Seladelpar for Primary Biliary Cholangitis

(IDEAL Trial)

Recruiting in Palo Alto (17 mi)

+66 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Gilead Sciences

Must be taking: Ursodeoxycholic acid

Must not be taking: Obeticholic acid, Fibrates

Disqualifiers: Advanced PBC, Other liver diseases, HIV, Alcohol abuse, Cancer, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

To Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects with Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)

Will I have to stop taking my current medications?

The trial requires that you have been on a stable dose of Ursodeoxycholic Acid (UDCA) for more than 3 months or be intolerant to it. You must stop taking obeticholic acid, fibrates, colchicine, methotrexate, azathioprine, long-term systemic corticosteroids, and certain other medications before joining the trial. It's best to discuss your current medications with the study team to see if any need to be stopped.

What data supports the effectiveness of the drug Seladelpar for primary biliary cholangitis?

Research shows that Seladelpar, a drug that helps regulate bile acid levels, improved symptoms like itching and fatigue in patients with primary biliary cholangitis who did not respond well to other treatments. It also showed positive effects on liver function and was generally safe over long-term use.

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Is Seladelpar safe for humans?

Seladelpar has been studied in several clinical trials for primary biliary cholangitis, and these studies have evaluated its safety over periods ranging from 3 months to 2 years. The trials generally found it to be safe, with no major safety concerns reported.

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What makes the drug Seladelpar unique for treating primary biliary cholangitis?

Seladelpar is unique because it is a selective PPAR-δ agonist, which helps regulate bile acid balance and has shown potent anti-cholestatic effects, improving symptoms like itching and fatigue in patients who do not respond well to standard treatment with ursodeoxycholic acid.

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Eligibility Criteria

This trial is for adults aged 18-75 with Primary Biliary Cholangitis (PBC) who haven't fully responded to or can't tolerate Ursodeoxycholic Acid. Participants must have certain levels of alkaline phosphatase, use dual contraception methods if they're of reproductive potential, and not have a history of drug abuse or recent treatments that could interfere with the study.

Inclusion Criteria

I have been on UDCA for the last year or am intolerant to it, with my last dose over 3 months ago.

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

ALP >1×ULN and <1.67×ULN

Exclusion Criteria

Laboratory parameters measured by the Central Laboratory at screening

Clinically important alcohol consumption, defined as more than 2 drink units per day in women and 3 drink units per day in men, or inability to quantify alcohol intake reliably

I haven't taken immunosuppressant drugs in the last 6 months.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either Seladelpar 10 mg or placebo for 52 weeks to assess the normalization of alkaline phosphatase levels

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Participant Groups

The IDEAL trial is testing whether Seladelpar can normalize alkaline phosphatase levels in PBC patients compared to a placebo. It's designed for those who don’t respond well to standard treatment. The participants will be randomly assigned either Seladelpar 10 mg or a placebo.

Group I: Seladelpar 10 mg Experimental Treatment 1 Intervention

Group II: Placebo 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

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Stanford Medicine Outpatient Center Redwood City, CA

University of Colorado Anschultz Medical Campus Aurora, CO

Beth Israel Deconess Medical Center Boston, MA

301 S Fair Oaks Ave Pasadena, CA

More Trial Locations

Who Is Running the Clinical Trial?

Gilead Sciences Lead Sponsor

CymaBay Therapeutics, Inc. Lead Sponsor

References

1. Netherlands 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. [2022] Many patients with primary biliary cholangitis have an inadequate response to first-line therapy with ursodeoxycholic acid. Seladelpar is a potent, selective agonist for the peroxisome proliferator-activated receptor-delta (PPAR-δ), which is implicated in bile acid homoeostasis. This first-in-class study evaluated the anti-cholestatic effects and safety of seladelpar in patients with an inadequate response to ursodeoxycholic acid.

2. United States pubmed.ncbi.nlm.nih.gov

Seladelpar improved measures of pruritus, sleep, and fatigue and decreased serum bile acids in patients with primary biliary cholangitis. [2022] Primary biliary cholangitis (PBC) can result in life-altering cholestatic pruritus and fatigue, but treatment options are limited. Seladelpar, a peroxisome proliferator-activated receptor-delta (PPARδ) agonist, has demonstrated potent anti-cholestatic effects in clinical studies. This open-label, uncontrolled phase 2 study in PBC patients evaluated the effects of 1-year of seladelpar treatment on measures of pruritus and quality of life.

3. United States 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. [2023] ENHANCE was a phase 3 study that evaluated efficacy and safety of seladelpar, a selective peroxisome proliferator-activated receptor-δ (PPAR) agonist, versus placebo in patients with primary biliary cholangitis with inadequate response or intolerance to ursodeoxycholic acid (UDCA).

4. Netherlands pubmed.ncbi.nlm.nih.gov

A phase II, randomized, open-label, 52-week study of seladelpar in patients with primary biliary cholangitis. [2023] We examined the efficacy and safety of seladelpar, a selective peroxisome proliferator-activated receptor-delta agonist, in adults with primary biliary cholangitis (PBC) at risk of disease progression (alkaline phosphatase [ALP] ≥1.67xupper limit of normal [ULN]) who were receiving or intolerant to ursodeoxycholic acid.

5. England pubmed.ncbi.nlm.nih.gov

Open-label, clinical trial extension: Two-year safety and efficacy results of seladelpar in patients with primary biliary cholangitis. [2023] Seladelpar is a potent and selective peroxisome proliferator-activated receptor-δ agonist that targets multiple cell types involved in primary biliary cholangitis (PBC), leading to anti-cholestatic, anti-inflammatory and anti-pruritic effects.

6. England pubmed.ncbi.nlm.nih.gov

Seladelpar: an investigational drug for the treatment of early-stage primary biliary cholangitis (PBC). [2023] Generic fibrates are used off-label as add-in therapy for the management of primary biliary cholangitis (PBC) but with unproven long-term liver-related survival benefits. The recently developed fibrate, seladelpar, has shown promising results in clinical trials, but these outcomes have been previously marred by safety concerns.