Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) for Bone Marrow Failure Syndrome

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Children's Hospital of Philadelphia, Philadelphia, PABone Marrow Failure Syndrome+5 MoreTransplant of stem cells with CD3+/CD19+ depletion (CliniMACs) - Device
Eligibility
< 65
All Sexes
What conditions do you have?
Select

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.

Eligible Conditions
  • Bone Marrow Failure Syndrome
  • Immunodeficiency
  • IPEX Syndrome
  • Inborn Errors of Metabolism
  • Leukemia

Treatment Effectiveness

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: 1 year

1 year
Graft versus Host Disease
Overall Survival
100 days
Graft Failure

Trial Safety

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

Expanded access to CliniMACs device for T cell depletion
Device
Experimental Group · 1 Intervention: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) · Intervention Types: Device

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor
661 Previous Clinical Trials
7,855,386 Total Patients Enrolled
Nancy J Bunin, MDPrincipal InvestigatorChildren's Hospital of Philadelphia
3 Previous Clinical Trials
245 Total Patients Enrolled

Eligibility Criteria

Age < 65 · All Participants · 2 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You cannot participate if you have certain diseases that can be treated with a transplant, such as certain types of leukemia or immune system disorders. Your organ function must also meet certain criteria, including good performance status, normal kidney and liver function, and a healthy heart. You must also not have any active untreated infections and have a negative pregnancy test if you are a woman of childbearing age.

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

Unverified Answer

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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.