Lomitapide | 3 |
Source ClinicalTrials.gov
Adverse events132
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 | Lomitapide | 2 | 19 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | Lomitapide | 1 | 19 | |
| other | Angina Pectoris | Cardiac disorders | Lomitapide | 4 | 19 | |
| other | Aortic Valve Incompetence | Cardiac disorders | Lomitapide | 1 | 19 | |
| other | Chest Pain | Cardiac disorders | Lomitapide | 1 | 19 | |
| other | Coronary Artery Disease | Cardiac disorders | Lomitapide | 1 | 19 | |
| other | Sinus Bradycardia | Cardiac disorders | Lomitapide | 1 | 19 | |
| other | Tinnitus | Ear and labyrinth disorders | Lomitapide | 1 | 19 | |
| other | Vertigo | Ear and labyrinth disorders | Lomitapide | 1 | 19 | |
| other | Hypothyroidism | Endocrine disorders | Lomitapide | 1 | 19 |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lomitapide | 1 | 19 |
| Total, other adverse events | Lomitapide | 17 | 19 |
| Total, serious adverse events | Lomitapide | 7 | 19 |
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/NCT00943306
GET https://conductscience.org/api/trials/v1/NCT00943306/registered?page=2