CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation for T and B Cell Depletion (CliniMACs)

January 16, 2024 updated by: Nancy Bunin, Children's Hospital of Philadelphia

CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation With the CliniMACs Device for T and B Cell Depletion

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

PRIMARY HYPOTHESIS: T cell depletion utilizing the CliniMACS device will allow more precise, specific and controlled graft engineering of peripheral blood stem cells from unrelated and partially matched related donors without an increase in relapse or graft rejection and grade III or IV acute graft vs. host disease (GVHD).

SECONDARY HYPOTHESIS: Use of the CliniMACS device will allow defined levels of T cell depletion to reflect the risk of severe GVHD in the donor/recipient pair.

Thus, patients with a relatively lower risk of severe GVHD will be assigned to Stratum 1 and receive a graft with lesser T cell depletion and a defined level of reinfused T cells. Patients with higher risk of severe GVHD or for whom there is no perceived clinical benefit of GVHD will be assigned to Stratum 2 and receive a more T cell-depleted graft.

Conditioning of the patient (except immunodeficiencies) includes :

  • Thiotepa 5 mg/kg days for 2 days
  • Cyclophosphamide 60 mg/kg days for 2 days
  • Total body irradiation 200 cGy given twice a day for 3 days

Following conditioning patient's will receive stem cells that have been processed using the CliniMACS device. This processing is done in the stem cell laboratory at The Children's Hospital of Philadelphia. The Stem Cell Lab is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) and maintain complete standard operating procedures (SOP's) and procedure records.

Processing of cells using the CliniMACS will occur in accordance with the Investigator Brochure and Technical Manual following the laboratory SOPs and using aseptic technique. The CHOP Stem Cell Lab has extensive prior experience with automated cell processing technologies, including the CellPro Ceprate device and the Isolex 300i.

Study Type

Interventional

Enrollment (Actual)

93

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 22 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

As of October 2014 this study closed enrollment to malignant diseases. This study remains open to:

Non-malignant diseases:

  1. Bone marrow failure, including severe aplastic anemia
  2. Immunodeficiencies

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 1. CliniMACS CD3+/CD19+ depletion
  • 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
T and B Cell depletion
Other Names:
  • T and B Cell depletion
  • DONOR PERIPHERAL STEM CELL TRANSPLANTATION
Other: 2. CliniMACS CD3+/CD19+ depletion

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
T and B Cell depletion
Other Names:
  • T and B Cell depletion
  • DONOR PERIPHERAL STEM CELL TRANSPLANTATION

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Successful Engraftment
Time Frame: 100 days Post Transplant
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital of Philadelphia

Investigators

  • Principal Investigator: Nancy J Bunin, MD, Children's Hospital of Philadelphia

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2005

Primary Completion (Actual)

January 15, 2017

Study Completion (Actual)

December 30, 2017

Study Registration Dates

First Submitted

December 19, 2007

First Submitted That Met QC Criteria

December 19, 2007

First Posted (Estimated)

December 21, 2007

Study Record Updates

Last Update Posted (Actual)

January 18, 2024

Last Update Submitted That Met QC Criteria

January 16, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 05-004222

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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