drugset / Trial / NCT07284641
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
NaSingle-groupOpen-labelTreatment
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).
Timeline
- Start
- 2026-05-04
- Primary completion
- 2030-05-04
- Completion
- 2031-02-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Mesenchymal stem cell | Cell therapy | — | — |
| Background | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | — | — |
| Background | Alemtuzumab | Monoclonal antibody | — | — |
| Background | Fludarabine | Small molecule | — | — |
| Background | Melphalan | Small molecule | — | — |
Indications
DiGeorge syndrome
GATA2 deficiency with susceptibility to MDS/AML
Omenn syndrome
STAT3-related early-onset multisystem autoimmune disease
chronic granulomatous disease
combined immunodeficiency due to partial RAG1 deficiency
common variable immunodeficiency
immune dysregulation, autoimmunity, and autoinflammation
immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome
immunodeficiency 88
immunodysregulation with variable immunodeficiency and autoimmunity
recombinase activating gene 2 deficiency