A Phase 2a Double Blind, Placebo Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Efficacy of CNP-104 in Subjects Ages 18-75 With Primary Biliary Cholangitis Who Are Unresponsive to UDCA and/or OCA

  • STATUS
    Recruiting
  • End date
    Jun 30, 2024
  • participants needed
    40
  • sponsor
    COUR Pharmaceutical Development Company, Inc.
Updated on 25 October 2022

Summary

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.

Description

Subjects ages 18-75 with primary biliary cholangitis will be screened up to 14 days prior to enrollment into the study. Screening will be completed to assess eligibility, obtain vital signs, collect laboratory samples and PD measurements, and to receive a FibroScan for liver fibrosis. Subjects will additionally complete an initial PBC-40 assessment and begin an Itch Diary, a questionnaire and scoring system to be completed by the patient every morning and evening through Day 120 and then monthly through end of study.

Subjects who meet all inclusion and no exclusion criteria after completing the screening visit will be enrolled in the study. Subjects will be randomized on Day 1 in a 1:1 ratio to receive either CNP-104 or Placebo (0.9% Sodium Chloride USP) by intravenous (IV) infusion. Subjects will be administered CNP-104 or Placebo on Day 1 and on Day 8.

Subjects will remain in the clinic on Day 1 and Day 8 from the time of admission (prior to administration of CNP-104 or Placebo) through the final procedure conducted 4 hours post-dose that same day unless an infusion reaction, or other adverse event, requires an extended duration of monitoring. Subjects will be discharged if safety parameters are acceptable to the investigator.

Seven days after the second administration of CNP-104 or Placebo, subjects must return to the clinic for collection of safety labs, PD measurements, and assessment of AEs and medication changes.

Subjects will continue to be followed for 2 years to assess safety, pharmacodynamics, and immunogenicity during the Post-Dosing period.

Details
Condition Primary Biliary Cholangitis
Treatment Placebo, CNP-104
Clinical Study IdentifierNCT05104853
SponsorCOUR Pharmaceutical Development Company, Inc.
Last Modified on25 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

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
Subjects with ALP > 1.5× ULN
Subjects with AST and ALT < 5× 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

Exclusion Criteria

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 -
)
Subjects with uncontrolled diabetes, as defined by HbA1c > 7%
Subjects who have used biologic agents including anti-cell and anti-cytokine therapies within 12 months prior to screening
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
Note: This criterion does not apply to liver function tests. Additionally
clinically significant laboratory test results at screening that are related
to the condition (PBC) are acceptable as long as all inclusion and no other
exclusion criteria are met
Subjects with positive test results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) antigen/antibody as determined at screening
Subjects with a history of or currently active immune disorders other that PBC (including autoimmune disease) unless the condition, after discussion with the Medical Monitor, has been deemed to be acceptable for the subject's participation in this study
Subjects with a history of or current active diseases requiring immunosuppressive drugs (including azathioprine, prednisone, prednisolone, budesonide, cyclosporine, tacrolimus, methotrexate, or mycophenolate mofetil) unless the condition, after discussion with the Medical Monitor, has been deemed to be acceptable for the subject's participation in this study
Subjects with a clinical history of significant cardiovascular disease as determined by the Investigator
Subjects with a complication or medical history of malignancy within past 5 years which, in the Investigator's opinion, makes the subject unsuitable for study participation
Subjects who, in the Investigator's opinion, will be unable to adhere to study procedures
Subjects who have received an investigational therapy other than CNP-104 within 28 days or 5 half-lives, whichever is longer, prior to screening
Subjects with any condition which, in the Investigator's opinion, makes the subject unsuitable for study participation
Known sensitivity to any components of CNP-104
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