A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)

Part of paid clinical trials in Scottsdale, Arizona.

Sponsor
Swedish Orphan Biovitrum
Study ID
NCT06782373
Phase
PHASE2
Status
Active Not Recruiting

Conditions

  • VEXAS
  • VEXAS Syndrome

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Pacritinib — DRUG
    Supplied in hard capsules.
  • Placebo — DRUG
    Supplied in hard capsules.

Study Details

This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. In phase 2a, approximately 78 participants will be randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. In Phase 2b, approximately 78 participants will be randomized. Phase 2b participants will initially be randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. After dose selection, Phase 2b further participants will be randomized to either the selected pacritinib dose or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.

Key Dates

First listed
Jan 17, 2025
Start date
May 28, 2025
Status verified
Sep 2026
Primary completion
Dec 30, 2026
Completion
May 22, 2030

Study Design

Enrollment
156 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Pacritinib
    To receive oral administration of pacritinib dose A for up to 24 weeks.
  • Experimental: Pacritinib + placebo
    To receive oral administration of pacritinib dose B plus placebo for up to 24 weeks
  • Placebo Comparator: Placebo
    To receive oral administration of placebo for up to 24 weeks.

Primary Outcome Measure

Overall Response Rate (ORR), defined as achieving Partial Clinical Response or better at any time during the double-blind treatment period. [ Time Frame: up to End of Week 24 ]

Locations (9)

FacilityCityStateZIPSite coordinators
Mayo Clinic - ScottsdaleScottsdaleArizona85259-
Dana Farber Cancer InstituteBostonMassachusetts02215-
Mayo Clinic - RochesterRochesterMinnesota55905-
NYU Langone HealthNew YorkNew York10016-
Cleveland Clinic - ClevelandClevelandOhio44195-
The James Cancer Hospital and Solove Research InstituteColumbusOhio43210-
UT MD Anderson Cancer CenterHoustonTexas77030-
University of Utah HealthcareSalt Lake CityUtah84132-
Fred Hutchinson Cancer CenterSeattleWashington98109-

Find similar trials in Scottsdale, AZ

By research site
Mayo Clinic - Scottsdale· Scottsdale, AZDana Farber Cancer Institute· Boston, MAMayo Clinic - Rochester· Rochester, MNNYU Langone Health· New York, NYCleveland Clinic - Cleveland· Cleveland, OHThe James Cancer Hospital and Solove Research Institute· Columbus, OH

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