Study to Evaluate the Safety, Tolerability, PDs, and Efficacy of CNP-104 in Subjects With Primary Biliary Cholangitis
brief 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.
detailed 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. This study was originally designed with 2 cohorts, Cohort 1 comprised of 6 subjects randomized 1:1 to placebo or 4 mg/kg, and Cohort 2 comprised of up to 34 subjects randomized 1:1 to placebo or 8 mg/kg. Under Protocol Amendment 6 (v7.0), the remaining subjects for Cohort 2 (approximately 16) will be randomized 1:3:1 to placebo, 4 mg/kg, and 8 mg/kg respectively.
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.
official title
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