Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) for Bone Marrow Failure Syndrome
Study Summary
This trial is for children with a serious disease who are not eligible for other stem cell transplant protocols. The goal is to expand access for patients who lack a fully HLA matched sibling donor.
- Bone Marrow Failure Syndrome
- Immunodeficiency
- IPEX Syndrome
- Inborn Errors of Metabolism
- Leukemia
Treatment Effectiveness
Effectiveness Progress
Study Objectives
1 Primary · 2 Secondary · Reporting Duration: 1 year
Trial Safety
Safety Progress
Trial Design
1 Treatment Group
Expanded access to CliniMACs device for T cell depletion
1 of 1
Experimental Treatment
100 Total Participants · 1 Treatment Group
Primary Treatment: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) · No Placebo Group · Phase < 1
Trial Logistics
Trial Timeline
Who is running the clinical trial?
Eligibility Criteria
Age < 65 · All Participants · 2 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:Frequently Asked Questions
What is the capacity for enrolling participants into this research endeavor?
"Indeed, the clinicaltrials.gov portal confirms that this trial is presently recruiting participants. Originally posted on December 1st 2013 and last updated November 8th 2022, a total of 100 patients are needed from one site to complete the study." - Anonymous Online Contributor
Are new participants accepted into this research endeavor?
"Affirmative. The data hosted by clinicaltrials.gov demonstrates that this experiment is presently recruiting participants, which was first posted on December 1st 2013 and updated most recently November 8th 2022. A hundred subjects are required from a single site for the trial to be successful." - Anonymous Online Contributor