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

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

NCT07284641•Paul Szabolcs

View on ClinicalTrials.gov

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).

Detailed Description

Hematopoietic stem cell transplant (HSCT) with reduced-intensity conditioning has been demonstrated as the best definitive therapy to correct many of these inheritable immune defects (Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD). This is a single center, open label, non-randomized, Phase II study in which subjects receive an allogenic, fully (8 of 8 match) or partially Human Leukocyte Antigen (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 (TBI). Graft sources include bone marrow or mobilized peripheral blood stem cells from either a related or unrelated donor. After stem cell infusion, subjects are followed for 2 years per standard of care practices.

Eligibility

Age

5 - 40 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Patient, parent, or legal guardian must have given written informed consent. For pediatric subjects who are developmentally able, assent or affirmation will be obtained.
•2. Male or female, 5 through 40 years old, inclusive, at the time of informed consent.
•3. Patients must have evidence of common variable immunodeficiency (CVID) or other autoimmune manifestation of a primary immune regulatory disorder (PIRD). Genetic screening is required by a targeting gene panel to determine presence of genetic variations that may lead to inborn errors of immunity.
•Examples of such diseases include, but are not limited to:
•* 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)
+21 more criteria

Exclusion Criteria

•1. Allergy to Dimethylsulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product
•2. Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, Polymerase chain reaction (PCR) testing, etc.
•3. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of stem cell transplant
•4. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the subject's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.

Locations (1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Key Dates

Start Date

March 1, 2026

Primary Completion Date

February 1, 2030

Completion Date

February 1, 2031

Last Updated

February 20, 2026

Enrollment

ESTIMATED participants

Conditions

Common Variable Immunodeficiency (CVID)Primary Immune Regulatory DisorderImmune DysregulationDiGeorge SyndromeSTAT 1 Gain of FunctionSTAT 3 Gain of FunctionHypomorphic RAG1 DeficiencyCD40 Ligand DeficiencyMendelian Susceptibility to Mycobacterial DiseaseGATA2 Associated ImmunodeficiencyCD40 DeficiencyHypomorphic RAG2 DeficiencyImmune Dysregulation Polyendocrinopathy Enteropathy X-Linked SyndromeOmenn SyndromeChronic Granulomatous Disease

Interventions

Hematopoietic stem cell transplant (HSCT)

BIOLOGICAL

Contacts

Shawna A McIntyre, RN

CONTACT

1-412-692-5552 mcintyresm@upmc.edu

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 20, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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