NCT06253117

Evaluation of Pirfenidone as a Novel Therapeutic Strategy Against Recurrent Acute Pancreatitis.

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: University of Alabama at Birmingham · Academic / Other
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

This clinical will evaluate the safety, tolerability and early efficacy of pirenidone in patients with recurrent acute pancreatitis.

Detailed description

Background: Recurrent Acute Pancreatitis (RAP) is occurrence of 2 or more distinct episodes of Acute Pancreatitis (AP) (separated by at least 3 months). RAP not only leads to significant morbidity and reduced quality of life; patients with RAP have a substantial (up to 40%) risk of progressing to chronic pancreatitis (CP). Unfortunately, there is no therapy, which can help prevent future attacks of pancreatitis in RAP patients. In this regard, our published work suggest that pirfenidone, a therapy approved by FDA for the treatment of Idiopathic Pulmonary Fibrosis (IPF), has the potential to emerge as a novel treatment for patients with RAP. Briefly, our results suggest that: 1) Pirfenidone, when administered prophylactically, reduces the risk of AP development; 2) Pirfenidone, when given to experimental animals with severe AP, leads to amelioration of local and systemic injury; and 3) Pirfenidone, when administered to experimental animals with ongoing RAP, reduces risk of progression to CP. Thus, our results suggest that pirfenidone has potential to emerge as novel therapeutic strategy for RAP patients. These studies have high translational value as pirfenidone is already in clinical use for IPF and has over 8 years of record of safety. Hypothesis: "Pirfenidone treatment in patients with RAP will be safe, tolerable, and efficacious." Objectives: The study has following primary and secondary objectives. Primary Objective: 1) To evaluate the safety and tolerability of pirfenidone compared to placebo, in patients with RAP. 2) To evaluate the efficacy of pirfenidone in reducing the laboratory markers of inflammation. Secondary Objective: 1) To evaluate the efficacy of pirfenidone in reducing- a) recurrence of AP; b) pancreatitis related emergency room visits and readmissions; c) severity of pancreatitis, if acute pancreatitis was to develop; d) Improving quality of life measures; e) Improvement in patient reported outcomes. 2) To develop a predictive biomarker of efficacy of pirfenidone, which can be incorporated in a future clinical trial. Specific aims and study design. Pilot Clinical Trial of the Safety, Tolerability and Efficacy of Pirfenidone in RAP. We will conduct a randomized, double-blind, placebo controlled pilot trial of pirfenidone in patients with RAP. RAP patients, 18-85 years of age who meet the eligibility criteria, will be recruited at one of the three participating sites (UAB, Mayo clinic Rochester and Brigham and Women's Hospital, Boston), and randomly assigned to pirfenidone or placebo treatment for 6 months. The primary endpoints of this clinical trial are a) feasibility; and b) safety. We have multiple efficacy secondary endpoints endpoint like cumulative incidence and rate of recurrent attacks of AP, severity of recurrent attacks of AP (mild/moderate/severe), readmissions and/or ER visits for pain, changes in quality of life as measured by PANQOLI and SF-12 health survey questionnaires, and changes in changes in patient reported outcome as measured by PAN-PROMISE score. We plan to recruit 60 patients at the three study sites over the duration of this clinical trial.

Conditions

• Recurrent Acute Pancreatitis

Interventions

Type	Name	Description
DRUG	Pirfenidone	Pirfenidone Days 1-7: 267 mg PO TID (801 mg/day) Days 8-14: 534 mg PO TID (1602 mg/day) Day 15 and thereafter: 801 mg PO TID; not to exceed 2403 mg/day Duration of treatment- total 6 months
DRUG	Placebo	Placebo

Timeline

• Start date: 2024-06-27
• Primary completion: 2028-09-01
• Completion: 2028-09-01
• First posted: 2024-02-12
• Last updated: 2025-10-14

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study