MedTrace Logo

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

NCT07284641 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2026-05-15

No results posted yet for this study

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).

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

Interventions

BIOLOGICAL

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.

Sponsors & Collaborators

  • Paul Szabolcs

    lead OTHER

Principal Investigators

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-04
Primary Completion
2030-05-04
Completion
2031-02-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07284641 on ClinicalTrials.gov