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

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Children's Hospital of Philadelphia
Sponsor:
Information provided by (Responsible Party):
Nancy Bunin, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT00579124
First received: December 19, 2007
Last updated: October 22, 2014
Last verified: October 2014
  Purpose

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 Intervention Phase
Immunodeficiencies
Device: CliniMACs
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:
  • Rates of success of engraftment, day 100 treatment related mortality, acute GVHD, relapse and EBV LPD. Patients who die will be considered failure for the engraftment success evaluation, and relapsed for that endpoint. [ Time Frame: 100 days ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 125
Study Start Date: March 2005
Estimated Study Completion Date: March 2017
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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:
  • CliniMACs
  • T and B Cell depletion
  • DONOR PERIPHERAL STEM CELL TRANSPLANTATION
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:
  • CliniMACs
  • T and B Cell depletion
  • DONOR PERIPHERAL STEM CELL TRANSPLANTATION

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.

  Eligibility

Ages Eligible for Study:   up to 22 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00579124

Contacts
Contact: Margaret T Tartaglione, R.N. 215 590-4029 tartaglione@email.chop.edu
Contact: Patricia Hankins, RN 215 590-5168 hankinsp@email.chop.edu

Locations
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Margaret Tartaglione, RN    215-590-4029    tartaglione@email.chop.edu   
Contact: Patricia Hankins, RN    215-590-5168    hankinsp@email.chop.edu   
Principal Investigator: Nancy J Bunin, MD         
Sub-Investigator: Stephan A Grupp, MD, PhD         
Sponsors and Collaborators
Children's Hospital of Philadelphia
Investigators
Principal Investigator: Nancy J Bunin, MD Children's Hospital of Philadelphia
  More Information

No publications provided

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
Study First Received: December 19, 2007
Last Updated: October 22, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital of Philadelphia:
Blood and Marrow Transplant
T cell Depletion
Unrelated
Related
Donor

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Immune System Diseases

ClinicalTrials.gov processed this record on October 23, 2014