What side effects are associated with this type of intervention?

Common treatments for Primary Biliary Cirrhosis (PBC) include D-penicillamine, which has been associated with increased mortality and substantial heterogeneity in outcomes. Statins, used for managing hypercholesterolemia in PBC patients, can lead to toxicity in those with significant cholestasis and may cause elevated liver enzymes. Treatments for pruritus, such as albumin dialysis, are generally well-tolerated but can have procedural risks. Overall, side effects vary by treatment and patient condition, necessitating careful monitoring and individualized therapy.

What prior FDA approvals exist?

The primary FDA-approved treatment for Primary Biliary Cirrhosis (PBC) is ursodeoxycholic acid (UDCA), which has been shown to improve liver function and delay disease progression. Another approved treatment is obeticholic acid, which is used in patients who do not respond adequately to UDCA. These treatments focus on improving bile flow and reducing liver inflammation. The trial CNP-104 is exploring new therapeutic options, although its specific mechanism of action is not detailed in the provided context.

What other types of research exist?

Promising alternatives to CNP-104 for Primary Biliary Cirrhosis patients include: 1) GPC3-targeted and curcumin-loaded phospholipid microbubbles (GPC3-CUR-MBs) for their superior anti-liver cancer effects in vitro and in vivo, and 2) cyclic RGD-decorated nanoparticles for efficient drug delivery to liver cells. Both therapies demonstrate targeted action and potential for liver-specific treatments.

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CNP-104 for Primary Biliary Cholangitis

Phase 1 & 2

Waitlist Available

Led By Christopher Bowlus, MD

Research Sponsored by COUR Pharmaceutical Development Company, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects with a PBC diagnosis as demonstrated by specific diagnostic factors

Female subjects who agree not to donate ova

Must not have

Subjects with immune disorders or diseases requiring immunosuppressive drugs

Subjects with concomitant liver diseases including chronic viral hepatitis B or C, autoimmune hepatitis, PSC, alcoholic liver disease, Wilson's disease, hemochromatosis, or Gilbert's syndrome

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through visit 7, an average of 90 days and visit 16, an average of 720 days

Summary

This trial tests a new drug, CNP-104, in adults with a liver disease called primary biliary cholangitis. The drug is given through a vein to check its safety and effectiveness. The study will monitor patients for a period of time.

Who is the study for?

Adults aged 18-75 with Primary Biliary Cholangitis (PBC) unresponsive to standard treatments, having specific diagnostic factors like high alkaline phosphatase or positive PBC-specific antibodies. Participants must not be pregnant, agree to use effective contraception, and have liver function within certain limits. Excludes those with other liver diseases, history of organ transplant or severe cardiovascular events.

What is being tested?

The trial is testing CNP-104's safety and effectiveness in treating PBC compared to a placebo over a period of about two years. It involves multiple doses of the drug and monitors how well it works and its long-term effects on patients who haven't responded well to existing treatments.

What are the potential side effects?

While the side effects for CNP-104 are not detailed here, common risks may include allergic reactions to components of the drug, potential impact on liver function given the patient population, and general medication-related issues such as nausea or fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with primary biliary cholangitis.

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I agree not to donate eggs during the trial.

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I agree not to donate sperm during the trial.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have an immune disorder or take drugs that weaken my immune system.

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I have a liver condition such as hepatitis, Wilson's disease, or Gilbert's syndrome.

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I have had cancer within the last 5 years.

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I have had a heart attack before.

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My liver function is moderately to severely impaired.

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My liver is not functioning properly.

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I can follow the study's required procedures.

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I have a serious heart condition.

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My diabetes is not under control.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through visit 7, an average of 90 days and visit 16, an average of 720 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through visit 7, an average of 90 days and visit 16, an average of 720 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and through visit 7, an average of 90 days and visit 16, an average of 720 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Laboratory safety assessments (hematology, serum chemistry, coagulation panel, urinalysis).

Serum Cytokines (TNF-α, IL-4, IL-6, IL-10, IL-1β, MCP-1, MIP-1α, IFN-γ)

Secondary study objectives

To assess the change from baseline in AMA

To assess the change from baseline in Serum Alkaline Phosphatase (ALP) levels, for safety only

To assess the change from baseline in Weekly Mean Itch Score

+4 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: 8 mg/Kg CNP-104Experimental Treatment1 Intervention

200 mL intravenous infusion on Day 1 and Day 8: 8 mg/Kg CNP-104

Group II: 4 mg/Kg CNP-104Experimental Treatment1 Intervention

200 mL intravenous infusion on Day 1 and Day 8: 4 mg/Kg CNP-104

Group III: PlaceboPlacebo Group1 Intervention

200 mL intravenous infusion on Day 1 and Day 8: Placebo

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Primary Biliary Cirrhosis (PBC) include ursodeoxycholic acid (UDCA) and obeticholic acid (OCA). UDCA works by reducing the concentration of toxic bile acids in the liver, thereby decreasing liver inflammation and damage. OCA, a farnesoid X receptor (FXR) agonist, helps to regulate bile acid synthesis and transport, reducing liver inflammation and fibrosis. These treatments are crucial for PBC patients as they help to slow disease progression, improve liver function, and alleviate symptoms such as pruritus. While the specific mechanism of CNP-104 is not detailed, it is likely aimed at modulating immune responses or bile acid metabolism, similar to existing therapies, to provide therapeutic benefits in PBC.