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VEX-AZA: Treating VEXAS Syndrome with Azacitidine. A Single-Arm, Multicenter Phase II Study on the Efficacy and Safety of Azacitidine in Patients with VEXAS Syndrome
EU CTIS ID: 2024-510715-30-00
What this study is testing
This trial's primary goal is to evaluate the effectiveness of Azacitidine therapy in reducing the UBA1 Variant Allele Frequency (VAF) in patients.
- 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
- Participants must possess one of the currently described UBA1 mutations with a VAF > 10%. The currently described UBA1 mutations are: p.Met41Leu (c.121A.C), p.Met41Val (c.121A.G), p.Met41Thr (c.122T.C), p.Ser56Phe (c.167C>T), p.(splice)(c.118-1G>C) or intronic deletions like (c.118-9_118-2del) affecting the splice site
- Participants must meet one of the following conditions: (1) Present with any degree of cytopenia, or (2) Exhibit inflammatory symptoms of VEXAS with/without cytopenia.
- Participants must be at least 18 years old and possess the cognitive capacity to provide informed consent.
- It is mandatory that male participants, who engage in sex with pre-menopausal (i.e. fertile) women, are willing to use barrier contraceptives (i.e condoms), during treatment with Azacitidine, and until three months after the last treatment. Additionally, it is highly recommended that their fertile partner also uses highly effective contraceptives during and for three months after the participants last treatment. Fertile female participants must exhibit a negative result on a pregnancy test before inclusion, demonstrate a commitment to monthly pregnancy testing and employment of safe contraceptive measures during treatment with Azacitidine, and until three months after the last treatment.
You likely can't join if
- Patients having received Prior therapy with hypomethylating agents (i.e., Azacitidine, Decitabine).
- Patients having received or is planned to receive alloHSCT.
- Patients with active cancer requiring treatment are excluded from participation in this study. Active cancer is defined as a diagnosis of cancer for which the patient is currently receiving treatment, such as chemotherapy, radiation therapy, immunotherapy, targeted therapy, or any other curative or disease-controlling intervention.
- Concomitant treatment with Disease modifying antirheumatic drugs (DMARDs), Chemotherapy/Cytostatic agents, Immunosuppressives, Radiotherapy any novel biological response modifying agents, is not allowed in the trials regardless of the underlying condition for which they are used. These treatments must be discontinued at least 14 days prior to inclusion.
- Patients who are diagnosed with severe comorbidities including decompensated cirrhosis, end-stage kidney disease requiring dialysis, Severe cardiac disease (LVEF <30%), Severe pulmonary disease (FEV1 < 50% of predicted or DLCO < 40%), or a severe immunodeficiency including a diagnosis of HIV. If any of these conditions are suspected but undiagnosed, relevant tests will be ordered prior to screening based on clinical suspicion. However, diagnostic tests for all potential severe comorbidities will not be conducted systematically for every candidate; clinical suspicion will guide the decision to investigate further.
- Patients with Eastern Cooperative Oncology Group (ECOG) Performance Status > 2
See the full eligibility criteria
Who can join
- Participants must possess one of the currently described UBA1 mutations with a VAF > 10%. The currently described UBA1 mutations are: p.Met41Leu (c.121A.C), p.Met41Val (c.121A.G), p.Met41Thr (c.122T.C), p.Ser56Phe (c.167C>T), p.(splice)(c.118-1G>C) or intronic deletions like (c.118-9_118-2del) affecting the splice site
- Participants must meet one of the following conditions: (1) Present with any degree of cytopenia, or (2) Exhibit inflammatory symptoms of VEXAS with/without cytopenia.
- Participants must be at least 18 years old and possess the cognitive capacity to provide informed consent.
- It is mandatory that male participants, who engage in sex with pre-menopausal (i.e. fertile) women, are willing to use barrier contraceptives (i.e condoms), during treatment with Azacitidine, and until three months after the last treatment. Additionally, it is highly recommended that their fertile partner also uses highly effective contraceptives during and for three months after the participants last treatment. Fertile female participants must exhibit a negative result on a pregnancy test before inclusion, demonstrate a commitment to monthly pregnancy testing and employment of safe contraceptive measures during treatment with Azacitidine, and until three months after the last treatment.
What rules you out
- Patients having received Prior therapy with hypomethylating agents (i.e., Azacitidine, Decitabine).
- Patients having received or is planned to receive alloHSCT.
- Patients with active cancer requiring treatment are excluded from participation in this study. Active cancer is defined as a diagnosis of cancer for which the patient is currently receiving treatment, such as chemotherapy, radiation therapy, immunotherapy, targeted therapy, or any other curative or disease-controlling intervention.
- Concomitant treatment with Disease modifying antirheumatic drugs (DMARDs), Chemotherapy/Cytostatic agents, Immunosuppressives, Radiotherapy any novel biological response modifying agents, is not allowed in the trials regardless of the underlying condition for which they are used. These treatments must be discontinued at least 14 days prior to inclusion.
- Patients who are diagnosed with severe comorbidities including decompensated cirrhosis, end-stage kidney disease requiring dialysis, Severe cardiac disease (LVEF <30%), Severe pulmonary disease (FEV1 < 50% of predicted or DLCO < 40%), or a severe immunodeficiency including a diagnosis of HIV. If any of these conditions are suspected but undiagnosed, relevant tests will be ordered prior to screening based on clinical suspicion. However, diagnostic tests for all potential severe comorbidities will not be conducted systematically for every candidate; clinical suspicion will guide the decision to investigate further.
- Patients with Eastern Cooperative Oncology Group (ECOG) Performance Status > 2
- Patients who have a condition where Azacitidine is contraindicated, including hypersensitivity to Azacitidine, advanced malignant hepatic tumors, pregnancy, and breastfeeding individuals.
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, 65+ years, 18-64 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.