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Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.
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 a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| lomitapide | Experimental | Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| lomitapide | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in LDL-C | Mean percent change from baseline | Baseline to Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Total Cholesterol | Mean percent change from baseline | Baseline to Week 56 |
| Change in Apo B | Mean percent change from baseline |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mariko Harada-Shiba, M.D., Ph.D. | National Cerebral and Cardiovascular Center Research Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kurume-shi | Fukuoka | 830-8522 | Japan | |||
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| ID | Title | Description |
|---|---|---|
| FG000 | Lomitapide | Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Efficacy Phase |
|
| ||||||||||||||||||
| Safety Phase |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Lomitapide | Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change in LDL-C | Mean percent change from baseline | Full analysis set for the efficacy phase, included all subjects who received study drug and had a baseline and at least one post-baseline assessment | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 26 |
|
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Efficacy Phase | Baseline to Week 26 | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Chest pain | General disorders | MedDRA 17.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alison Long, MD - VP Clinical | Aegerion Pharmaceuticals, Inc. | 857-242-5142 | alison.long@aegerion.com |
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| ID | Term |
|---|---|
| D000090542 | Homozygous Familial Hypercholesterolemia |
| ID | Term |
|---|---|
| D006938 | Hyperlipoproteinemia Type II |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| C473731 | BMS201038 |
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| Baseline to Week 56 |
| Change in Triglycerides | Mean percent change from baseline | Baseline to Week 56 |
| Change in Non-HDL-C | Mean percent change from baseline | Baseline to Week 56 |
| Change in VLDL-C | Mean percent change from baseline | Baseline to Week 56 |
| Change in Lp(a) | Mean percent change from baseline | Baseline to Week 56 |
| Change in HDL-C | Mean percent change from baseline | Baseline to Week 56 |
| Change in Apo AI | Mean percent change from baseline | Baseline to Week 56 |
| Change in LDL-C | Mean percent change from baseline | Baseline to Week 56 |
| Kanazawa |
| Ishikawa-ken |
| 920-8641 |
| Japan |
| Osakashi | Osaka | 530-00001 | Japan |
| Suita-shi | Osaka | 565-8565 | Japan |
| Tokorozawa | Saitama | 359-1142 | Japan |
| Bunkyo-ku | Tokyo | 113-8519 | Japan |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
| Secondary | Change in Total Cholesterol | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in Apo B | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in Triglycerides | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in Non-HDL-C | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in VLDL-C | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in Lp(a) | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in HDL-C | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in Apo AI | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| Secondary | Change in LDL-C | Mean percent change from baseline | Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase | Posted | Mean | Standard Deviation | Percent change | Baseline to Week 56 |
|
|
|
| 9 |
| 0 |
| 9 |
| 9 |
| 9 |
| EG001 | Safety Phase | Week 26 to Week 56 | 0 | 8 | 1 | 8 | 7 | 8 |
| Nausea | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Faeces soft | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Haemorrhoids | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Periodontitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 17.0 | Systematic Assessment |
|
| Device breakage | General disorders | MedDRA 17.0 | Systematic Assessment |
|
| Feeling abnormal | General disorders | MedDRA 17.0 | Systematic Assessment |
|
| Malaise | General disorders | MedDRA 17.0 | Systematic Assessment |
|
| Chemical burn of skin | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
|
| Heat stroke | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
|
| Subcutaneous haematoma | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
|
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 17.0 | Systematic Assessment |
|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 17.0 | Systematic Assessment |
|
| Liver function test abnormal | Investigations | MedDRA 17.0 | Systematic Assessment |
|
| Blood creatine phosphokinase increased | Investigations | MedDRA 17.0 | Systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA 17.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Mylagia | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 17.0 | Systematic Assessment |
|
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Pruritus allergic | Skin and subcutaneous tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA 17.0 | Systematic Assessment |
|
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA 17.0 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 17.0 | Systematic Assessment |
|
Described in site contract
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |