Trial · NCT00730236
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
NCT00730236, Phase 3, Completed, Aegerion Pharmaceuticals, Inc.; results first posted 2013-02-22
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH). Trials, record NCT00730236. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00730236
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
- NCT number
- NCT00730236
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 29 participants, actual
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- AEGR-733
- First posted
- 2008-08-08
- Last updated
- 2018-03-20
- Results first posted
- 2013-02-22
Source ClinicalTrials.gov
Registered outcomes12
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 and Week 26 | Percent change from Baseline in LDL-C |
| Secondary | Percent Change From Baseline in Total Cholesterol (TC) | Baseline and Week 26 | Percent change from Baseline in TC |
| Secondary | Percent Change From Baseline for Apolipoprotein B (Apo B) | Baseline and Week 26 | Percent change from Baseline for Apo B |
| Secondary | Percent Change From Baseline in Triglycerides | Baseline and Week 26 | Percent change from Baseline in triglycerides |
| Secondary | Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) | Baseline and Week 26 | Percent change from Baseline in HDL-C |
| Secondary | Percent Change From Baseline in Non-HDL-C | Baseline and Week 26 | Percent change from Baseline in non-HDL-C |
| Secondary | Percent Change From Baseline in Apolipoprotein AI (Apo AI) | Baseline and Week 26 | Percent change from Baseline in Apo AI |
| Secondary | Absolute Change From Baseline in Hepatic Fat Percent | Baseline and Week 78 | Absolute change from Baseline in hepatic fat percent |
| Secondary | Absolute Change From Baseline in Alanine Aminotransferase (ALT) | Baseline and Week 78 | Absolute change from Baseline in ALT |
| Secondary | Absolute Change From Baseline in Aspartate Aminotransferase (AST) | Baseline and Week 78 | Absolute change from Baseline in AST |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
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 and Week 26 | Intention To Treat (ITT) Population | Percent Change | MEAN |
| SECONDARY | Percent Change From Baseline in Total Cholesterol (TC) | Baseline and Week 26 | ITT Population | Percent Change | MEAN |
| SECONDARY | Percent Change From Baseline for Apolipoprotein B (Apo B) | Baseline and Week 26 | ITT Population | Percent Change | MEAN |
| SECONDARY | Percent Change From Baseline in Triglycerides | Baseline and Week 26 | ITT Population | Percent Change | MEAN |
| SECONDARY | Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) | Baseline and Week 26 | ITT Population | Percent Change | MEDIAN |
| SECONDARY | Percent Change From Baseline in Non-HDL-C | Baseline and Week 26 | ITT Population | Percent Change | MEAN |
| SECONDARY | Percent Change From Baseline in Apolipoprotein AI (Apo AI) | Baseline and Week 26 | ITT Population | Percent Change | MEAN |
| SECONDARY | Absolute Change From Baseline in Hepatic Fat Percent | Baseline and Week 78 | All patients treated | Percent Hepatic Fat | MEAN |
| SECONDARY | Absolute Change From Baseline in Alanine Aminotransferase (ALT) | Baseline and Week 78 | All patients treated | U/L | MEAN |
| SECONDARY | Absolute Change From Baseline in Aspartate Aminotransferase (AST) | Baseline and Week 78 | All patients treated | U/L | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group12
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) | Lomitapide Escalated | -40.1 | Standard Deviation 31.25 | Percent Change | |
| Percent Change From Baseline in Total Cholesterol (TC) | Lomitapide Escalated | -36.4 | Standard Deviation 28.2 | Percent Change | |
| Percent Change From Baseline for Apolipoprotein B (Apo B) | Lomitapide Escalated | -39.4 | Standard Deviation 30.01 | Percent Change | |
| Percent Change From Baseline in Triglycerides | Lomitapide Escalated | -29.0 | Standard Deviation 55.72 | Percent Change | |
| Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) | Lomitapide Escalated | -6.9 | Standard Deviation 19.76 | Percent Change | |
| Percent Change From Baseline in Non-HDL-C | Lomitapide Escalated | -40.0 | Standard Deviation 29.66 | Percent Change | |
| Percent Change From Baseline in Apolipoprotein AI (Apo AI) | Lomitapide Escalated | -6.5 | Standard Deviation 16.12 | Percent Change | |
| Absolute Change From Baseline in Hepatic Fat Percent | Lomitapide Escalated | 6.9 | Standard Deviation 5.03 | Percent Hepatic Fat | |
| Absolute Change From Baseline in Alanine Aminotransferase (ALT) | Lomitapide Escalated | 15.0 | Standard Deviation 29.05 | U/L | |
| Absolute Change From Baseline in Aspartate Aminotransferase (AST) | Lomitapide Escalated | 8.9 | Standard Deviation 20.22 | U/L |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 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 Escalated | 29 | |
| Overall Study | COMPLETED | Lomitapide Escalated | 23 | |
| Overall Study | NOT COMPLETED | Lomitapide Escalated | 6 |
Source ClinicalTrials.gov
Adverse events58
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 | Diarrhoea | Gastrointestinal disorders | Lomitapide Escalated | 23 | 29 | |
| other | Nausea | Gastrointestinal disorders | Lomitapide Escalated | 19 | 29 | |
| other | Vomiting | Gastrointestinal disorders | Lomitapide Escalated | 10 | 29 | |
| other | Dyspepsia | Gastrointestinal disorders | Lomitapide Escalated | 10 | 29 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Lomitapide Escalated | 9 | 29 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Lomitapide Escalated | 4 | 29 | |
| other | Alanine aminotransferase increased | Investigations | Lomitapide Escalated | 5 | 29 | |
| other | Chest pain | General disorders | Lomitapide Escalated | 7 | 29 | |
| other | Abdominal pain | Gastrointestinal disorders | Lomitapide Escalated | 8 | 29 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Lomitapide Escalated | 5 | 29 |
1 to 10 of 58Source ClinicalTrials.gov
See all 58 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lomitapide Escalated | ||
| Total, other adverse events | Lomitapide Escalated | 23 | 29 |
| Total, serious adverse events | Lomitapide Escalated | 3 | 29 |
Source ClinicalTrials.gov
Papers4
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/NCT00730236
GET https://conductscience.org/api/trials/v1/NCT00730236/events?page=2