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Stem Cell Transplant for CVID and Other Immune Disorders

This study is looking at a special type of stem cell transplant, called Hematopoietic Stem Cell Transplantation (HSCT), for people with Common Variable Immunodeficiency (CVID) and other immune problems like Primary Immune Regulatory Disorders (PIRD). The transplant uses stem cells from a donor that are a close match to yours. Before the transplant, you would receive a reduced conditioning regimen (treatment to prepare your body) using medicines like alemtuzumab/Campath, anti-thymocyte globulin/rabbit ATG, Fludarabine, Melphalan, and total body irradiation. Researchers want to see how well people survive two years after the transplant. This study is open to males and females between 5 and 40 years old. The study aims to enroll 25 participants, but its current status is unclear.

Study design
This is a single-center, open-label study where all 25 participants will receive the stem cell transplant. It is not randomized, meaning there is no comparison group.
What's involved
After receiving the stem cell transplant, you will be followed for two years according to standard medical practices.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for two years after the stem cell transplant to monitor their survival.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07284641

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

Recruiting
PHASE2Ages 5–40InterventionalTreatment
Paul Szabolcs
~25 participants
Updated 2026-05-15 on ClinicalTrials.gov
What's tested:Hematopoietic stem cell transplant (HSCT)

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Survival post-HSCT
Measured over 2 years post transplant

Conditions

Common Variable Immunodeficiency (CVID)
Primary Immune Regulatory Disorder
Immune Dysregulation
DiGeorge Syndrome
STAT 1 Gain of Function
STAT 3 Gain of Function
Hypomorphic RAG1 Deficiency
CD40 Ligand Deficiency
Mendelian Susceptibility to Mycobacterial Disease
GATA2 Associated Immunodeficiency
CD40 Deficiency
Hypomorphic RAG2 Deficiency
Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome
Omenn Syndrome
Chronic Granulomatous Disease

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Where it's being run

1 sites across 1 states
Pennsylvania1

Study leadership

  • Paul Szabolcs, MD · PRINCIPAL_INVESTIGATOR · UPMC Children's Hospital of Pittsburgh

Who to contact

Shawna A McIntyre, RN
[email protected]
1-412-692-5552
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Common variable immunodeficiency (CVID)
Combined Immunodeficiency (CID)
Immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX syndrome), IPEX like syndromes
Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome
Chronic Granulomatous Disease (CGD)
Signal Transducer and Activator of Transcription (STAT 1) Gain of Function (STAT1 GOF)
Signal Transducer and Activator of Transcription (STAT 3) Gain of Function (STAT3 GOF)
Hypomorphic Recombination-Activating Genes (RAG) 1 and RAG 2
CD40 or CD40L deficiency
Mendelian Susceptibility to Mycobacterial Disease
GATA-binding factor 2 (GATA2) Associated Immunodeficiency
Mouth and Genital Ulcers with Inflamed Cartilage Syndrome (MAGIC) 4. Must have previously failed, due to lack of response or intolerance, mycophenolate mofetil and a B cell-depleting antibody, such as Rituximab 5. Glomerular Filtration Rate (GFR) ≥50 mL/min/1.73 m2 6. Aspartate Aminotransferase (AST) ≤4x upper limit of normal 7. Alanine Aminotransferase (ALT) ≤4x upper limit of normal 8. Direct bilirubin ≤ 2.5 mg/dL 9. Human Immunodeficiency Virus (HIV) negative by serology and PCR 10. Human T-cell Lymphotropic Virus (HTLV) negative by serology 11. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26% 12. Forced Vital Capacity (FVC) and Forced Expiratory Volume in 1 second (FEV1) ≥40% predicted for age 13. Peripheral Capillary Oxygen Saturation (SpO2) of \>92% at rest on room air 14. Subjects must be a minimum of 8 weeks post-solid organ transplant prior to start of conditioning, if applicable 15. Negative pregnancy test for females \>10 years old or who have reached menarche, unless surgically sterilized. 16. All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 12 months after stem cell transplant or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defects. 17. Subject and/or parent guardian informed of the potential risks of infertility following stem cell transplant and advised to discuss sperm banking or oocyte harvesting. 18. Transplant endorsement from clinical immunologist

What this trial measures

  • Survival post-HSCT2 years post transplant

    review of the existing medical records to check on the participant's survival status