This trial is active, not recruiting.

Condition familial hypercholesterolemia - homozygous
Treatment lomitapide
Phase phase 3
Sponsor Aegerion Pharmaceuticals, Inc.
Start date April 2014
End date April 2015
Trial size 10 participants
Trial identifier NCT02173158, AEGR-733-030


Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
lomitapide Juxtapid, Lojuxta

Primary Outcomes

Percent change in LDL-C
time frame: 26 Weeks

Secondary Outcomes

Long term safety
time frame: 56 Weeks

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: 1. Japanese male and female HoFH patients aged ≥ 18 years of age 2. Diagnosis of functional HoFH 3. Body weight ≥ 40 kg and < 136 kg 4. Negative pregnancy test at screening Exclusion Criteria: 1. Uncontrolled hypertension 2. History of chronic renal insufficiency 3. History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening 4. Any major surgical procedure occurring < 3 months prior to the screening visit 5. Cardiac insufficiency 6. Previous organ transplantation 7. History of a non-skin malignancy within the previous 3 years 8. Patients who are not able to limit their alcohol intake 9. Participation in an investigational drug study within 6 weeks prior to the screening visit 10. Known significant gastrointestinal bowel disease 11. Nursing mothers 12. Serious or unstable medical or psychological conditions 13. Requirement for certain prohibited medications known to be potentially hepatotoxic 14. Use of strong or moderate inhibitors of CYP3A4 15. Use of simvastatin at doses >10 mg per day 16. Documented diagnosis of any liver disease

Additional Information

Official title A Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy
Principal investigator Mariko Harada-Shiba, M.D., Ph.D.
Description This is a Phase 3, single-arm, open-label, multicenter clinical trial to evaluate both the efficacy and long-term safety of lomitapide in Japanese patients with HoFH receiving maximally-tolerated, stable lipid-lowering therapy. This study is comprised of two phases: a primary 26-week Efficacy Phase followed by a 30-week Safety Phase.
Trial information was received from ClinicalTrials.gov and was last updated in June 2015.
Information provided to ClinicalTrials.gov by Aegerion Pharmaceuticals, Inc..