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NCT05027945 · ClinicalTrials.gov registry record · Phase 2

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome

A Phase 2 study of Hematopoietic Stem Cell Transplantation and Immunodeficiency, sponsored by National Cancer Institute (NCI).

Recruiting
Registry status
Phase 2
Development phase
54
Enrollment target
1
Study location

NCT05027945 is a Phase 2 study of Hematopoietic Stem Cell Transplantation and Immunodeficiency that is actively recruiting participants, run by National Cancer Institute (NCI). The registered enrollment target is 54 participants, below the 9,788-participant average among 26 other Hematopoietic Stem Cell Transplantation trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT05027945, a Phase 2 study of Hematopoietic Stem Cell Transplantation and Immunodeficiency, is actively recruiting participants, sponsored by National Cancer Institute (NCI).

RECRUITING
Registry status
Phase 2
Development phase
54 participants
Enrollment target
1
Study location

Study Summary

Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers. Researchers want to see if the transplant can treat VEXAS Syndrome. Objective: To see if stem cell transplants can be successfully performed in people with VEXAS and even improve the disease. Eligibility: People ages 18-75 who have VEXAS Syndrome that has caused significant health problems and standard treatment either has not worked or is not available. Design: Participants will be screened with: Physical exam Medical review Blood and urine tests Heart and lung function tests Bone marrow biopsy Participants will have a chest x-ray. They will have an imaging scan of the head, chest, abdomen, pelvis, and sinus. They will have a bone density scan. They will have a dental exam and eye exam. They will meet with specialists. They will repeat some screening tests. Participants will be admitted to the NIH hospital. They have a central venous catheter put into a vein in the chest or neck. They will receive drugs to prepare their bone marrow for the transplant. They may have total body irradiation. They will receive the donor stem cells through the catheter. They will get other drugs to prevent complications and infections. After discharge, they must stay in the DC area for 3 months for weekly study visits. Participants will have study visits 30, 60, 100, 180, 210, 240, 300, and 360 days later. After that, they will have yearly visits for 2 years and then be contacted yearly by phone....

Interventions

  • DRUG Busulfan test dose
  • DRUG Busulfan
  • DRUG Tacrolimus
  • DRUG Mycophenolate mofetil (MMF)
  • PROCEDURE Allogeneic HSCT

Study Locations (1)

Maryland

  • National Institutes of Health Clinical Center - Bethesda

Trial Details

FieldValue
Enrollment Target 54 participants
Start Date 2023-02-23
Est. Completion 2026-07-01
Phase Phase 2

Sponsor

National Cancer Institute (NCI)

3,257 total trials

What the Registry Record Tells You About NCT05027945

The ClinicalTrials.gov registry entry for NCT05027945 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 9,788-participant average among 26 other Hematopoietic Stem Cell Transplantation trials with a reported enrollment target (99% lower). The listed sponsor is National Cancer Institute (NCI), which has 3,257 total studies on file at ClinicalTrials.gov.

The record links to 2 conditions, with Hematopoietic Stem Cell Transplantation appearing as the primary indexed condition, and to 5 interventions - of which Busulfan test dose is the first listed.

NCT05027945 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.

Frequently Asked Questions

What is clinical trial NCT05027945 about?

NCT05027945 is a clinical study titled "A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome". Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers. Researchers want to see if the transplant can treat VEXAS Syndrome. Objective: To see if stem cel...

What is the current status of trial NCT05027945?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 54 participants. The study started on 2023-02-23. Estimated completion is 2026-07-01.

What conditions does trial NCT05027945 study?

This clinical trial studies the following conditions: Hematopoietic Stem Cell Transplantation, Immunodeficiency.

What interventions are being tested in trial NCT05027945?

The interventions under investigation include: Busulfan test dose (DRUG), Busulfan (DRUG), Tacrolimus (DRUG), Mycophenolate mofetil (MMF) (DRUG), Allogeneic HSCT (PROCEDURE).

Who is sponsoring clinical trial NCT05027945?

This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05027945 being conducted?

This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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