NCT00579124

Brief Summary

This is a pilot study with 2 strata to evaluate engraftment and graft vs. host disease (GVHD) in patients receiving unrelated or partially matched related donor peripheral stem cells using the CliniMACS system to positively deplete T cells to prevent severe GVHD. Feasibility will be tested, focusing on engraftment, treatment-related mortality (with a specific focus on interstitial pneumonitis) and severe GVHD.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
93

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Jan 2005

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2005

Completed
3 years until next milestone

First Submitted

Initial submission to the registry

December 19, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 21, 2007

Completed
9.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 15, 2017

Completed
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2017

Completed
6.1 years until next milestone

Results Posted

Study results publicly available

January 18, 2024

Completed
Last Updated

February 3, 2025

Status Verified

January 1, 2025

Enrollment Period

12 years

First QC Date

December 19, 2007

Results QC Date

October 11, 2023

Last Update Submit

January 30, 2025

Conditions

Immunodeficiencies

Keywords

Blood and Marrow TransplantT cell DepletionUnrelatedRelatedDonor

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Successful Engraftment

    Successful engraftment will be whether subjects have achieved an absolute neutrophil count (ANC) \>500 and platelet count \>20 x 109/l by 100 days after transplant

    100 days Post Transplant

Study Arms (2)

1. CliniMACS CD3+/CD19+ depletion

OTHER

* 6/6 or 8/8 matched (fully matched) * 1 antigen or allele mismatched (mismatch at A or B or DRB1) * 2 antigen or allele mismatched (mismatch ONLY at A and B but NOT at DRB1 plus either A or B). Patients will receive grafts that have undergone CD3+ and CD19+ depletion. The CD3(-) fraction will be infused.

Device: CliniMACs

2. CliniMACS CD3+/CD19+ depletion

OTHER

Stratum 2. CliniMACS CD3+/CD19+ depletion: * Haploidentical match * 2 antigen and/or allele mismatched where one of the mismatches includes DRB1 For patients in Stratum 2 we will perform CD3+ (T cell) and CD19+ (B cell) depletion. There will be no T cell add back in this stratum.

Device: CliniMACs

Interventions

CliniMACsDEVICE

T and B Cell depletion

Also known as: T and B Cell depletion, DONOR PERIPHERAL STEM CELL TRANSPLANTATION
1. CliniMACS CD3+/CD19+ depletion2. CliniMACS CD3+/CD19+ depletion

Eligibility Criteria

AgeUp to 22 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • As of October 2014 this study closed enrollment to malignant diseases. This study remains open to:
  • Non-malignant diseases:
  • Bone marrow failure, including severe aplastic anemia
  • Immunodeficiencies

You may not qualify if:

  • \. Patients who have had prior stem cell transplant (SCT) and bone marrow transplant (BMT) are excluded for study enrollment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

  • Seif AE, Li Y, Monos DS, Heidemann SC, Aplenc R, Barrett DM, Casper JT, Freedman JL, Grupp SA, Margolis DA, Olson TS, Teachey DT, Keever-Taylor CA, Wang Y, Talano JM, Bunin NJ. Partially CD3+-Depleted Unrelated and Haploidentical Donor Peripheral Stem Cell Transplantation Has Favorable Graft-versus-Host Disease and Survival Rates in Pediatric Hematologic Malignancy. Biol Blood Marrow Transplant. 2020 Mar;26(3):493-501. doi: 10.1016/j.bbmt.2019.11.022. Epub 2019 Nov 22.

    PMID: 31765697RESULT

Related Links

MeSH Terms

Conditions

Immunologic Deficiency Syndromes

Condition Hierarchy (Ancestors)

Immune System Diseases

Results Point of Contact

Title
Patricia Hankins
Organization
Children's Hospital of Philadelphia
hankinsp@chop.edu
215 590 5168

Study Officials

  • Nancy J Bunin, MD

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
BMT Medical Director

Study Record Dates

First Submitted

December 19, 2007

First Posted

December 21, 2007

Study Start

January 1, 2005

Primary Completion

January 15, 2017

Study Completion

December 30, 2017

Last Updated

February 3, 2025

Results First Posted

January 18, 2024

Record last verified: 2025-01

Locations

US(1)