Actively Recruiting
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
Led by Paul Szabolcs · Updated on 2026-05-06
25
Participants Needed
1
Research Sites
246 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD).
CONDITIONS
Official Title
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient, parent, or legal guardian has given written informed consent; assent obtained if appropriate.
- Male or female aged 5 through 40 years at consent.
- Evidence of CVID or autoimmune manifestation of PIRD confirmed by genetic screening.
- Examples include CVID, CID, IPEX syndrome, Omenn syndrome, DiGeorge syndrome, CGD, STAT1/STAT3 Gain of Function, hypomorphic RAG1/RAG2 deficiencies, CD40 or CD40L deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 associated immunodeficiency, MAGIC syndrome.
- Previously failed or intolerant to mycophenolate mofetil and a B cell-depleting antibody like Rituximab.
- Glomerular filtration rate ≥ 50 mL/min/1.73 m2.
- Aspartate aminotransferase ≤ 4 times upper limit of normal.
- Alanine aminotransferase ≤ 4 times upper limit of normal.
- Direct bilirubin ≤ 2.5 mg/dL.
- HIV negative by serology and PCR.
- HTLV negative by serology.
- Cardiac ejection fraction ≥ 40% or shortening fraction ≥ 26%.
- Forced vital capacity and forced expiratory volume in 1 second ≥ 40% predicted for age.
- Peripheral capillary oxygen saturation > 92% at rest on room air.
- At least 8 weeks post solid organ transplant before conditioning start, if applicable.
- Negative pregnancy test for females > 10 years old or post-menarche unless surgically sterilized.
- Females of childbearing potential and sexually active males agree to use FDA-approved birth control for 12 months post-transplant or during harmful medication use.
- Informed about potential infertility risks and advised on sperm banking or oocyte harvesting.
- Transplant endorsement from clinical immunologist.
You will not qualify if you...
- Allergy to Dimethylsulfoxide (DMSO) or any ingredient used in stem cell product manufacturing.
- Uncontrolled systemic infection as confirmed by tests such as blood cultures or PCR.
- Receipt of any licensed or investigational live attenuated vaccine within 4 weeks before stem cell transplant.
- Past or current medical problems or findings that the investigator believes pose additional risks, interfere with study compliance, or impact data quality or interpretation.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
Research Team
S
Shawna A McIntyre, RN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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