Phase 3
N=19
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT00943306 ↗Enrolled (actual)
19
Serious AEs
36.8%
Results posted
May 2018
Primary outcome: Primary: Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) — -45.5 Percent Change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- lomitapide (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Aegerion Pharmaceuticals, Inc.
- Primary completion
- Sep 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) |
-51.1 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) |
-51.1 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) |
-51.1 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) |
-51.1 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) |
-51.1 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) |
-51.1 | — |
| SECONDARY Percent Change in Total Cholesterol |
-43.9 | — |
| SECONDARY Percent Change in Total Cholesterol |
-43.9 | — |
| SECONDARY Percent Change in Total Cholesterol |
-43.9 | — |
| SECONDARY Percent Change in Total Cholesterol |
-43.9 | — |
| SECONDARY Percent Change in Total Cholesterol |
-43.9 | — |
| SECONDARY Percent Change in Total Cholesterol |
-43.9 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) |
-60.9 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) |
-60.9 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) |
-60.9 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) |
-60.9 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) |
-60.9 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) |
-60.9 | — |
| SECONDARY Percent Change in Triglycerides |
29.2 | — |
| SECONDARY Percent Change in Triglycerides |
29.2 | — |
| SECONDARY Percent Change in Triglycerides |
29.2 | — |
| SECONDARY Percent Change in Triglycerides |
29.2 | — |
| SECONDARY Percent Change in Triglycerides |
29.2 | — |
| SECONDARY Percent Change in Triglycerides |
29.2 | — |
| SECONDARY Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-46.6 | — |
| SECONDARY Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-46.6 | — |
| SECONDARY Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-46.6 | — |
| SECONDARY Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-46.6 | — |
| SECONDARY Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-46.6 | — |
| SECONDARY Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-46.6 | — |
| SECONDARY Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) |
30.6 | — |
| SECONDARY Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) |
30.6 | — |
| SECONDARY Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) |
30.6 | — |
| SECONDARY Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) |
30.6 | — |
| SECONDARY Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) |
30.6 | — |
| SECONDARY Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) |
30.6 | — |
| SECONDARY Percent Change in Lp(a) |
-10.4 | — |
| SECONDARY Percent Change in Lp(a) |
-10.4 | — |
| SECONDARY Percent Change in Lp(a) |
-10.4 | — |
| SECONDARY Percent Change in Lp(a) |
-10.4 | — |
| SECONDARY Percent Change in Lp(a) |
-10.4 | — |
| SECONDARY Percent Change in Lp(a) |
-10.4 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) |
-23.5 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) |
-23.5 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) |
-23.5 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) |
-23.5 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) |
-23.5 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) |
-23.5 | — |
| SECONDARY Percent Change in Apolipoprotein AI (Apo AI) |
-30.5 | — |
| SECONDARY Percent Change in Apolipoprotein AI (Apo AI) |
-30.5 | — |
| SECONDARY Percent Change in Apolipoprotein AI (Apo AI) |
-30.5 | — |
| SECONDARY Percent Change in Apolipoprotein AI (Apo AI) |
-30.5 | — |
| SECONDARY Percent Change in Apolipoprotein AI (Apo AI) |
-30.5 | — |
| SECONDARY Percent Change in Apolipoprotein AI (Apo AI) |
-30.5 | — |
Summary
This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.
Eligibility Criteria
Inclusion Criteria
- Completed UP1002 or 733-005.
- Willing and able to provide consent and comply with the requirements of the study protocol.
Exclusion Criteria
- Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.
Data sourced from ClinicalTrials.gov (NCT00943306). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.