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Allogeneic Hematopoietic Stem Cell Transplant
Stem Cell Transplant for VEXAS Syndrome
Phase 2
Recruiting
Led By Dennis D Hickstein, M.D.
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
- Pulmonary symptoms of VEXAS with pulmonary infiltrates, pleural effusion
- Hemoglobin <= 10.0g/dL or red cell transfusion-dependence (at least 4 units of PRBCs in the 8 weeks prior to treatment initiation) or meeting criteria for myeloid neoplasm (MN) by WHO criteria
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day +100 and +1 year post hsct
Awards & highlights
Study Summary
This trial is testing whether stem cell transplants can be used to treat VEXAS syndrome, a disease that causes significant health problems. Eligible participants must be 18-75 years old and have VEXAS syndrome that has not responded to standard treatment. The trial involves several screening tests, hospitalization, and follow-up visits.
Who is the study for?
Adults aged 18-75 with VEXAS Syndrome, significant health issues from the disease, and no success with standard treatments can join. They need a compatible stem cell donor, meet specific health criteria like good heart function and kidney clearance, agree to contraception use for one year post-transplant, and stay near NIH for at least 3 months after.Check my eligibility
What is being tested?
The trial is testing if blood stem cells from a donor can treat VEXAS Syndrome. Participants will undergo physical exams, imaging scans, bone marrow biopsy; receive chemotherapy drugs like Cyclophosphamide and Busulfan; possibly total body irradiation; then get the donor's stem cells through a catheter.See study design
What are the potential side effects?
Possible side effects include reactions to medication such as fatigue or nausea; organ damage due to chemotherapy or radiation; increased risk of infections because of immune suppression by drugs like Sirolimus and Mycophenolate mofetil.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have lung symptoms like fluid in lungs or chest due to VEXAS.
Select...
My hemoglobin is 10.0g/dL or lower, or I've needed at least 4 blood transfusions in the last 8 weeks, or I meet the criteria for a myeloid neoplasm.
Select...
This criterion does not apply to me.
Select...
I have inflammation in my joints, ears, or nose.
Select...
I am between 18 and 75 years old.
Select...
I have symptoms of VEXAS syndrome.
Select...
My platelet count is 75,000 or lower, or I've needed at least 4 platelet transfusions in the last 8 weeks.
Select...
I can care for myself but cannot carry on normal activity or do active work.
Select...
I have a donor who is a close match to my tissue type.
Select...
I do not have inflammation in major organs like the heart, stomach, or eyes.
Select...
My kidney function is within the required range.
Select...
I have skin symptoms like rashes or swelling around the eyes, confirmed by a biopsy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ day +100 and +1 year post hsct
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day +100 and +1 year post hsct
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Reversal of clinical phenotype of VEXAS
Sustained donor engraftment
Secondary outcome measures
Overall survival and event free survival
Safety of allo HSCT
incidence of grade III-IV acute GVHD and moderate to severe chronic GVHD
Side effects data
From 2014 Phase 2 trial • 28 Patients • NCT0135024593%
Fatigue
89%
Diarrhea
86%
Mucositis
86%
Fever
82%
Rash
71%
Tachycardia
68%
Anxiety
68%
Hypotension
64%
Insomnia
64%
Headache
61%
Abdominal pain
54%
Dyspnea
46%
Constipation
46%
Back pain
43%
Hypertension
43%
Dizziness/lightheaded
39%
Nausea
39%
Congestion
39%
Lethargic
39%
Dry mouth
36%
Cough
36%
Itching
36%
Dry skin
36%
Dysuria
32%
Epigastric pain/heartburn
32%
Nocturia
32%
Electrolyte imbalance
29%
Anorexia
29%
Chills
29%
Muscle pain
29%
Chest pain
25%
Neuropathy
25%
Decreased appetite
25%
Rigors
25%
Hyperbilirubinemia
25%
Gastroesophageal reflux disease
25%
Depression
25%
Decreased urine output
25%
Trouble sleeping
25%
Fluid overload
25%
Increased creatinine
21%
Tremors
21%
Hypoxia
21%
Leg pain
21%
Clostridium difficile
21%
Pericardial effusion
18%
Numbness/tingling
18%
Cytomegalovirus reactivation
18%
Hyponatremia
14%
Shoulder pain
14%
CMV reactivation
14%
Hip pain
14%
Blurry vision
14%
Tachypnea
14%
Edema
14%
Ear pain
14%
Arm swelling
14%
Increased LFTs
11%
Hiccups
11%
Arm pain
11%
Sweats
11%
Visual acuity change/weakness
11%
Flatulence
11%
Sore throat
11%
General pain
11%
Dysphagia
11%
Jaw pain
11%
Rhinorrhea
11%
Abdominal cramps
11%
Hypokalemia
11%
Knee pain
11%
Hemorrhoids
11%
Ventricular systolic dysfunction
11%
Pericather thrombus
11%
Photophobia
11%
Urinary incontinence
11%
Neck stiffness
11%
Dry eyes
11%
Feet pain
7%
HHV-6 reactivation
7%
Infection
7%
Urinary frequency
7%
Hematuria
7%
Malaise
7%
Pain at PICC site
7%
Agitation
7%
HHV-6 infection
7%
Rectal pain
7%
Hallucinations
7%
Parasthesia of foot
7%
Erythema
7%
Hand pain
7%
Cold-like symptoms
7%
Flushing/erythema
7%
Vomiting
7%
Hyperpigmentation
7%
Vasovagal syncope
7%
Upper respiratory infection
7%
Wheezing
7%
Heel/ankle pain
7%
Atrial fibrillation
7%
Dry cough
7%
Bloated feeling
7%
Pleural effusion
7%
Retinal hemorrhage
7%
Neck pain
7%
Bradycardia
7%
Orthostatic hypotension
7%
Hyperglycemia
7%
Altered mental status
7%
Hives
7%
Hyperlipidemia
7%
Bone pain
7%
Dehydration
7%
Thrush
4%
Low TSH
4%
Altered taste
4%
Muscle weakness
4%
Mouth ulceration
4%
Joint aches
4%
Hypomagnesemia
4%
Skin irritation
4%
Swollen ankles
4%
Urinary retention
4%
Sinusitis
4%
Night sweats
4%
Eye pain
4%
Paresthesia
4%
Indigestion
4%
Bone marrow cellularity
4%
Colitis
4%
BK cystitis
4%
Subdural hematoma
4%
Brain injury from subdural hematoma
4%
Sepsis
4%
Cholecystitis
4%
Acute infusion reaction
4%
Oral discomfort
4%
Ulcer
4%
Palpitations
4%
Tongue sores
4%
Odynophagia
4%
Alopecia
4%
Clubbing of extremities
4%
Gum pain at site of tooth extraction
4%
Cheek pain
4%
Vision - flashing lights
4%
Mild desquamation on fingers
4%
Knee - decreased range of motion
4%
Plaque on tongue
4%
Decreased sense of well-being
4%
Puffy face
4%
Leg cramping
4%
Perinephric fluid
4%
Head cold
4%
Hearing problems
4%
Diminished breath sounds
4%
BK viremia
4%
Papules
4%
Groin pain
4%
Nose dryness
4%
Burning of soles of hands and feet
4%
Mild distress
4%
Frustration
4%
Meningitis
4%
Orange urine
4%
Seizures
4%
Hypernatremia
4%
Muscle spasms
4%
Abdominal distension
4%
Penile lesion
4%
Polyuria
4%
Aspiration
4%
Internal jugular thrombus
4%
Opisthotonus
4%
Allergic rhinitis
4%
Blisters
4%
Gastroparesis pain
4%
Neutropenic colitis
4%
Ringing in ears
4%
Hypoglycemia
4%
Hypopigmentation
4%
Speech or memory change
4%
Sensitivity to hot/cold
4%
Swollen face
4%
Pulmonary infiltrates
4%
Pain at lung biopsy site
4%
Difficulty walking
4%
Malnutrition
4%
Weight loss
4%
Platelet infusion reaction
4%
Hot flashes
4%
Auditory hallucinations
4%
Elevated ferritin
4%
Tearing of eyes
4%
Pneumonitis
4%
Hypercholesterolemia
4%
Vagina - whitish coating
4%
Discouraged
4%
Blindness
4%
Penile/scrotum pain
4%
Penile blister
4%
Tongue lumps
4%
Nightmares
4%
Parotitis
4%
Difficulty concentrating
4%
Enlarged lymph node
4%
Volume overload
4%
Swollen eyelid
4%
Visual disturbance
4%
Shin pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
TJU 2 Step Regimen
Trial Design
2Treatment groups
Experimental Treatment
Group I: Arm BExperimental Treatment9 Interventions
Reduced intensity regimen (Fludarabine, low dose cyclophosphamide, 200cGY TBI, busulfan)+HSCT+GVHD prophylaxis
Group II: Arm AExperimental Treatment7 Interventions
Reduced intensity regimen (Fludarabine, busulfan)+HSCT+GVHD prophylaxis
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Busulfan
2008
Completed Phase 3
~1120
Total Body Irradiation (TBI)
2016
Completed Phase 3
~1220
Allogeneic HSCT
2015
Completed Phase 2
~60
Sirolimus
2013
Completed Phase 4
~2750
Mycophenolate mofetil (MMF)
2008
Completed Phase 3
~2250
Cyclophosphamide (CY)
2016
Completed Phase 3
~980
Fludarabine
2012
Completed Phase 3
~1100
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,665 Previous Clinical Trials
40,925,817 Total Patients Enrolled
Dennis D Hickstein, M.D.Principal InvestigatorNational Cancer Institute (NCI)
5 Previous Clinical Trials
235 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have a condition or characteristic that is not related to the disease being studied.I have lung symptoms like fluid in lungs or chest due to VEXAS.I do not have any active infections that would make a transplant unsafe.I have a UBA1 mutation or symptoms of VEXAS syndrome.My hemoglobin is 10.0g/dL or lower, or I've needed at least 4 blood transfusions in the last 8 weeks, or I meet the criteria for a myeloid neoplasm.This criterion does not apply to me.I have multiple myeloma.I have inflammation in my joints, ears, or nose.I am between 18 and 75 years old.My condition is related to my disease.I have symptoms of VEXAS syndrome.My platelet count is 75,000 or lower, or I've needed at least 4 platelet transfusions in the last 8 weeks.I've tried standard treatments for my condition without success or cannot tolerate them.I have no allergies to steroids, cyclophosphamide, busulfan, sirolimus, MMF, or G-CSF.I do not have HIV.I can care for myself but cannot carry on normal activity or do active work.I have experienced fevers, fatigue, and weight loss recently.I have a donor who is a close match to my tissue type.I do not have inflammation in major organs like the heart, stomach, or eyes.I can understand and am willing to sign the consent form.My organs are functioning well.My kidney function is within the required range.I have skin symptoms like rashes or swelling around the eyes, confirmed by a biopsy.
Research Study Groups:
This trial has the following groups:- Group 1: Arm A
- Group 2: Arm B
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How perilous is exposure to Total Body Irradiation (TBI) for individuals?
"Based on the available data, our team at Power rated TBI's safety as a 2. This signifies that while there is evidence of its security profile, no studies have indicated efficacy yet."
Answered by AI
Is enrollment open at this point in time for the research project?
"Affirmative. Clinicaltrials.gov verifies that this medical study, initially posted on December 4th 2022 is actively searching for volunteers. An estimated 37 patients across one location are required to complete the trial."
Answered by AI
Could you elaborate on the extant research related to Total Body Irradiation (TBI)?
"Currently, there are 1,053 clinical trials actively researching Total Body Irradiation (TBI), with 185 of them in Phase 3. Philadelphia is the most common location for these studies to be conducted; however, there are 30110 other sites where you can find clinical trials testing TBI treatments."
Answered by AI
Does this investigation allow for senior citizens to sign up?
"This medical trial has a participant age range of 18 to 75, as aligned with the pre-established criteria."
Answered by AI
How many participants is this trial accommodating?
"Affirmative. According to records hosted on clinicaltrials.gov, this medical investigation is currently recruiting participants - a process which has been active since December 4th 2022 and was last updated November 28th 2022. The study requires 37 individuals from 1 distinct location to be recruited in total."
Answered by AI
Am I qualified to partake in this experiment?
"The current clinical trial is seeking 37 individuals suffering from this syndrome, aged between 18 and 75. The key criteria for applicants include meeting the 8/8 or 7/8 HLA-matched related or unrelated donor requirements, a Karnofsky performance status of 40% minimum, non-disease related characteristics, as well as being willing to remain at NIH hospital or within two hours drive thereof for up to 100 days after transplantation (or longer in case of complications), with an adult caregiver present during outpatient period if applicable."
Answered by AI
What medical issues might necessitate the application of Total Body Irradiation (TBI)?
"Total Body Irradiation (TBI) is a therapeutic approach used to treat organ transplantation, multiple sclerosis, leukemia, myelocytic acute and rejection; as well as kidney transplants."
Answered by AI
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