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

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
Paul Szabolcs
Study ID
NCT07284641
Phase
PHASE2
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
5 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • Hematopoietic stem cell transplant (HSCT) — BIOLOGICAL
    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.

Study Details

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).

Key Dates

Start date
May 4, 2026
Status verified
May 2026
Primary completion
May 4, 2030
Completion
Feb 1, 2031

Study Design

Enrollment
25 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: Hematopoietic 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

Primary Outcome Measure

Survival post-HSCT [ Time Frame: 2 years post transplant ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224
Shawna McIntyre
[email protected]
4126925552
A

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By research site
UPMC Children's Hospital of Pittsburgh· Pittsburgh, PA

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