CNP-104 for Primary Biliary Cholangitis

Recruiting at 21 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

42 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What makes the drug CNP-104 unique for treating primary biliary cholangitis?

CNP-104 is unique because it is being tested specifically for primary biliary cholangitis, a condition with limited treatment options, and it may offer a new approach compared to existing therapies. Unlike other treatments, CNP-104 is being evaluated for its potential to address the underlying disease mechanisms, which could improve quality of life for patients. ^{1 2 3 4 5}

Research Team

Christopher Bowlus, MD

Principal Investigator

UC Davis

Eligibility Criteria

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

I am between 18 and 75 years old and not pregnant.

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

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

Treatment Details

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.

Participant Groups

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+

References

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

Impact of setanaxib on quality of life outcomes in primary biliary cholangitis in a phase 2 randomized controlled trial. [2023]

2. Netherlands pubmed.ncbi.nlm.nih.gov

The prognostic value of antibodies to gp210 among patients with primary biliary cholangitis in Northeast China. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Symptoms and health-related quality of life in Japanese patients with primary biliary cholangitis. [2019]

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

Impact of combination antiretroviral therapy in the NOD.c3c4 mouse model of autoimmune biliary disease. [2021]

5. Pakistan pubmed.ncbi.nlm.nih.gov

New insights into sex differences in primary biliary cholangitis. Gender difference in primary biliary cholangitis. [2022]