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New treatment option for VEXAS Syndome
Official title Momelotinib in VEXAS Syndrome
ClinicalTrials.gov ID: NCT07098936
What this study is testing
What is Momelotinib treatment?
Momelotinib treatment is an investigational medicine, given as an once-daily, being studied as a potential treatment for vexas syndome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- ECOG (Eastern Cooperative Oncology Group) performance status 0-2 at the time of screening
- Age ≥ 18 years
- Written informed consent
- Diagnosis of VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with UBA1 (Ubiquitin Like Modifier Activating Enzyme 1)...
- Patients with uncontrolled symptoms related to VEXAS with prior treatment line(s) (including steroids)
You likely can't join if
- Patients with MDS (Myelodysplastic syndrome) scheduled for allogeneic stem cell transplant or high risk MDS according to IWG (International Working...
- Patients who are or have been already treated with Janus Kinase (JAK) inhibitors for VEXAS syndrome or another indication
- Patients who are unable to receive a starting daily dose of momelotinib of at least 100 mg
- people with any other active malignancies are not eligible, except for the following: adequately treated basal cell or squamous cell skin cancer, in...
- Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident...
- Known infection with acute and chronic active Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus
See the full eligibility criteria
- ECOG (Eastern Cooperative Oncology Group) performance status 0-2 at the time of screening
- Age ≥ 18 years
- Written informed consent
- Diagnosis of VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with UBA1 (Ubiquitin Like Modifier Activating Enzyme 1) mutation and clinically symptomatic disease requiring immunosuppressive...
- Patients 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/immunomodulatory therapy than glucocorticoids, a wash out period of 28 days is required prior momelotinib onset
- Erythropoietin/luspatercept used as a growth factor treatment is not allowed 28 days prior enrollment
- Adequate liver function (serum transaminases ≤ 3 x ULN (Upper Limits of Normal), Bilirubin ≤ 1.5 x ULN (isolated bilirubin \> 1.5 x ULN is acceptable if bilirubin fractionated and direct bilirubin \< 35%)
- Adequate renal function (creatinine clearance with MDRD (Modification of Diet in Renal Disease) formula \> 30 ml/min)
- Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must:
- Have a negative serum or urine pregnancy test within 24 hours prior to beginning treatment on this study. Lactating patients are excluded.
- 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...
- Agree to learn about the procedures for preservation of egg before starting treatment.
- Male patients must:
- 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...
- Agree to learn about the procedures for preservation of sperm before starting treatment.
- Patients with MDS (Myelodysplastic syndrome) scheduled for allogeneic stem cell transplant or high risk MDS according to IWG (International Working Group) 2023
- Patients who are or have been already treated with Janus Kinase (JAK) inhibitors for VEXAS syndrome or another indication
- Patients who are unable to receive a starting daily dose of momelotinib of at least 100 mg
- people 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...
- 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...
- Known infection with acute and chronic active Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus
- 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Angers, France
- Clermont-Ferrand, France
- Lille, France
- Nantes, France
- Paris, France
- Pessac, France
- Pierre-Bénite, France
- Rennes, France
- Toulouse, France
- Tours, France
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 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Angers, France; Clermont-Ferrand, France; Lille, France; Nantes, France; Paris, France; Pessac, France and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.