Actively Recruiting
Momelotinib in VEXAS Syndrome
Led by Groupe Francophone des Myelodysplasies · Updated on 2025-12-10
57
Participants Needed
11
Research Sites
153 weeks
Total Duration
On this page
Sponsors
G
Groupe Francophone des Myelodysplasies
Lead Sponsor
G
GlaxoSmithKline
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Momelotinib in VEXAS Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- ECOG performance status 0-2 at screening
- Age 18 years or older
- Written informed consent provided
- Diagnosis of VEXAS syndrome with UBA1 mutation and symptomatic disease needing immunosuppressive treatment and at least 10 mg/day glucocorticoids
- Uncontrolled symptoms related to VEXAS despite prior treatments including steroids
- Patients refractory or dependent on steroids
- Only one steroid therapy allowed at inclusion (e.g., prednisone or equivalent)
- 28-day washout required if treated with immunosuppressive or immunomodulatory therapies other than glucocorticoids
- No erythropoietin or luspatercept use 28 days before enrollment
- Adequate liver function (serum transaminases ≤ 3 x ULN, bilirubin ≤ 1.5 x ULN with exceptions)
- Adequate kidney function (creatinine clearance > 30 ml/min)
- Women of childbearing potential must have negative pregnancy test and agree to effective contraception and egg preservation counseling
- Male patients must agree to condom use during treatment and sperm preservation counseling
You will not qualify if you...
- Patients with MDS scheduled for allogeneic stem cell transplant or high-risk MDS
- Prior treatment with Janus Kinase (JAK) inhibitors for VEXAS or other indications
- Unable to start momelotinib at at least 100 mg daily
- Active malignancies except certain treated skin or cervical cancers or cancers disease-free for at least 3 years
- Uncontrolled heart failure, recent heart attack, stroke, or related serious cardiovascular events within 12 weeks
- Known active infection with HIV, Hepatitis B or C
- Medical or psychiatric conditions preventing informed consent
- Active infections requiring therapy
- History of Progressive Multifocal Leukoencephalopathy
- Active gastrointestinal conditions affecting absorption
- Lack of health insurance
- Known allergy to momelotinib or its components
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 11 locations
1
CHU d'Angers - Service des Maladies du sang
Angers, France, 49933
Not Yet Recruiting
2
CHU Estaing - Service d'Hématologie Clinique
Clermont-Ferrand, France, 63000
Not Yet Recruiting
3
Hôpital Claude Huriez - Service de Médecine Interne
Lille, France, 59037
Not Yet Recruiting
4
CHU Nantes - Hôtel Dieu - Service d'Hématologie Clinique
Nantes, France, 44093
Not Yet Recruiting
5
Hôpital Saint Louis - Service hématologie séniors
Paris, France, 75010
Not Yet Recruiting
6
Hôpital Saint-Antoine - Service de Médecine Interne
Paris, France, 75012
Not Yet Recruiting
7
CHU de Haut-Lévèque - Centre F. Magendie - Service des Maladies du sang
Pessac, France, 33604
Not Yet Recruiting
8
CH Lyon sud - Service d'Hématologie Clinique
Pierre-Bénite, France, 69495
Actively Recruiting
9
Hôpital Pontchaillou Service d'hématologie clinique et service de médecine interne
Rennes, France, 35033
Not Yet Recruiting
10
IUCT Oncopole Département d'hématologie / Unité de médecine interne
Toulouse, France, 31059
Not Yet Recruiting
11
CHU de Tours - Hôpital Bretonneau - Service de Médecine Interne
Tours, France, 37044
Not Yet Recruiting
Research Team
M
Maël HEIBLIG, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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