Livmarli for Alagille Syndrome
(LEAP Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to evaluate the long-term safety and outcomes of using Livmarli for individuals with Alagille syndrome (a genetic disorder affecting the liver and other organs) or Progressive familial intrahepatic cholestasis (a rare liver disease). Researchers will observe participants over five years to assess Livmarli's effectiveness and identify any long-term effects. Individuals diagnosed with either condition and already prescribed Livmarli might be suitable candidates for this study. As a Phase 4 trial, Livmarli has already received FDA approval and proven effective, and this research seeks to understand how the treatment benefits more patients.
Do I have to stop taking my current medications for the Livmarli trial?
The trial information does not specify if you need to stop taking your current medications. However, you cannot have taken an investigational drug within 30 days before starting Livmarli.
What is the safety track record for Livmarli?
Research has shown that Livmarli, a treatment for certain liver conditions, is generally safe. For Progressive Familial Intrahepatic Cholestasis (PFIC), the FDA has approved Livmarli to treat itching caused by liver issues. Studies indicate it is usually well-tolerated, though some patients might notice changes in liver test results, which are common with this condition.
For Alagille Syndrome (ALGS), Livmarli has over five years of safety data. As with PFIC, some patients may experience changes in liver test results, which is expected. Serious side effects, such as liver injury, can occur, but they are rare.
Overall, Livmarli has been used safely for these conditions, but regular monitoring remains important. Patients should always discuss any concerns with a healthcare provider.12345Why are researchers excited about this trial?
Unlike the standard treatments for progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS), which often involve medications like ursodeoxycholic acid or surgical interventions, Livmarli offers a novel approach by targeting the root of the problem—bile acids. Livmarli is a selective inhibitor of the ileal bile acid transporter (IBAT), which helps reduce the toxic buildup of bile acids in the liver and bloodstream. This makes Livmarli unique because it not only aims to alleviate symptoms but also addresses the underlying cause of these conditions. Researchers are excited about Livmarli because it holds the potential to improve long-term liver health and quality of life for patients with these challenging disorders.
What evidence suggests that Livmarli might be an effective treatment for Alagille syndrome or Progressive familial intrahepatic cholestasis?
In this trial, participants with Progressive Familial Intrahepatic Cholestasis (PFIC) or Alagille syndrome (ALGS) will receive Livmarli. Previous studies have shown promising results for Livmarli in both conditions. Specifically, for PFIC, 64% of patients experienced much less itching within the first six months, and it also helped children grow taller. For ALGS, 84% of patients had a significant decrease in itching during the first year of treatment, with benefits lasting up to seven years. This research supports Livmarli's potential effectiveness in managing symptoms of both conditions.678910
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants are treated with Livmarli for long-term safety and clinical outcomes
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Livmarli
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
* A clinically and/or genetically confirmed PFIC diagnosis * Prescribed Livmarli
* A clinically and/or genetically confirmed ALGS diagnosis * Participant prescribed Livmarli
Find a Clinic Near You
Who Is Running the Clinical Trial?
Mirum Pharmaceuticals, Inc.
Lead Sponsor
Citations
Event-free survival of maralixibat-treated patients with ...
In the pivotal clinical trial (ICONIC; NCT02160782) of maralixibat for children with ALGS, participants demonstrated significant improvement in pruritus with 84 ...
Efficacy | LIVMARLI® (maralixibat) | HCP
During the first year of treatment, 84% (n=26/31) of patients with Alagille syndrome experienced clinically meaningful improvements in cholestatic pruritus ...
Real-world experience of maralixibat in Alagille syndrome
Results: Maralixibat appeared to be effective with reductions in pruritus compared to baseline. Consistent with clinical trials, maralixibat was ...
Long-Term SafEty and Clinical Outcomes of LivmArli in ...
The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille ...
Clinical benefits of maralixibat for patients with Alagille ...
P-89. Clinical benefits of maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: data from the MERGE study.
Safety Profile | LIVMARLI® (maralixibat) | HCP
Backed by >5 years of safety data,* LIVMARLI has a well-characterized safety and tolerability profile for cholestatic pruritus in patients with Alagille ...
Important Safety Information | LIVMARLI® (maralixibat)
LIVMARLI can cause serious side effects, including: Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but may ...
LIVMARLI® (maralixibat) oral solution - accessdata.fda.gov
The safety and effectiveness of LIVMARLI have not been established in patients with ALGS less than. 3 months of age. PFIC. The safety and effectiveness of ...
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ema.europa.eu
ema.europa.eu/en/documents/product-information/livmarli-epar-product-information_en.pdfLivmarli, INN-maralixibat - European Medicines Agency
Livmarli is not recommended for children with Alagille syndrome under 2 months of age, or children with PFIC under 3 months of age. This is ...
For patients with Alagille syndrome
IMPORTANT SAFETY INFORMATION. WARNINGS AND PRECAUTIONS. Liver Test Abnormalities: Patients enrolled in clinical trials had abnormal liver tests at baseline.
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