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

ConditionsCommon 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
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age5-40
SponsorPaul Szabolcs

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

Patient, parent, or legal guardian must have given written informed consent. For pediatric subjects who are developmentally able, assent or affirmation will be obtained.

Male or female, 5 through 40 years old, inclusive, at the time of informed consent.

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.

Common variable immunodeficiency (CVID)

Disqualifiers

Allergy to Dimethylsulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product

Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, Polymerase chain reaction (PCR) testing, etc.

Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of stem cell transplant

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Hematopoietic stem cell transplant (HSCT)

    Biological/Vaccine

    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.

Treatment groups

25 Participants
are divided into 1 treatment group
Group A: Hematopoietic stem cell transplant (HSCT)Other 1 intervention

Trial outcomes

Primary outcomes

1

Survival post-HSCT

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

Time frame
2 years post transplant

Secondary outcomes

1

engraftment, based upon chimerism data

review of chimerism test results in the existing medical records to check on degree of donor engraftment measured by the percentage of donor-derived blood cells in the HSCT recipient

Time frame
1 month, 2 months, 3 months, 6 months, 12 months, 18 months, 24 months
2

Assess myeloid, B and T cell chimerism

review test results from medical records to check how donor and recipient cells populate different immune lineages post-transplant

Time frame
up to 2 years post translant
3

Assess Immunoglobulin A (IgA), Immunoglobulin (IgM), and Immunoglobulin E (IgE) reconstitution

review of the existing medical records to check on the participant's humoral immune recovery.

Time frame
up to 2 years post transplant
4

independence of immunoglobulin replacement (IVIG, IgG)

Determine and probability

Time frame
1 and 2 year post transplant

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Paul Szabolcs

Lead sponsor

University of Pittsburgh

Sponsor institution

Source ClinicalTrials.gov
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