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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
Must not have
History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan, sirolimus, MMF, G-CSF) used in the study
HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day +100 and +1 year post hsct
Awards & highlights
No Placebo-Only Group

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.
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.
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 below
Select...
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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have no allergies to steroids, cyclophosphamide, busulfan, sirolimus, MMF, or G-CSF.
Select...
I do not have HIV.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day +100 and +1 year post hsct
This trial's timeline: 3 weeks for screening, Varies for treatment, and day +100 and +1 year post hsct for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Reversal of clinical phenotype of VEXAS
Sustained donor engraftment
Secondary study objectives
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 • NCT01350245
93%
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%
Hip pain
14%
CMV reactivation
14%
Blurry vision
14%
Tachypnea
14%
Edema
14%
Ear pain
14%
Arm swelling
14%
Increased LFTs
11%
Arm pain
11%
Hiccups
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%
Hematuria
7%
Urinary frequency
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%
Muscle weakness
4%
Mouth ulceration
4%
Altered taste
4%
Low TSH
4%
Hypomagnesemia
4%
Joint aches
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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
Tacrolimus
2019
Completed Phase 4
~5510
Total Body Irradiation (TBI)
2016
Completed Phase 3
~1220
Mycophenolate mofetil (MMF)
2008
Completed Phase 3
~2250
Busulfan
2008
Completed Phase 4
~1710
Allogeneic HSCT
2015
Completed Phase 2
~60
Cyclophosphamide (CY)
2016
Completed Phase 3
~980
Fludarabine
2012
Completed Phase 4
~1830

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,958 Previous Clinical Trials
41,112,526 Total Patients Enrolled
Dennis D Hickstein, M.D.Principal InvestigatorNational Cancer Institute (NCI)
5 Previous Clinical Trials
236 Total Patients Enrolled

Media Library

Allogeneic HSCT (Allogeneic Hematopoietic Stem Cell Transplant) Clinical Trial Eligibility Overview. Trial Name: NCT05027945 — Phase 2
Bone Marrow Transplant Research Study Groups: Arm A, Arm B
Bone Marrow Transplant Clinical Trial 2023: Allogeneic HSCT Highlights & Side Effects. Trial Name: NCT05027945 — Phase 2
Allogeneic HSCT (Allogeneic Hematopoietic Stem Cell Transplant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05027945 — Phase 2
~23 spots leftby Jul 2026
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