RecruitingPhase 2NCT07284641

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

Sponsor

Paul Szabolcs

Enrollment

25 participants

Start Date

May 4, 2026

Study Type

INTERVENTIONAL

Conditions

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

Summary

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).

Eligibility

Min Age: 5 YearsMax Age: 40 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called Hematopoietic stem cell transplant (HSCT) for people with cd40 deficiency, cd40 ligand deficiency, and other related conditions. The study is currently recruiting participants at 1 location. People eligible for this study include aged 5 Years to 40 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALHematopoietic stem cell transplant (HSCT)

The participant will receive an allogenic, fully (8 of 8 match) or partially HLA-matched (6-7/8 HLA-matched), stem cell transplant utilizing a conditioning regimen of alemtuzumab/Campath, anti-thymocyte globulin/rabbit ATG, Fludarabine and Melphalan and total body irradiation.

Locations(1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

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NCT07284641

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