Seladelpar for Primary Biliary Cholangitis

Not currently recruiting at 214 trial locations

Overseen By Gilead Clinical Study Information Center

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Gilead Sciences

Must not be taking: Fibrates, Obeticholic acid

Disqualifiers: Cancer, Hepatitis, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 60 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the safety and tolerability of Seladelpar for individuals with Primary Biliary Cholangitis (PBC), a condition that gradually destroys the bile ducts in the liver. The trial includes two experimental groups taking either 5 mg or 10 mg of Seladelpar. It is suitable for those who have previously participated in a PBC study with Seladelpar. Participants should not have other significant medical conditions, such as active infections or cancer, that could interfere with the trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for PBC.

Do I have to stop taking my current medications for this trial?

The trial protocol does not specify if you must stop all current medications, but certain medications are prohibited. You must stop using colchicine, methotrexate, azathioprine, long-term systemic steroids, fibrates, obeticholic acid, and any experimental treatments for PBC within specific timeframes before screening. Other medications affecting liver or GI functions may also be prohibited and should be discussed with the medical monitor.

Will I have to stop taking my current medications?

You may need to stop taking certain medications before joining the trial. Specifically, you cannot use colchicine, methotrexate, azathioprine, long-term systemic steroids, fibrates, obeticholic acid, or any experimental treatments for PBC within a few months before the trial. It's best to discuss your current medications with the trial team to see if any need to be adjusted.

Is there any evidence suggesting that Seladelpar is likely to be safe for humans?

Research has shown that seladelpar is generally safe and well-tolerated for people with primary biliary cholangitis (PBC). Clinical trials found its safety comparable to a placebo, indicating it does not cause more side effects than a fake treatment. Even patients with liver scarring (cirrhosis) tolerated it well. In studies, patients used seladelpar for up to two years without major problems. This evidence strongly supports seladelpar's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for PBC?

Unlike the standard treatments for Primary Biliary Cholangitis, which often include ursodeoxycholic acid or obeticholic acid, Seladelpar offers a unique approach. Researchers are excited because Seladelpar works by targeting the PPAR-delta pathway, which may provide anti-inflammatory and anti-cholestatic effects. This new mechanism of action could potentially improve liver health and reduce symptoms more effectively than existing options. Additionally, Seladelpar is available in different dosages (5 mg and 10 mg capsules), allowing for tailored treatment plans.

What evidence suggests that Seladelpar might be an effective treatment for Primary Biliary Cholangitis?

Research has shown that seladelpar effectively treats primary biliary cholangitis (PBC). Studies have found that 62% of patients using seladelpar experienced improved liver function. The treatment also reduces severe itching, known as pruritus, and has few side effects. In this trial, participants will receive either 5 mg or 10 mg capsules of seladelpar. Seladelpar is considered a good option for second-line therapy, meaning it can be used if the first treatment is ineffective. It helps improve the disease and symptoms over time. Overall, evidence supports its potential to help people with PBC feel better and manage their condition. ^{1 3 6 7 8}

Who Is on the Research Team?

Gilead Study Director

Principal Investigator

Gilead Sciences

Are You a Good Fit for This Trial?

This trial is for individuals who have previously participated in a seladelpar study for Primary Biliary Cholangitis (PBC), are not pregnant or breastfeeding, and agree to use two forms of birth control. It's not open to those with certain liver conditions, kidney issues, cancer within the last 2 years, chronic viral hepatitis, or on specific medications affecting liver function.

Inclusion Criteria

You have previously participated in a study involving the drug seladelpar for PBC (primary biliary cholangitis).

I am using two forms of birth control during and 90 days after the study.

Must have given written informed consent (signed and dated)

Exclusion Criteria

My liver disease is advanced, with low albumin and high bilirubin levels.

My liver function score is 12 or higher, considering my anticoagulation medication.

I am not pregnant or breastfeeding.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive seladelpar to evaluate long-term safety and tolerability

60 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive seladelpar for long-term evaluation

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Seladelpar

Trial Overview The safety and tolerability of Seladelpar capsules at doses of 5 mg and 10 mg are being tested over a long term in subjects with PBC. This open-label study allows all participants to know which treatment they're receiving.

How Is the Trial Designed?

Group I: Seladelpar 5 mg Capsules Experimental Treatment 1 Intervention

Group II: Seladelpar 10 mg Capsule Experimental Treatment 1 Intervention

Seladelpar is already approved in United States, European Union for the following indications:

Approved in United States as Livdelzi for:

• Primary biliary cholangitis

Approved in European Union as Livdelzi for:

• Primary biliary cholangitis

Who Is Running the Clinical Trial?

Gilead Sciences

Lead Sponsor

Trials

1,150

Recruited

878,000+

Daniel O'Day

Gilead Sciences

Chief Executive Officer since 2019

MBA from Columbia University

Dietmar Berger

Gilead Sciences

Chief Medical Officer

MD and PhD from Albert-Ludwigs University School of Medicine

CymaBay Therapeutics, Inc.

Lead Sponsor

Trials

23

Recruited

3,200+

Published Research Related to This Trial

In a 1-year open-label study involving 101 patients with primary biliary cholangitis (PBC), seladelpar treatment significantly improved pruritus in 58% of patients on a lower dose and 93% on a higher dose, indicating its efficacy in alleviating this distressing symptom.

Patients also reported substantial improvements in quality of life, including reduced sleep disturbances related to itching, alongside significant biochemical changes such as reductions in serum bile acids, highlighting seladelpar's potential as an effective treatment for PBC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Seladelpar improved measures of pruritus, sleep, and fatigue and decreased serum bile acids in patients with primary biliary cholangitis. Kremer, AE., Mayo, MJ., Hirschfield, G., et al.[2022]

In a phase 3 study, seladelpar (10 mg) significantly improved liver biochemistry in patients with primary biliary cholangitis (PBC) who did not respond well to standard treatment, with 78.2% of patients meeting the primary efficacy endpoint compared to only 12.5% in the placebo group.

Seladelpar also effectively reduced pruritus (itching) and showed a good safety profile, with no serious treatment-related adverse events reported, indicating it is a well-tolerated option for patients with PBC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study. Hirschfield, GM., Shiffman, ML., Gulamhusein, A., et al.[2023]

Seladelpar, a selective PPAR-δ agonist, was found to be safe for long-term use in 106 patients with primary biliary cholangitis (PBC), with no serious treatment-related adverse events reported over a 2-year period.

The treatment significantly improved biochemical markers of cholestasis and liver injury, with normalization rates of alkaline phosphatase increasing from 66% to 79% and bilirubin normalization in 43% of patients with elevated levels at baseline by the end of the second year.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Open-label, clinical trial extension: Two-year safety and efficacy results of seladelpar in patients with primary biliary cholangitis. Mayo, MJ., Vierling, JM., Bowlus, CL., et al.[2023]

In a 12-week phase 2 trial involving 41 patients with primary biliary cholangitis who did not respond adequately to ursodeoxycholic acid, seladelpar significantly reduced alkaline phosphatase levels, with a mean decrease of 53% at 50 mg and 63% at 200 mg compared to placebo.

Despite its efficacy in normalizing alkaline phosphatase levels, seladelpar was associated with grade 3 increases in liver enzymes, leading to the early termination of the study, indicating a need for further investigation at lower doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Seladelpar (MBX-8025), a selective PPAR-δ agonist, in patients with primary biliary cholangitis with an inadequate response to ursodeoxycholic acid: a double-blind, randomised, placebo-controlled, phase 2, proof-of-concept study. Jones, D., Boudes, PF., Swain, MG., et al.[2022]

In a 52-week study involving adults with primary biliary cholangitis (PBC), seladelpar showed significant, dose-dependent reductions in alkaline phosphatase (ALP) levels, indicating improved liver function, with reductions of up to 41% at 8 weeks for the highest dose.

Seladelpar was found to be safe and well-tolerated, with no serious treatment-related adverse events reported, and it did not worsen pruritus (itching), a common symptom in PBC patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase II, randomized, open-label, 52-week study of seladelpar in patients with primary biliary cholangitis. Bowlus, CL., Galambos, MR., Aspinall, RJ., et al.[2023]

Seladelpar has shown significant improvements in liver biochemistry and may alleviate symptoms in patients with primary biliary cholangitis (PBC), suggesting it could be an effective treatment option.

Safety concerns regarding liver toxicity associated with seladelpar appear to have been resolved, making it a promising candidate for use as a second-line therapy in PBC compared to existing off-label fibrates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Seladelpar: an investigational drug for the treatment of early-stage primary biliary cholangitis (PBC). Wetten, A., Jones, DEJ., Dyson, JK.[2023]

Citations

1. gilead.com gilead.com/news/news-details/2025/gilead-to-showcase-interim-long-term-efficacy-data-for-livdelzi-seladelpar-in-primary-biliary-cholangitis-pbc-and-investigational-bulevirtide-in-chronic-hepatitis-delta

Gilead to Showcase Interim Long Term Efficacy Data for ... Three late-breaking presentations will share new data on Livdelzi® (seladelpar) in people living with primary biliary cholangitis (PBC), ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12394184/

The Efficacy and Safety of Seladelpar for Primary Biliary ... Seladelpar improved liver biomarkers of cholestasis and reduced pruritus in patients with PBC without significantly increasing the adverse ...

3. livdelzihcp.com livdelzihcp.com/efficacy-pruritus

Efficacy Data | LIVDELZI® (seladelpar) 62% of patients achieved biochemical response with LIVDELZI 1. RESPONSE was a 12-month, randomized, double-blind phase 3 pivotal trial that assessed the ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03301506

Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) An open label long-term study to evaluate the safety and tolerability of seladelpar in subjects with primary biliary cholangitis (PBC).

5. va.gov va.gov/formularyadvisor/DOC_PDF/MON_Seladelpar_LIVDELZI_in_Primary_Biliary_Cholangitis_Monograph_Jan_2025.pdf

Seladelpar (LIVDELZI) in Primary Biliary Cholangitis ... The results suggested that seladelpar has potential for use as a second-line therapy for improving disease activity and symptoms. Long-term Safety and Efficacy.

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39107982/

Evaluating the safety and efficacy of seladelpar for adults ... Expert opinion: Clinical trials results demonstrate the safety and tolerability of seladelpar use for PBC, including in patients with cirrhosis.

7. askgileadmedical.com askgileadmedical.com/docs/conference/EASL%202025_PBC_Gulamhusein_WED-171_Cirrhosis%20PHT_Poster_FINAL@pdf

Safety of Seladelpar in Primary Biliary Cholangitis Patients ... hypertension (PHT), safety outcomes and liver- related adverse events (AEs) were overall similar between seladelpar and placebo. • In this ...

8. journals.lww.com journals.lww.com/ajg/fulltext/9900/long_term_efficacy_and_safety_of_selective_ppar_.1814.aspx

Long-Term Efficacy and Safety of Selective PPARδ Agonist... Seladelpar demonstrated durable improvements in cholestatic biomarkers and pruritus in patients with primary biliary cholangitis with up to 2 years of treatment ...