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TAVNEOS for Otolaryngologic Manifestations of Granulomatosis With Polyangiitis

ConditionsGranulomatosis With PolyangiitisWegener'sGPA
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorRobert Spiera, MD

About this trial

This is a single center double-blind placebo-controlled study. Patients with GPA and active ears, nose, and throat (ENT) disease in at least two ENT domains, as defined after endoscopic visualization of the upper airway and audiometric evaluation, if applicable, by a single otolaryngologist using a validated GPA ENT disease activity score, will be eligible for inclusion. Patients will be treated with standard of care (SOC) treatment as determined by their treating rheumatologist. In addition to SOC, patients will be randomized to receive TAVNEOS 30mg BID or placebo. Patients will be followed for 52 weeks with standardized ENT assessment along with rheumatologic evaluation of overall disease activity with BVAS.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

GPA diagnosis defined by score of ≥5 on 2022 ACR/EULAR Classification Criteria for GPA

Bloody rhinorrhea (Daily blood stained nasal discharge)

Objective stridor (Stridor assessed by doctor)

Inflammation on nasal examination (Ulcers, granulation, friable mucosa on rigid nasal endoscopy. Excluding crusting)

Disqualifiers

Creatinine >4.0mg/dl or GFR <15 at baseline or dependence on dialysis

Respiratory failure requiring mechanical ventilatory support or had experienced alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study

Previous treatment with TAVNEOS within 6 months of screening

Inability to comply with study and/or follow-up procedures at investigator discretion.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Avacopan

    Drug

    BID dose of 30 mg TAVNEOS (3-10mg capsules)

  • Placebo

    Drug

    BID dose of 30mg TAVNEOS-matching placebo (3-10mg capsules)

Treatment groups

30 Participants
are divided into 2 treatment groups
Group A: TAVNEOSActive comparator 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Proportion of patients in ENT remission without relapse

The proportion of patients in ENT remission without relapse in each treatment group at week 52 with no glucocorticoid (GC) exposure 4 weeks prior to week 52, where ENT remission is defined as a score of 0 on GPA ENT disease activity score

Time frame
Week 52

Secondary outcomes

1

Mean ENT disease activity score

Comparison of mean ENT disease activity scores between treatment groups at week 52

Time frame
Week 52
2

Change in ENT disease activity score

Change in ENT GPA disease activity score between week 26 and week 52

Time frame
Between week 26 and week 52
3

Proportion of patients in ENT remission with BVAS 0 at week 52 and sustained remission

Proportion of patients in remission with BVAS 0 at week 52 and sustained remission from week 26 to week 52 with BVAS 0 and no glucocorticoids for 4 weeks prior to assessment

Time frame
Week 52
4

Cumulative steroid dose

Time frame
Through study completion, an average of 60 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Robert Spiera, MD

Lead sponsor

Hospital for Special Surgery, New York

Sponsor institution

Amgen

Collaborator

Source ClinicalTrials.gov
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