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Momelotinib in VEXAS syndrome
EU CTIS ID: 2024-519779-24-00
What this study is testing
Safety run-in :To determine the maximum tolerated dose (MTD) of momelotinib (MMB) in VEXAS patients Phase II : To determine clinical efficacy of MMB at 24 weeks on VEXAS related symptoms in UBA1 mutated and symptomatic patients refractory/dependent to steroids
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- ECOG performance status 0-2 at the time of screening
- Adequate liver function (serum transaminases ≤ 3N, Bilirubin ≤ 1.5 x ULN (isolated bilirubin > 1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin < 35%))
- Adequate renal function (creatinine clearance with MDRD formula > 30 ml/min)
- Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must: 1- Have a negative serum or urine pregnancy test within 24-hours prior to beginning treatment on this study. Lactating patients are excluded. 2- Agree to use, and to be able to comply with, effective contraception without interruption, 4 weeks before starting study drug throughout the entire duration study drug therapy (including doses interruptions) and for 12 weeks after the end of the study drug therapy. 3- Agree to learn about the procedures for preservation of egg before starting treatment.
- Male patients must: 1- Agree the need for the use of a condom if engaged in sexual activity with a woman of childbearing potential during the entire period of treatment, even if disruption of treatment and during 12 weeks after end of treatment. 2- Agree to learn about the procedures for preservation of sperm before starting treatment.
- Age ≥ 18 years
You likely can't join if
- Patients with MDS currently scheduled for allogeneic stem cell transplant or high risk MDS according to IWG 2023.
- Active gastrointestinal conditions that may affect absorption
- No affiliation to a health insurance system.
- Known hypersensitivity to the study investigational medicinal product, the metabolites, or formulation excipients.
- Patients who are or have been already treated with JAK inhibitors for VEXAS syndrome or another indication.
- Patients who are unable to receive a starting daily dose of MMB of at least 100 mg.
See the full eligibility criteria
Who can join
- ECOG performance status 0-2 at the time of screening
- Adequate liver function (serum transaminases ≤ 3N, Bilirubin ≤ 1.5 x ULN (isolated bilirubin > 1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin < 35%))
- Adequate renal function (creatinine clearance with MDRD formula > 30 ml/min)
- Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must: 1- Have a negative serum or urine pregnancy test within 24-hours prior to beginning treatment on this study. Lactating patients are excluded. 2- Agree to use, and to be able to comply with, effective contraception without interruption, 4 weeks before starting study drug throughout the entire duration study drug therapy (including doses interruptions) and for 12 weeks after the end of the study drug therapy. 3- Agree to learn about the procedures for preservation of egg before starting treatment.
- Male patients must: 1- Agree the need for the use of a condom if engaged in sexual activity with a woman of childbearing potential during the entire period of treatment, even if disruption of treatment and during 12 weeks after end of treatment. 2- Agree to learn about the procedures for preservation of sperm before starting treatment.
- Age ≥ 18 years
- Written informed consent
- Diagnosis of VEXAS syndrome with UBA1 mutation and clinically symptomatic disease requiring immunosuppressive treatment and at least 10 mg/d of glucocorticoids
- With uncontrolled symptoms related to VEXAS with prior treatment line(s) (including steroids)
- Patients refractory/dependent to steroids
- Single concomitant steroids therapy (e.g. prednisone or equivalent) at the time of inclusion is allowed
- For patients treated with other immunosuppressive/immuno-modulatory therapy than glucocorticoids, a wash out period of 28 days is required prior MMB onset
- Erythropoietin/luspatercept used as a growth factor is not allowed 28 days prior enrollment
What rules you out
- Patients with MDS currently scheduled for allogeneic stem cell transplant or high risk MDS according to IWG 2023.
- Active gastrointestinal conditions that may affect absorption
- No affiliation to a health insurance system.
- Known hypersensitivity to the study investigational medicinal product, the metabolites, or formulation excipients.
- Patients who are or have been already treated with JAK inhibitors for VEXAS syndrome or another indication.
- Patients who are unable to receive a starting daily dose of MMB of at least 100 mg.
- Subjects with any other active malignancies are not eligible, except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which subject has been disease-free for at least 3 years.
- Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism within 12 weeks prior to initiation of MMB.
- Known infection with acute and chronic active HIV, HBV, HCV infections.
- Any medical or psychiatric condition not allowing the informed consent of the subject
- Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy
- Previous history of Progressive Multifocal Leuko-encephalopathy (PML)
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.