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

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ClinicalTrials.gov Identifier: NCT00579124
Recruitment Status : Active, not recruiting
First Posted : December 21, 2007
Last Update Posted : November 15, 2016
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Immunodeficiencies Device: CliniMACs Phase 2

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 Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 85 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation With the CliniMACs Device for T and B Cell Depletion
Study Start Date : March 2005
Estimated Primary Completion Date : March 2018
Estimated Study Completion Date : March 2018
Arms and Interventions

Arm Intervention/treatment
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
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

Outcome Measures

Primary Outcome Measures :
  1. Determine rate of 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

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 22 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00579124

United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
Principal Investigator: Nancy J Bunin, MD Children's Hospital of Philadelphia
More Information

Responsible Party: Nancy Bunin, BMT Medical Director, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00579124     History of Changes
Other Study ID Numbers: 2005-3-4222
First Posted: December 21, 2007    Key Record Dates
Last Update Posted: November 15, 2016
Last Verified: November 2016

Keywords provided by Nancy Bunin, Children's Hospital of Philadelphia:
Blood and Marrow Transplant
T cell Depletion

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Immune System Diseases