Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant



Status:Recruiting
Conditions:Osteoporosis, Blood Cancer, Blood Cancer, Blood Cancer, Blood Cancer, Infectious Disease, HIV / AIDS, Lymphoma, Anemia, Hematology
Therapuetic Areas:Hematology, Immunology / Infectious Diseases, Oncology, Rheumatology
Healthy:No
Age Range:Any - 26
Updated:1/23/2019
Start Date:April 2014
End Date:December 2020
Contact:Neena Kapoor, MD
Email:nkapoor@chla.usc.edu

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Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

This study will evaluate pediatric patients with malignant or non-malignant blood cell
disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa
and beta cells that comes from a partially matched family donor. The study will assess
whether immune cells, called T cells, from the family donor, that are specially grown in the
laboratory and given back to the patient along with the stem cell transplant can help the
immune system recover faster after transplant. As a safety measure these T cells have been
programmed with a self-destruct switch so that they can be destroyed if they start to react
against tissues (graft versus host disease).

This is a Phase 2 extension study evaluating the safety and feasibility of BPX-501 T cells
infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic
stem cell transplant (HSCT) in pediatric patients. The purpose of this clinical trial is to
determine whether BPX-501 infusion can enhance immune reconstitution in those patients with
hematologic disorders, with the potential for reducing the severity and duration severe acute
graft versus host disease (GvHD).

The trial will also evaluate the treatment of GvHD by the infusion of dimerizer drug
(AP1903/rimiducid) in those subjects who present with GVHD that does not adequately respond
to standard of care therapy.

Inclusion Criteria:

1. Age > 1 month and < 26 years

2. Life expectancy > 10 weeks

3. Subjects deemed eligible for allogeneic stem cell transplantation.

4. Subjects with life-threatening hematological malignancies (high-risk ALL in 1st CR,
ALL in 2nd or subsequent CR, AML in 1st CR, AML in 2nd or subsequent CR,
myelodysplastic syndromes, non-Hodgkin lymphomas in 2nd or subsequent CR, other
hematologic malignancies eligible for stem cell transplantation per institutional
standard);

5. Non-malignant disorders amenable to cure by an allograft:

1. primary immune deficiencies,

2. severe aplastic anemia not responding to immune suppressive therapy,

3. osteopetrosis,

4. hemoglobinopathies, (thalassemias, and sickle cell anemia, and Diamond-Blackfan
anemia among others)

5. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal
malignant evolution (MDS, AML) Note: Subjects will be eligible if they meet
either item 4 OR item 5.

6. Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically
identical relative or 10/10 unrelated donor evaluated using high resolution molecular
typing) or presence of rapidly progressive disease not permitting time to identify an
unrelated donor

7. A minimum genotypic identical match of 5/ 10 is required.

8. The donor and recipient must be identical, as determined by high resolution typing, at
least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-
DRB1 and HLA-DQB1.

9. Lansky/Karnofsky score > 50

10. Signed written informed consent

Exclusion Criteria:

1. 1. Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous
allograft at the time of inclusion

2. Subject receiving an immunosuppressive treatment for GVHD treatment due to a previous
allograft at the time of inclusion

3. Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal
value), or of renal function (creatinine clearance < 30 mL / min)

4. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive
heart failure or left ventricular ejection fraction < 40%)

5. Current active infectious disease (including positive HIV serology or viral RNA)

6. Serious concurrent uncontrolled medical disorder

7. Pregnant or breastfeeding subject

8. For subjects who have received more than 1 x 10E5 alpha/beta T cells/kg with the graft
infusion the clinical trial site must contact the sponsor for approval to be eligible
to receive BPX-501 infusion.
We found this trial at
10
sites
1100 Fairview Avenue North
Seattle, Washington 98109
(206) 667-5000
Principal Investigator: Lauri M Burroughs, MD
Phone: 206-667-6993
Fred Hutchinson Cancer Research Center At Fred Hutchinson Cancer Research Center, our interdisciplinary teams of...
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Neena Kapoor, MD
Phone: 323-361-2217
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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1405 Clifton Road NE
Atlanta, Georgia 30322
404-785-6000
Principal Investigator: Lakshmanan Krishnamurti, MD
Phone: 404-785-0653
Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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Boston, Massachusetts 02215
Principal Investigator: Susanne Baumeister, MD
Phone: 617-919-7008
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Bronx, New York 10467
Principal Investigator: Michelle Lee, MD
Phone: 718-741-2076
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1011 North Galloway Avenue
Dallas, Texas 75390
Principal Investigator: Victor M Aquino, MD
Phone: 214-456-1430
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Houston, Texas
Principal Investigator: Swati Naik, MD
Phone: 832-824-4881
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Palo Alto, California 94304
Principal Investigator: Alice Bertaina, MD
Phone: 650-725-9032
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Portland, Oregon 97239
Principal Investigator: Eneida Nemecek, MD
Phone: 503-494-3639
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Principal Investigator: David Jacobsohn, MD, ScM
Phone: 202-476-6850
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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