Actively Recruiting

Early Phase 1
Age: 0 - 30Years
All Genders
NCT02356653

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Led by Children's Hospital of Philadelphia · Updated on 2026-02-20

100

Participants Needed

1

Research Sites

839 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.

CONDITIONS

Official Title

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Who Can Participate

Age: 0 - 30Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients lacking a fully HLA matched sibling donor and candidates for allogeneic hematopoietic stem cell transplant (HSCT) not suitable for other current protocols using CliniMACs for CD3+/CD19+ depletion
  • Patients with transplantable diseases including metabolic storage diseases correctable by HSCT, bone marrow failure syndromes, immunodeficiencies/immune dysregulation syndromes including HLH, hemoglobinopathies requiring HSCT, acute and chronic leukemias, lymphomas, myelodysplastic syndrome, and other non-malignant blood, metabolic, or immune disorders recommended for HSCT
  • Signed informed consent
  • Lansky or Karnofsky performance score of 60 or higher
  • Adequate hematologic and organ function per institutional standards
  • Infectious disease evaluation per institutional standards
  • Negative pregnancy test for participants of childbearing potential
  • Identified living donor meeting regulatory and institutional criteria, including unrelated donors matched 10/10, 9/10, or 8/10 with specific HLA matching requirements, and related donors meeting infectious disease screening and mobilization criteria
  • Emergency cases with graft failure or severe marrow suppression may have some criteria waived
Not Eligible

You will not qualify if you...

  • Uncontrolled bacterial, viral, or fungal infections
  • Availability of a suitable fully HLA matched sibling donor
  • Donor unable to donate peripheral stem cells
  • Pregnant participants

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Trial Site Locations

Total: 1 location

1

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

Loading map...

Research Team

M

Megan Atkinson

CONTACT

P

Patricia Hankins, BSN, RN, CCRC

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here