Familial Hypercholesterolemia - HomozygousPhase 3NCT02173158
Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
A Phase 3 study testing lomitapide in people with Familial Hypercholesterolemia - Homozygous, run by Aegerion Pharmaceuticals, Inc. at 6 sites in Japan.
Trial summary
Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Hypercholesterolemia - Homozygous
- What is being tested?
- lomitapide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 6 sitesJapan
- Study dates
- 2 Apr 2014to 17 Dec 2015
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy
- Diagnosis of functional HoFH
- Body weight ≥ 40 kg and < 136 kg
- Negative pregnancy test at screening
Who cannot join
- Uncontrolled hypertension
- History of chronic renal insufficiency
- History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening
- Any major surgical procedure occurring < 3 months prior to the screening visit
- Cardiac insufficiency
- Previous organ transplantation
Show all 16Show fewer
- History of a non-skin malignancy within the previous 3 years
- Patients who are not able to limit their alcohol intake
- Participation in an investigational drug study within 6 weeks prior to the screening visit
- Known significant gastrointestinal bowel disease
- Nursing mothers
- Serious or unstable medical or psychological conditions
- Requirement for certain prohibited medications known to be potentially hepatotoxic
- Use of strong or moderate inhibitors of CYP3A4
- Use of simvastatin at doses >10 mg per day
- Documented diagnosis of any liver disease
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Japan6 sites
Study site
Bunkyo-ku, Tokyo, 113-8519
Study site
Kanazawa, Ishikawa-ken, 920-8641
Study site
Kurume-shi, Fukuoka, 830-8522
Study site
Osakashi, Osaka, 530-00001
Study site
Suita-shi, Osaka, 565-8565
Study site
Tokorozawa, Saitama, 359-1142
About this study
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
Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.
Read the detailed 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 a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.
- Being tested
- lomitapide
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
lomitapideDrug
Also called Juxtapid, Lojuxta
| Study group | Role | What they receive |
|---|---|---|
| lomitapide | Experimental | Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. Receives: Drug: lomitapide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in LDL-C
Mean percent change from baseline
Measured: Baseline to Week 26
Secondary outcomes (9)
Change in Total Cholesterol
Mean percent change from baseline
Measured: Baseline to Week 56
Change in Apo B
Mean percent change from baseline
Measured: Baseline to Week 56
Change in Triglycerides
Mean percent change from baseline
Measured: Baseline to Week 56
Change in Non-HDL-C
Mean percent change from baseline
Measured: Baseline to Week 56
Change in VLDL-C
Mean percent change from baseline
Measured: Baseline to Week 56
Change in Lp(a)
Mean percent change from baseline
Measured: Baseline to Week 56
Change in HDL-C
Mean percent change from baseline
Measured: Baseline to Week 56
Change in Apo AI
Mean percent change from baseline
Measured: Baseline to Week 56
Change in LDL-C
Mean percent change from baseline
Measured: Baseline to Week 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2014 (estimated)
- Study start2 Apr 2014
- Primary completion3 Apr 2015
- Study completion17 Dec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Familial Hypercholesterolemia - HomozygousStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mariko Harada-Shiba, M.D., Ph.D., Principal investigator, National Cerebral and Cardiovascular Center Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02173158
- Sponsor's protocol ID
- AEGR-733-030
- Status last verified
- Feb 2018
- First submitted
- 23 Jun 2014
- Last updated
- 10 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02173158
- Acronym / sponsor ID
- AEGR-733-030
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 2 Apr 2014 (actual)
- Primary completion
- 3 Apr 2015 (actual)
- Study completion
- 17 Dec 2015 (actual)
- First posted
- 24 Jun 2014 (estimated)
- Results
- Posted 10 Oct 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.
Detailed 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 a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| lomitapide | Experimental | Drug: lomitapideMaximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy
- Diagnosis of functional HoFH
- Body weight ≥ 40 kg and < 136 kg
- Negative pregnancy test at screening
Key exclusion criteria (16 listed)
- Uncontrolled hypertension
- History of chronic renal insufficiency
- History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening
- Any major surgical procedure occurring < 3 months prior to the screening visit
- Cardiac insufficiency
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| lomitapideAlso: Juxtapid, Lojuxta | Drug | lomitapide |
Primary outcome
Percent Change in LDL-C
Time frame: Baseline to Week 26
Mean percent change from baseline
9 secondary outcomes
Change in Total Cholesterol
Time frame: Baseline to Week 56
Change in Apo B
Time frame: Baseline to Week 56
Change in Triglycerides
Time frame: Baseline to Week 56
Change in Non-HDL-C
Time frame: Baseline to Week 56
Change in VLDL-C
Time frame: Baseline to Week 56
Change in Lp(a)
Time frame: Baseline to Week 56
Change in HDL-C
Time frame: Baseline to Week 56
Change in Apo AI
Time frame: Baseline to Week 56
Change in LDL-C
Time frame: Baseline to Week 56
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Familial Hypercholesterolemia - Homozygous
Active, not recruitingObservational study
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Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
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Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
RecruitingPhase 3
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
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A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Cardiovascular, Metabolic Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mariko Harada-Shiba, M.D., Ph.D. principal investigator
National Cerebral and Cardiovascular Center Research Institute
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing lomitapide in people with Familial Hypercholesterolemia - Homozygous, run by Aegerion Pharmaceuticals, Inc. at 6 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Oct 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change in LDL-C (time frame: Baseline to Week 26). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2018.