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Momelotinib in VEXAS Syndrome

NCT07098936 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 57

Last updated 2025-12-10

No results posted yet for this study

Summary

Multicenter, phase II trial with safety run-in to evaluate the efficacy and safety of momelotinib in patients with VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with or without associated myelodysplastic syndrome (MDS).

The study will consist of two consecutive steps, a dose-finding safety run-in and a single-arm prospective phase II.

During safety run-in phase, three fixed dose levels will be tested according to a 3+3 design, using cohorts of size 3 in order to establish the maximum tolerated dose.

After this safety run-in phase, patients included in phase II will be treated with momelotinib at the maximum tolerated dose preliminary fixed.

Patients included in the phase II will receive momelotinib continuously until disease progression or loss of response, at physician's discretion.

All patients included in the study will receive glucocorticoids (prednisone/prednisolone equivalent) at baseline (at least \> 10mg/day).

Response assessment regarding VEXAS related symptoms will be evaluated after 4, 12, 24 and 48 weeks. Response assessment regarding MDS features will be evaluated at 12 and 24 weeks.

Conditions

  • VEXAS Syndome
  • Myelo Dysplastic Syndrome

Interventions

DRUG

Momelotinib treatment

Patients included will receive momelotinib continuously until disease progression or loss of response, at physician's discretion.

Sponsors & Collaborators

  • GlaxoSmithKline

    collaborator INDUSTRY

  • Groupe Francophone des Myelodysplasies

    lead OTHER

Principal Investigators

  • Maël HEIBLIG, MD · CH Lyon Sud

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-11-25
Primary Completion
2028-06-30
Completion
2028-11-30

Countries

  • France

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07098936 on ClinicalTrials.gov