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LeukemiaNCT03145545

Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

A study testing Apha/beta T and CD19+ cell depletion using CliniMACS device in people with Leukemia and Bone Marrow Failure Syndrome, run by Children's Hospital of Philadelphia at 1 site in United States.

Available

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

The primary objective 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). Read more about this study

Who is it for?
Ages 1 month and older
What is being tested?
Apha/beta T and CD19+ cell depletion using CliniMACS device
Type of treatment
Medical device
How many people
Not given
Where
1 siteUnited States
Study dates
Start not givento not given
Phase
Expanded access
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 month and older
  • All sexes
  • Child, Adult, Older adult

Who may be able to join

  • Signed, informed consent
  • Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
  • Patients who do not meet criteria for current open, institutional protocols using CliniMACs device for β T/CD19+ depletion
  • Patients with the following transplantable diseases:
  • Non-malignant diseases
  • Metabolic storage diseases correctable by HSCT
Show all 31Show fewer
  • Bone marrow failure syndromes
  • Immunodeficiencies/immune dysregulation syndromes
  • Sickle cell disease or thalassemia
  • Other diseases treated with HSCT
  • o Malignant diseases
  • Acute leukemias
  • Chronic leukemias
  • Lymphomas
  • Myelodyplastic syndrome
  • Inclusion Criteria: Primary transplant or non-emergent subsequent transplant
  • The conditioning prescribed to the patient will be determined based on the disease and organ status and will include agents that are standard. Appropriate combinations of chemotherapy, immunotherapy and/or radiation will be determined on an individual basis. Patient eligibility will be assessed as per our current institutional standard operating procedures. Patients that meet the following criteria may be eligible:
  • Lansky or Karnofsky performance ≥ 60
  • Hematologic and Organ Function as per current institutional SOP
  • Infectious Evaluation as per current institutional SOP
  • Inclusion Criteria: Urgent subsequent transplant, with conditioning, in the setting of graft failure or severe marrow dysfunction
  • For subjects undergoing an urgent subsequent transplant, conditioning will be individualized based on the underlying disease, prior transplant history, and current organ function. Subjects with the following may be eligible:
  • Adequate organ function as determined by the transplant physician to safely tolerate the planned conditioning regimen
  • No active, uncontrolled infection
  • Patients whose initial transplant was performed under this protocol, under another clinical protocol, or using a bone marrow graft
  • Previously collected TCRαβ/CD19 depleted stem cells from this or another approved protocol may be used if available.
  • Inclusion Criteria: Unconditioned Stem Cell Boost/Transplant These subjects will not be required to meet the performance status, hematologic and organ function, or infectious evaluation criteria listed above, as conditioning will not be administered.
  • Patients with cytopenias due to bone marrow dysfunction, declining donor chimerism, or other treatment needs as determined by the subject's clinical course
  • Patients with active infections are eligible, as the stem cell boost will be administered without conditioning
  • Previously collected TCRαβ/CD19 depleted stem cells from this or another approved protocol may be used if available.
  • Patients who received their initial transplant either on this protocol or on a separate protocol

Who cannot join

  • Suitable and available HLA matched sibling donor. However, patients with fully matched related donors (including siblings) may be eligible in special circumstances where use of an unmanipulated bone marrow graft has increased risks for donor or recipient.
  • Donor unable to donate peripheral stem cells
  • Pregnant participants
  • Donor Eligibility Patients must have an identified living donor
  • Donor selection will comply with 21 CFR 1271*
  • Unrelated donor that meets the matching criteria of the NMDP with allele matching at HLA -A, -B, -C, -DRB1, and -DQB1: Unrelated donors may be a 10/10 match, a 9/10 match, or an 8/10 match if one of the mismatches is at DQB1.
Show all 13Show fewer
  • Related donor mismatched at one to five HLA alleles (haploidentical)
  • Matched related donor may be considered suitable donors for this protocol if a peripheral stem cell donation is deemed by the clinical team to be a safer donation option (if donor is not a suitable candidate for a bone marrow harvest) or if there is concern that bone marrow harvest would not yield adequate cell doses Additionally, a matched related donor would be considered suitable for this protocol if there are safety concerns regarding a patient receiving a standard T cell replete bone marrow transplant due to individualized GVHD risk or risk related to standard GVHD prevention medications.
  • Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, HepB, HepC PCR negative.
  • We assess donor eligibility as per our Allogenic Donor Evaluation for Eligibility standard operating procedure. These procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is FACT accredited.
  • Related donors will be consented and enrolled under IRB approved research protocol for cell collection per IRB 04-004078 CHP 784 Clinical and Research Collection and Future Research Use of Bone Marrow, Stem Cells or T Cells.
  • Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis.
  • The donors selected for this IDE will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors, NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases

Trial locations and contacts

1 site in 1 country.

Megan Atkinson contact

215-590-2820

cttsbmtintake@chop.edu

Patricia Hankins, BSN, RN contact

215-590-5168

hankinsp@chop.edu

United States1 site

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Megan Atkinson, 215-590-2820, cttsbmtintake@chop.edu

    Patricia Hankins, BSN, RN, 215-590-5168, hankinsp@chop.edu

    Available

Other trials in United States

About this study

Official title: Expanded Access Protocol Using TCR Alpha/Beta T Cell/CD19+ Depleted Donor Peripheral Stem Cells

The primary objective 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 Institutional Review Board (IRB) approved protocols that utilize CliniMACs technology for T depletion.

Read the detailed description

Only 25-30% of patients who may benefit from HSCT have a matched related donor. An unrelated cord blood may not be available due to size or matching criteria, or if a reduced intensity regiment is recommended. The risk of severe graft vs. host disease (GVHD) and other complications is higher with unrelated donors, or partially matched related donors. At the Children's Hospital of Philadelphia (CHOP) there is extensive experience using mismatched unrelated donors or partially matched related donors with complete or partial T depletion to reduce the risk of severe GVHD.

Being tested
Apha/beta T and CD19+ cell depletion using CliniMACS device
Medical subject headings
Leukemia, Bone Marrow Failure Disorders, Immunologic Deficiency Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Apha/beta T and CD19+ cell depletion using CliniMACS deviceDevice

    Stem cells will be processed using the CliniMACS device for alpha/beta and CD19+ T cell depletion. Processing of cells using the CliniMACS will occur in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 May 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Expanded access
IDs, dates and publications
ClinicalTrials.gov ID
NCT03145545
Sponsor's protocol ID
16-013527
Status last verified
Jul 2026
First submitted
5 May 2017
Last updated
8 Jul 2026
Results posted
No

Publications

  • Beatty PG, Anasetti C, Hansen JA, Longton GM, Sanders JE, Martin PJ, Mickelson EM, Choo SY, Petersdorf EW, Pepe MS, et al. Marrow transplantation from unrelated donors for treatment of hematologic malignancies: effect of mismatching for one HLA locus. Blood. 1993 Jan 1;81(1):249-53.

    PubMed 8417795

  • Speiser DE, Tiercy JM, Rufer N, Grundschober C, Gratwohl A, Chapuis B, Helg C, Loliger CC, Siren MK, Roosnek E, Jeannet M. High resolution HLA matching associated with decreased mortality after unrelated bone marrow transplantation. Blood. 1996 May 15;87(10):4455-62.

    PubMed 8639808

  • Davies SM, Shu XO, Blazar BR, Filipovich AH, Kersey JH, Krivit W, McCullough J, Miller WJ, Ramsay NK, Segall M, et al. Unrelated donor bone marrow transplantation: influence of HLA A and B incompatibility on outcome. Blood. 1995 Aug 15;86(4):1636-42.

    PubMed 7632974

  • Flomenberg N, Baxter-Lowe LA, Confer D, Fernandez-Vina M, Filipovich A, Horowitz M, Hurley C, Kollman C, Anasetti C, Noreen H, Begovich A, Hildebrand W, Petersdorf E, Schmeckpeper B, Setterholm M, Trachtenberg E, Williams T, Yunis E, Weisdorf D. Impact of HLA class I and class II high-resolution matching on outcomes of unrelated donor bone marrow transplantation: HLA-C mismatching is associated with a strong adverse effect on transplantation outcome. Blood. 2004 Oct 1;104(7):1923-30. doi: 10.1182/blood-2004-03-0803. Epub 2004 Jun 10.

    PubMed 15191952

  • Ash RC, Casper JT, Chitambar CR, Hansen R, Bunin N, Truitt RL, Lawton C, Murray K, Hunter J, Baxter-Lowe LA, et al. Successful allogeneic transplantation of T-cell-depleted bone marrow from closely HLA-matched unrelated donors. N Engl J Med. 1990 Feb 22;322(8):485-94. doi: 10.1056/NEJM199002223220801.

    PubMed 2300120

  • Bunin N, Aplenc R, Iannone R, Leahey A, Grupp S, Monos D, Pierson G. Unrelated donor bone marrow transplantation for children with severe aplastic anemia: minimal GVHD and durable engraftment with partial T cell depletion. Bone Marrow Transplant. 2005 Feb;35(4):369-73. doi: 10.1038/sj.bmt.1704803.

    PubMed 15640818

  • Bunin N, Aplenc R, Grupp S, Pierson G, Monos D. Unrelated donor or partially matched related donor peripheral stem cell transplant with CD34+ selection and CD3+ addback for pediatric patients with leukemias. Bone Marrow Transplant. 2006 Jan;37(2):143-9. doi: 10.1038/sj.bmt.1705211.

    PubMed 16284615

  • Camitta B, Ash R, Menitove J, Murray K, Lawton C, Hunter J, Casper J. Bone marrow transplantation for children with severe aplastic anemia: use of donors other than HLA-identical siblings. Blood. 1989 Oct;74(5):1852-7.

    PubMed 2529004

  • Handgretinger R, Lang P. The history and future prospective of haplo-identical stem cell transplantation. Cytotherapy. 2008;10(5):443-51. doi: 10.1080/14653240802251507.

    PubMed 18615344

  • Barfield RC, Otto M, Houston J, Holladay M, Geiger T, Martin J, Leimig T, Gordon P, Chen X, Handgretinger R. A one-step large-scale method for T- and B-cell depletion of mobilized PBSC for allogeneic transplantation. Cytotherapy. 2004;6(1):1-6. doi: 10.1080/14653240310004411.

    PubMed 14985161

  • Chen X, Hale GA, Barfield R, Benaim E, Leung WH, Knowles J, Horwitz EM, Woodard P, Kasow K, Yusuf U, Behm FG, Hayden RT, Shurtleff SA, Turner V, Srivastava DK, Handgretinger R. Rapid immune reconstitution after a reduced-intensity conditioning regimen and a CD3-depleted haploidentical stem cell graft for paediatric refractory haematological malignancies. Br J Haematol. 2006 Nov;135(4):524-32. doi: 10.1111/j.1365-2141.2006.06330.x. Epub 2006 Sep 28.

    PubMed 17010105

  • Bethge WA, Faul C, Bornhauser M, Stuhler G, Beelen DW, Lang P, Stelljes M, Vogel W, Hagele M, Handgretinger R, Kanz L. Haploidentical allogeneic hematopoietic cell transplantation in adults using CD3/CD19 depletion and reduced intensity conditioning: an update. Blood Cells Mol Dis. 2008 Jan-Feb;40(1):13-9. doi: 10.1016/j.bcmd.2007.07.001. Epub 2007 Sep 14.

    PubMed 17869547

  • Ciofani M, Zuniga-Pflucker JC. Determining gammadelta versus alphass T cell development. Nat Rev Immunol. 2010 Sep;10(9):657-63. doi: 10.1038/nri2820. Epub 2010 Aug 20.

    PubMed 20725107

  • Starr TK, Jameson SC, Hogquist KA. Positive and negative selection of T cells. Annu Rev Immunol. 2003;21:139-76. doi: 10.1146/annurev.immunol.21.120601.141107. Epub 2002 Oct 16.

    PubMed 12414722

  • Drobyski WR, Majewski D. Donor gamma delta T lymphocytes promote allogeneic engraftment across the major histocompatibility barrier in mice. Blood. 1997 Feb 1;89(3):1100-9.

    PubMed 9028343

Questions about this trial

What is this clinical trial studying?

A study testing Apha/beta T and CD19+ cell depletion using CliniMACS device in people with Leukemia and Bone Marrow Failure Syndrome, run by Children's Hospital of Philadelphia at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 31 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Megan Atkinson (215-590-2820, cttsbmtintake@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03145545, which also lists 27 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2026.

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