Study to Evaluate the Safety, Tolerability, PDs, and Efficacy of CNP-104 in Subjects With Primary Biliary Cholangitis

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Primary Biliary Cholangitis

In brief

This study is a Phase 2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-104. The study consists of a 120 day primary study followed by a 20 month long-term safety and durability of response follow-up period.

Key facts

Study ID: NCT05104853
Run by: COUR Pharmaceutical Development Company, Inc.
People needed: 42
Starts: 2022-01-25
Expected to finish: 2026-01-30
Last updated by the study team: 2026-03-10

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Subjects who are willing and able to provide Institutional Review Board (IRB) approved written informed consent and privacy language as per national regulations.
Men and non-pregnant women, ages 18-75 years inclusive.
Subjects with a PBC diagnosis as demonstrated by the presence of 2 or more of the following 3 diagnostic factors:
Alkaline phosphatase > 1.5× ULN for at least 6 months
Positive AMA titer or, if AMA negative or in low titer (<1:40), positive PBC-specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components [PDC-E2, 2-oxo-glutaric acid dehydrogenase complex])
Liver biopsy findings consistent with PBC
Subjects who are unresponsive to UDCA and/or OCA after 6 months of treatment at a stable dose as measured by ALP > 1.5× ULN.
For subjects on any medication used to treat the symptoms of PBC (ex. UDCA, OCA, seladelpar), subjects must be on a stable dose for a minimum of 3 months prior to enrollment and must agree not to change their dose through study Day 60 unless reviewed by the medical monitor and approved by the site investigator.
Subjects with ALP > 1.5× ULN.
Subjects with AST and ALT < 5× ULN.
Subjects with hemoglobin ≥ 10 g/dL.
Subjects with total bilirubin < 2× ULN.
Men and women of child-bearing potential (WOCBP) must agree to practice a highly effective method of contraception that may include, but is not limited to, abstinence, sex only with persons of the same sex, monogamous relationship with vasectomized partner, vasectomy, hysterectomy, bilateral tubal ligation, licensed hormonal methods, intrauterine device (IUD) beginning at the time of screening through Day 90.
Female subjects who agree not to donate ova starting at initial screening and through Day 90.
Male subjects who agree to not donate sperm starting at screening and through Day 90.

You may not qualify if…

Subjects with a Class B or Class C Child-Pugh score.
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.
Subjects who have previously undergone liver transplantation.
Subjects with decompensated liver disease as defined by the presence or history of any of the following:
MELD score > 15
Hepatic encephalopathy
Ascites
Hepatorenal syndrome or serum creatinine > 2 mg/dL
Total Bilirubin > 3.0 mg/dL
INR >1.8 unless on anticoagulation such as Coumadin
History of variceal hemorrhage
Subjects with a history of cerebrovascular accident in the past 12 months.
Subjects with history of myocardial infarction, as defined by any of the following criteria:
Development of pathological Q waves with or without symptoms
Imaging evidence of a region of loss of viable myocardium that is thinned and fails to contract, in the absence of a non-ischemic cause
Pathological findings of a healed or healing myocardial
Subjects with chronic kidney disease, as defined by estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 for at least 3 months (per CKD EPI Equation - 2021).
Subjects with uncontrolled diabetes, as defined by HbA1c > 7%.
Subjects who have used the following medications:
Methotrexate within 90 days of screening.
Immunotherapy drugs unless approved by the medical monitor.
Subjects with a history of tuberculosis or positive PPD skin test.
Subjects who have received administration of any live vaccine (other than intranasal Influenza) within 28 days or subunit vaccine within 14 days prior to screening or are planning to receive any vaccination before Day 90.
Subjects who have used systemic steroids within 3 months prior to screening.
Subjects with laboratory test results at screening or prior to study dosing that are outside the normal limits and considered by the Investigator to be clinically significant.

Where it is running

Southern California Research Center — Coronado, California, United States
OM Research — Lancaster, California, United States
University of California Davis Health — Sacramento, California, United States
Peak Gastroenterology Associates — Colorado Springs, Colorado, United States
Yale School of Medicine — New Haven, Connecticut, United States
University of Florida - Hepatology Research — Gainesville, Florida, United States
Mayo Clinic Florida — Jacksonville, Florida, United States
Florida Research Institute — Lakewood Rch, Florida, United States
GI PROS Research — Naples, Florida, United States
Cleveland Clinic - Florida — Weston, Florida, United States
Digestive Healthcare of Georgia — Atlanta, Georgia, United States
University of Chicago Medical Center — Chicago, Illinois, United States
Massachusetts General Hospital — Boston, Massachusetts, United States
Henry Ford Health System — Novi, Michigan, United States
Washington University School of Medicine in St. Louis — St Louis, Missouri, United States
Montefiore Medical Center — The Bronx, New York, United States
Duke University Medical Center — Durham, North Carolina, United States
Texas Liver Institute — San Antonio, Texas, United States
University of Virginia — Charlottesville, Virginia, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.