Diffuse Alveolar HemorrhagePhase 1NCT07556484
Pharmacokinetics Of Emulsified Avacopan Applied By NG Tube
A Phase 1 study testing Avacopan in people with Diffuse Alveolar Hemorrhage and Antineutrophil Cytoplasmic Antibody Positive Vasculitis, run by Mayo Clinic at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diffuse Alveolar Hemorrhage, Antineutrophil Cytoplasmic Antibody Positive Vasculitis
- What is being tested?
- Avacopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2027to 1 Aug 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Michael Stachowitz contact
United States1 site, 1 recruiting
- Recruiting
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Josie Baum, 507-422-9010, baum.josephine@mayo.edu
Ulrich Specks, MD
About this study
Official title: 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.
- Being tested
- Avacopan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvacopanDrug
Also called Tavneos
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.
| Study group | Role | What they receive |
|---|---|---|
| Avacopan | Experimental | Subjects will receive solubilized avacopan at a dose of 30 mg twice daily for 3 consecutive days, applied via NG tube. Receives: Drug: Avacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 72 hours
Secondary outcomes (1)
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.
Measured: 72 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2026
- Study start1 Aug 2027 (estimated)
- Primary completion1 Aug 2028 (estimated)
- Study completion1 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diffuse Alveolar HemorrhageStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ulrich Specks, MD, Mayo Clinic
- Investigators
- Ulrich Specks, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07556484
- Sponsor's protocol ID
- 24-009571
- Status last verified
- Oct 2026
- First submitted
- 8 Apr 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No