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New treatment option for Immunodeficiency
Official title A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
ClinicalTrials.gov ID: NCT05027945
What this study is testing
What is Busulfan test dose?
Busulfan test dose is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for immunodeficiency.
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
- Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers.
- 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 to 75
You may be able to join if
- Non-disease related
- Age \>= 18-year-old and \<= 75-year-old
- Availability of an 8/8 or 7/8 HLA-matched related or unrelated donor, or a haploidentical related donor
- Karnofsky performance status of \>= 40%
- Adequate end-organ function, defined as follow:
You likely can't join if
- HCT Comorbidity Index \>= 5. Note: Comorbidities that are specifically addressed in the inclusion criteria will not be included in the calculation of...
- Participants with multiple myeloma. Note: participants with low risk smoldering multiple myeloma or monoclonal gammopathy of unknown significance...
- Participants who are receiving any other investigational agents within the last 30 days before treatment initiation.
- HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan...
- Pregnant individuals are excluded from this study because the study agents have the potential for teratogenic or abortifacient effects. Because there...
See the full eligibility criteria
- Non-disease related
- Age \>= 18-year-old and \<= 75-year-old
- Availability of an 8/8 or 7/8 HLA-matched related or unrelated donor, or a haploidentical related donor
- Karnofsky performance status of \>= 40%
- Adequate end-organ function, defined as follow:
- Left ventricular ejection fraction \> 35%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to treatment initiation.
- Creatinine \ = 30 ml/min;
- Serum conjugated bilirubin \< 3.0 mg/dl; serum ALT and AST \<= 5 times upper limit of normal.
- Pulmonary function tests: FEV1 and DLCO \>30%
- Ability of subject to understand and the willingness to sign a written informed consent document.
- As therapeutic agents used in this trial may be harmful to a fetus, individuals of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) at the...
- Willingness to remain in the NIH hospital or, if discharged, live within 2 hours drive from the NIH, for a minimum of 100 days after transplant or longer, if there are complications. If outpatient in the first 100 days...
- Somatic mutation in UBA1 performed by a CLIA or CAP certified laboratory. NOTE: Participants without a mutation or unknown mutation status may be eligible if they have a clinical history that is characteristic of an...
- Inflammatory clinical phenotype for VEXAS syndrome with at least one VEXAS disease manifestation below:
- constitutional symptoms including fevers, fatigue, and weight loss
- cutaneous symptoms of VEXAS including biopsy proven neutrophilic dermatosis, cutaneous vasculitis, periorbital inflammation
- pulmonary symptoms of VEXAS with pulmonary infiltrates, pleural effusion
- musculoskeletal or cartilaginous involvement including inflammatory arthritis, ear chondritis, and nasal chondritis
- inflammatory disease in other major organ systems including cardiac, gastrointestinal, ocular, etc.
- Presence of cytopenia defined as at least one of the following: i. Absolute neutrophil count \ = 0.5mg/kg per day of prednisone for the above listed inflammatory condition or intolerance or refractory to use of...
- HCT Comorbidity Index \>= 5. Note: Comorbidities that are specifically addressed in the inclusion criteria will not be included in the calculation of HCT-CI score.
- Participants with multiple myeloma. Note: participants with low risk smoldering multiple myeloma or monoclonal gammopathy of unknown significance will not be excluded)
- Participants who are receiving any other investigational agents within the last 30 days before treatment initiation.
- HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan, tacrolimus, MMF, filgrastim or filgrastim biosimilar) used in the study.
- Pregnant individuals are excluded from this study because the study agents have the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing...
- Uncontrolled intercurrent illness or social situations (as determined by a licensed master social worker) that would limit compliance with study requirements.
- Presence of active uncontrolled infections that in the opinion of the PI would make it unsafe to proceed with transplantation.
- Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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 to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. 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.