Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
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ClinicalTrials.gov Identifier: NCT03301168 |
Recruitment Status :
Active, not recruiting
First Posted : October 4, 2017
Last Update Posted : October 5, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Acute Lymphoblastic Leukemia Leukemia, Acute Myeloid (AML), Child Lymphoma, Non-Hodgkin Myelodysplastic Syndromes Primary Immune Deficiency Disorder Osteopetrosis Cytopenia Hemoglobinopathy in Children Anemia, Aplastic | Biological: BPX-501 T cells Drug: Rimiducid | Phase 1 Phase 2 |
Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial. More info ...
This is a Phase 1/2 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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 120 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders |
Actual Study Start Date : | April 2014 |
Estimated Primary Completion Date : | May 2021 |
Estimated Study Completion Date : | May 2034 |

Arm | Intervention/treatment |
---|---|
Experimental: BPX-501 T cells and Rimiducid
TCR alpha beta depleted graft infusion with addback of BPX-501 T cells. Rimiducid: Dimerizer drug administered to subjects who present with Grade I-IV acute GVHD with inadequate response to steroids within 48 hours of treatment or mild to severe chronic GVHD with inadequate response to steroids within 7 days of treatment. |
Biological: BPX-501 T cells
T cells transduced with CaspaCIDe® safety switch
Other Name: rivogenlecleucel Drug: Rimiducid administered to inactivate BPX-501 cells in the event of GVHD
Other Name: AP1903 |
- Adverse Event [ Time Frame: Month 24 ]Demonstrate safety of BPX-501 MTD
- TRM/NRM [ Time Frame: Day 180, Month 12 ]Assess the cumulative incidence of non-relapse/transplant related mortality
- Disease-free survival [ Time Frame: Month 24 ]Disease-free survival rates after transplantation
- Relapse [ Time Frame: Month 12 ]Cumulative incidence of relapse
- Engraftment [ Time Frame: Month 24 ]Cumulative incidence of neutrophil and platelet engraftment, primary & secondary graft failure
- GvHD [ Time Frame: Month 24 ]Cumulative incidence and severity of acute and chronic GvHD
- Rimiducid Efficacy [ Time Frame: Month 24 ]Time to resolution of acute or chronic GvHD after administration of rimiducid
- Infection [ Time Frame: Month 24 ]Rate of infectious complications
- Hospitalizations [ Time Frame: Month 24 ]Duration of hospitalization and rehospitalization

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Ages Eligible for Study: | 1 Month to 26 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age > 1 month and < 26 years
- Life expectancy > 10 weeks
- Subjects deemed eligible for allogeneic stem cell transplantation.
- 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);
-
Non-malignant disorders amenable to cure by an allograft:
- primary immune deficiencies,
- severe aplastic anemia not responding to immune suppressive therapy,
- osteopetrosis,
- hemoglobinopathies, (thalassemias, and sickle cell anemia, and Diamond-Blackfan anemia among others)
- 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.
- 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
- A minimum genotypic identical match of 5/ 10 is required.
- 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.
- Lansky/Karnofsky score > 50
- Signed written informed consent
Exclusion Criteria:
- Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
- Subject receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 mL / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
- Current active infectious disease (including positive HIV serology or viral RNA)
- Serious concurrent uncontrolled medical disorder
- Pregnant or breastfeeding subject
- 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.

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): NCT03301168
United States, California | |
Children's Hospital Los Angeles | |
Los Angeles, California, United States, 90027 | |
Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine | |
Palo Alto, California, United States, 94304 | |
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Georgia | |
Children's Healthcare of Atlanta | |
Atlanta, Georgia, United States, 30322 | |
United States, Massachusetts | |
Dana-Farber Boston Children's Cancer and Blood Disorders Center | |
Boston, Massachusetts, United States, 02215 | |
United States, New York | |
Children's Hospital at Montefiore | |
Bronx, New York, United States, 10467 | |
United States, Oregon | |
Oregon Health Sciences University - Doernbecher Children's Hospital | |
Portland, Oregon, United States, 97239 | |
United States, Texas | |
University of Texas Southwestern-Children's Medical Center | |
Dallas, Texas, United States, 77390 | |
Baylor College of Medicine/ Texas Children's Hospital | |
Houston, Texas, United States, 77030 | |
United States, Washington | |
Fred Hutchinson Cancer Research Center | |
Seattle, Washington, United States, 98109 |
Study Director: | Bellicum Pharmaceuticals | Bellicum Pharmaceuticals, Inc. |
Responsible Party: | Bellicum Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03301168 |
Other Study ID Numbers: |
BP-U-004 |
First Posted: | October 4, 2017 Key Record Dates |
Last Update Posted: | October 5, 2020 |
Last Verified: | October 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
ALL AML hematologic neoplasms |
hematologic malignancies primary immune deficiences allogeneic stem cell transplant |
Leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma Lymphoma, Non-Hodgkin Leukemia, Myeloid, Acute Osteopetrosis Myelodysplastic Syndromes Hemoglobinopathies Anemia, Aplastic Immunologic Deficiency Syndromes Neoplasms by Histologic Type Neoplasms Bone Marrow Diseases Hematologic Diseases Leukemia, Lymphoid |
Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Lymphoma Genetic Diseases, Inborn Osteosclerosis Osteochondrodysplasias Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Anemia Leukemia, Myeloid |