Pharmacokinetics Of Emulsified Avacopan Applied By NG Tube

Pharmacokinetics Of Emulsified Avacopan In ANCA-associated Vasculitis With Severe Diffuse Alveolar Hemorrhage

The purpose of this study is to determine the 72-hour pharmacokinetics of emulsified avacopan at a dose of 30 mg twice daily given to up to 6 patients with active severe GPA or MPA with diffuse alveolar hemorrhage (DAH) requiring mechanical ventilation for respiratory support.

Study Overview

• Status: Recruiting
• Conditions: Diffuse Alveolar Hemorrhage; Antineutrophil Cytoplasmic Antibody Positive Vasculitis
• Intervention / Treatment: Drug: Avacopan

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Michael Stachowitz; Email: stachowitz.michael@mayo.edu
• Study Contact Backup: Name: Josie Baum; Phone Number: 507-422-9010; Email: baum.josephine@mayo.edu

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic in Rochester
      • Principal Investigator: Ulrich Specks, MD
      • Contact: Josie Baum; Phone Number: 507-422-9010; Email: baum.josephine@mayo.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria

• Fulfillment of the definitions of the Second Chapel Hill Consensus Conference for ANCA-associated vasculitis (either granulomatosis with polyangiitis (GPA) or microscopic polyangiitis MPA).
• Positivity for ANCA, directed against PR3 or MPO.
• Diffuse alveolar hemorrhage.
• Respiratory failure requiring mechanical ventilation.
• Severe newly diagnosed disease or severe relapsing disease. Severe new or relapsing disease is defined as at least one major BVAS/WG item or a score ≥ 3 and the investigator deems standard treatment for severe disease is necessary.
• Minimum BVAS-WG of 3.
• Requirement of standard-of-care remission induction therapy for active severe ANCA-associated vasculitis (GPA or MPA).

Exclusion Criteria

• Diagnosis with eosinophilic granulomatosis with polyangiitis (EGPA, formally Churg-Strauss syndrome) as defined by the Chapel Hill consensus conference.
• Allergies: History of severe allergic reaction to avacopan
• History of documented anti-glomerular basement membrane disease (anti-GBM disease)
• Previous administration of avacopan within the last 5 days.
• Concomitant use of a strong CYP3A4 inhibitor.
• Aspartate aminotransferase [AST], alanine amino transferase [ALT], alkaline phosphatase, or total bilirubin elevation >2.5 times the upper limit of normal (unless attributed to vasculitis) on routine liver function testing obtained within 3 days prior to anticipated treatment with avacopan.
• Evidence of prior active or current Hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency (HIV) infection.
• Active serious infection, including localized infection.
• Pregnancy and breastfeeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Avacopan; Subjects will receive solubilized avacopan at a dose of 30 mg twice daily for 3 consecutive days, applied via NG tube.	Drug: Avacopan; Avacopan capsules will be solubilized in heated water and administered to patients with Diffuse Alveolar Hemorrhage (DAH) due to GPA or MPA via nasogatric tube.; Other Names: Tavneos

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Avacopan blood level over time	The primary objective of this study is to determine the blood (serum) level changes of Avacopan over time (pharmacokinetics) when Avacopan capsules are solubilized in heated water and administered to patients with Diffuse Alveolar Hemorrhage (DAH) due to GPA or MPA via nasogastric tube. Blood will be drawn pre-dose, post-dose 30 minutes, 1-hour, 2-hour, 4-hour, and 6-hour for the first dose and pre-dose for the remaining 72 hours and stored for analysis.	72 hours

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measure in Markers of Neutrophil Activation on Serum Samples	The exploratory objective is to measure changes in markers of neutrophil activation on serum samples (such as neutrophil elastase and/or neutrophil gelatinase-associated lipocalin (NGAL)) collected during the conduct of this study. This will not cause any additional burden to the subject. Samples will be collected from the one research blood draw already being performed per visit.	72 hours

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Ulrich Specks, MD, Mayo Clinic

Publications and helpful links

Helpful Links

• Mayo Clinic Clinical Trials

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2026
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2028

Study Registration Dates

• First Submitted: April 8, 2026
• First Submitted That Met QC Criteria: April 24, 2026
• First Posted (Actual): April 29, 2026

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 24, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: avacopan
• Other Study ID Numbers: 24-009571

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No