CNP-104 for Primary Biliary Cholangitis

Not currently recruiting at 24 trial locations

Overseen By Jerry A Staser

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: COUR Pharmaceutical Development Company, Inc.

Must be taking: UDCA, OCA

Must not be taking: Methotrexate, Immunotherapy, Steroids, others

Disqualifiers: Liver diseases, Kidney disease, Diabetes, others

Compensation: Varies

Number of Visits: 3

Duration: 2 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CNP-104 for individuals with primary biliary cholangitis (PBC), a liver disease that can cause liver damage over time. The trial aims to assess the safety and effectiveness of CNP-104 at different doses compared to a placebo. Participants must have a confirmed PBC diagnosis and find current treatments insufficient. Those struggling with PBC symptoms despite taking medications like UDCA or OCA might find this trial suitable. As a Phase 1, Phase 2 trial, the research focuses on understanding how CNP-104 works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial requires that participants stay on a stable dose of any medication used to treat the symptoms of Primary Biliary Cholangitis (PBC) for at least 3 months before joining and not change the dose until Day 60 of the study, unless approved by the medical team. If you are taking other medications, it's best to discuss with the study team to see if any changes are needed.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research is examining the safety of CNP-104 for treating primary biliary cholangitis (PBC), a liver condition. Although detailed safety information from human studies remains limited, the FDA has granted CNP-104 Orphan Drug Designation, indicating its potential for treating PBC. As this trial marks the first human testing of CNP-104, it is designed to closely monitor any side effects or reactions. Ensuring safety is a top priority, and researchers aim to assess how well participants tolerate different doses of CNP-104. Participants in this trial will receive close monitoring to ensure their safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CNP-104 for Primary Biliary Cholangitis (PBC) because it offers a novel approach compared to the current standard treatments like ursodeoxycholic acid (UDCA) and obeticholic acid (OCA). Unlike these treatments, which primarily work by modifying bile acid composition or reducing liver inflammation, CNP-104 is designed to directly target the underlying autoimmune processes of PBC. This potential to address the root cause of the disease could mean more effective management and possibly slower progression of PBC. Additionally, CNP-104 is administered intravenously, which might ensure precise dosing and potentially quicker action.

What evidence suggests that CNP-104 might be an effective treatment for Primary Biliary Cholangitis?

Research has shown that CNP-104 might effectively treat primary biliary cholangitis (PBC). This trial will test different dosages of CNP-104, with some participants receiving 4 mg/Kg and others 8 mg/Kg, while a separate group receives a placebo. Studies suggest that CNP-104 could halt disease progression, offering hope as a treatment that alters the disease's course. Early results over 120 days have shown promise in reducing symptoms and improving liver function. The FDA has granted it orphan drug status, recognizing its potential benefits for PBC patients. Overall, these findings suggest CNP-104 could become an important new option for people living with this condition. ^{2 6 7 8 9}

Who Is on the Research Team?

Christopher Bowlus, MD

Principal Investigator

UC Davis

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have been on a stable dose of my PBC medication for at least 3 months.

Subjects who are willing and able to provide Institutional Review Board (IRB) approved written informed consent and privacy language as per national regulations

Subjects must meet specific laboratory criteria including ALP, AST, ALT, hemoglobin, and total bilirubin levels

Exclusion Criteria

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

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

I have had cancer within the last 5 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

1 visit (in-person)

Treatment

Participants receive CNP-104 or Placebo by intravenous infusion on Day 1 and Day 8

2 weeks

2 visits (in-person)

Post-Dosing Follow-up

Participants are monitored for safety, pharmacodynamics, and immunogenicity

2 years

Regular visits (in-person and virtual)

Long-term Follow-up

Participants continue to be monitored for safety and durability of response

20 months

What Are the Treatments Tested in This Trial?

Interventions

• CNP-104
• Placebo

Trial Overview 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.

How Is the Trial Designed?

Group I: 8 mg/Kg CNP-104 Experimental Treatment 1 Intervention

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

Group II: 4 mg/Kg CNP-104 Experimental Treatment 1 Intervention

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

Group III: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

COUR Pharmaceutical Development Company, Inc.

Lead Sponsor

Trials

7

Recruited

250+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05104853

NCT05104853 | Study to Evaluate the Safety, Tolerability, ... Study to Evaluate the Safety, Tolerability, PDs, and Efficacy of CNP-104 in Subjects with Primary Biliary Cholangitis. ClinicalTrials.gov ID NCT05104853.

2. pharmacytimes.com pharmacytimes.com/view/fda-grants-orphan-drug-designation-to-cnp-104-for-primary-biliary-cholangitis

FDA Grants Orphan Drug Designation to CNP-104 for ... The FDA granted orphan drug designation to CNP-104 (COUR Pharmaceuticals) for the treatment of patients with primary biliary cholangitis (PBC).

3. hcplive.com hcplive.com/view/positive-phase-2a-data-suggest-disease-modifying-potential-cnp-104-pbc

Positive Phase 2a Data Suggest Disease-Modifying ... Study findings suggest CNP-104 may be poised to become the first disease-modifying treatment for PBC, with results through 120 days highlighting its potential ...

4. lumiraventures.com lumiraventures.com/news/cour-pharmaceuticals-announces-positive-top-line-results-from-phase-2a-study-of-cnp-104-in-primary-biliary-cholangitis

COUR Pharmaceuticals Announces Positive Top-line ... These data suggest that CNP-104 has the potential to halt disease progression which would be a transformational advancement for people living ...

5. mayo.edu mayo.edu/research/clinical-trials/diseases-conditions/primary-biliary-cholangitis

Primary Biliary Cholangitis Clinical Trials The purpose of this study is to assess the safety and tolerability of CNP-104, and to assess the change in Serum Alkaline Phosphatase (ALP) levels among ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05104853

NCT05104853 | Study to Evaluate the Safety, Tolerability, ... Study to Evaluate the Safety, Tolerability, PDs, and Efficacy of CNP-104 in Subjects with Primary Biliary Cholangitis. ClinicalTrials.gov ID NCT05104853.

7. hcplive.com hcplive.com/view/fda-grants-orphan-drug-designation-cnp-104-primary-biliary-cholangitis

FDA Grants Orphan Drug Designation to CNP-104 for ... CNP-104 received Orphan Drug Designation for PBC, highlighting its potential as a disease-modifying treatment by targeting pathogenic T-cells.

8. mayo.edu mayo.edu/research/clinical-trials/cls-20538041

Study to Evaluate the Safety, Tolerability, PDs, and Efficacy ... The purpose of this study is to assess the safety and tolerability of CNP-104, and to assess the change in Serum Alkaline Phosphatase (ALP) ...

9. courpharma.com courpharma.com/pipeline/

Pipeline | COUR Pharma CNP-104 for primary biliary cholangitis (PBC). PBC is a chronic autoimmune disease that targets the bile ducts in the liver, leading to progressive bile duct ...