300 Participants Needed

Lomitapide for Hypercholesterolemia

(LOWER Trial)

Recruiting at 47 trial locations
SO
JB
Overseen ByJanet Boylan
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: Amryt Pharma
Must be taking: Lomitapide
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on the long-term safety and effectiveness of the medication lomitapide, which helps manage cholesterol levels. The study seeks individuals who have already incorporated lomitapide into their regular treatment plan. Participants must understand the study requirements and agree to share their health information. This trial excludes individuals taking other experimental medications concurrently. As a Phase 4 trial, this research aims to understand how this FDA-approved and proven effective treatment benefits a broader range of patients.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot be on any investigational drugs (new drugs not yet approved) when you start lomitapide.

What is the safety track record for lomitapide?

Research shows that lomitapide is usually well-tolerated, but it can cause some side effects. Common issues include digestive problems such as diarrhea, nausea, and an upset stomach. Some individuals might also experience back pain or constipation. More serious concerns involve liver problems, so doctors need to monitor liver health with blood tests for those taking lomitapide. Although these side effects might sound concerning, understanding them can aid in management. Prospective trial participants should discuss these possible effects with their doctor.12345

Why are researchers excited about this trial?

Lomitapide is unique because it works by directly inhibiting the microsomal triglyceride transfer protein (MTP), which plays a crucial role in the production of low-density lipoprotein (LDL) cholesterol. Unlike standard treatments for high cholesterol, which often include statins or PCSK9 inhibitors, lomitapide offers a different approach by targeting the production of cholesterol at its source. Researchers are excited about lomitapide because it provides an alternative for patients who might not respond well to existing therapies or who have specific conditions like homozygous familial hypercholesterolemia, where traditional treatments might not be as effective.

What evidence suggests that lomitapide might be an effective treatment for long-term safety and effectiveness?

Research has shown that lomitapide, the treatment under study in this trial, effectively lowers bad cholesterol levels. In one study, patients experienced a reduction of about 53.5% after 24 weeks. Another study found a 50% reduction at the end of 78 weeks. Long-term data indicate that some patients had a reduction of nearly 60% over nine years. Notably, many patients reached healthy cholesterol levels, suggesting that lomitapide can significantly help manage high cholesterol.678910

Who Is on the Research Team?

JB

Janet Boylan

Principal Investigator

Amryt Pharmaceuticals

Are You a Good Fit for This Trial?

Inclusion Criteria

I am 7 years or older and can provide a signed form agreeing to participate.
I have started treatment with lomitapide before or at the time I joined this study.
I understand the study's requirements and can consent, or my child can participate with my consent.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Treatment

Participants receive lomitapide as prescribed by their physician

10 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

10 years

What Are the Treatments Tested in This Trial?

Interventions

  • Lomitapide
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: LomitapideExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Amryt Pharma

Lead Sponsor

Trials
9
Recruited
1,000+

Citations

Long-term effectiveness and safety of lomitapide in patients ...These long-term, real-world data demonstrate that lomitapide substantially reduced LDL-C for up to seven years. Most patients achieved LDL-C ...
Long-term experience with lomitapide treatment in patients ...A mean 53.5% reduction in LDL-C, 53.9% reduction in nonhigh-density lipoprotein cholesterol (non-HDL-C) and –52.4%, in apoB was observed after 24 weeks of ...
Long-Term Efficacy and Safety of the Microsomal ...In a pivotal 78-week open-label trial, lomitapide, titrated to the maximal tolerable dose, decreased LDL-C by 50% at the end of the efficacy ...
Efficacy and safety of lomitapide in homozygous familial ...Indeed, the four patients with the longest follow-up (about 9 years) experienced a median percent LDL-C reduction of 59.6% (IQR 10.2–69.8) using ...
Long-term safety and efficacy of lomitapide in patients with ...After treatment, there was a mean 33% reduction in LDL-C (45% in patients remaining on lomitapide), 65.4% achieved LDL-C <100 mg/dL, and 41.1% ...
Lomitapide - StatPearls - NCBI Bookshelf - NIHLomitapide might lead to gastrointestinal adverse effects such as diarrhea, hepatic steatosis, and increased hepatic enzymes. Pharmacists should ...
Lomitapide (oral route) - Side effects & dosageMore common · Arm, back, or jaw pain · chest pain or discomfort · chest tightness or heaviness · dark urine · fast, irregular, pounding, or racing heartbeat or pulse ...
Lomitapide Side Effects: Common, Severe, Long TermMore common side effects · acid or sour stomach · back pain · belching · chills · constipation · cough · decreased weight · diarrhea ...
JUXTAPID (lomitapide) - accessdata.fda.govJUXTAPID may cause serious side effects including: 1. Liver problems. JUXTAPID can cause liver problems. • Your doctor should do blood tests to check your liver ...
Juxtapid (lomitapide): Uses, Side Effects, Interactions, ...Common side effects include digestive issues, such as diarrhea, nausea, vomiting, and upset stomach. Serious side effects include liver problems ...
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