Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant
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ClinicalTrials.gov Identifier: NCT02065869 |
Recruitment Status :
Active, not recruiting
First Posted : February 19, 2014
Last Update Posted : November 17, 2020
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Condition or disease | Intervention/treatment | Phase |
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Acute Lymphoblastic Leukemia Leukemia, Acute Myeloid (AML), Child Lymphoma, Non-Hodgkin Myelodysplastic Syndrome Primary Immunodeficiency Anemia, Aplastic Osteopetrosis Hemoglobinopathies Cytopenia Fanconi Anemia Diamond Blackfan Anemia Thalassemia Anemia, Sickle Cell | Biological: BPX-501 T cells Drug: rimiducid | Phase 2 |
This is a Phase II 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 who progress or do not respond to standard of care treatment.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 193 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | April 2014 |
Actual Primary Completion Date : | February 2019 |
Estimated Study Completion Date : | February 2033 |

Arm | Intervention/treatment |
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Experimental: BPX-501 T cells and rimiducid
TCR alpha beta depleted graft infusion with addback of BPX-501 T cells (rivogenlecleucel). Rimiducid/AP1903: Dimerizer drug administered to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment |
Biological: BPX-501 T cells
1x10E6 cells/kg infused on Day 0
Other Name: rivogenlecleucel Drug: rimiducid 0.4mg/kg administered IV to treat GVHD
Other Name: AP1903 |
- Event-Free -Survival [ Time Frame: 180 days after transplant ]events include TRM (or NRM for malignant patients), severe GVHD (acute Grade 2-4 organ or extensive chronic GVHD) and life threatening infections (Grade 4)
- TRM (non-malignant) or NRM (malignant) [ Time Frame: 180 days after transplant ]transplant related mortality or non relapse mortality (patients with malignant disease)
- Cumulative incidence and severity of acute (grade 2-4) and chronic GvHD at 180 days [ Time Frame: 180 days after transplant ]grades 2-4 acute and chronic GvHD
- Time to resolution of acute GvHD after administration of rimiducid [ Time Frame: 180 days after transplant ]resolution of acute GvHD
- Immune reconstitution [ Time Frame: 180 days after transplant ]absolute CD3, CD4 and CD8 count
- Disease free/cGvHD [ Time Frame: 180 days after transplant ]Disease free/cGvHD survival
- Disease status [ Time Frame: 180 days after transplant ]Disease status of each specific disease indication

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, Learn About Clinical Studies.
Ages Eligible for Study: | 1 Month to 18 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age < 18 years and > 1 month (< 1 month upon approval by Sponsor)
- Life expectancy > 10 weeks
- Patients deemed clinically eligible for allogeneic stem cell transplantation.
- Patients may have failed prior allograft
- Patients with life-threatening acute leukemia (high-risk ALL in 1st CR, ALL in 2nd CR, high-risk AML in 1st CR, AML in 2nd CR.) or myelodysplastic syndromes. Morphological CR must be documented and minimal residual disease measurement before transplantation is recommended.
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Non-malignant disorders deemed curable by allogeneic transplantation: a. primary immune deficiencies, b. severe aplastic anemia not responding to immune suppressive therapy, c. osteopetrosis d. selected cases of erythroid disorders such as β0 β0 thalassemia major, sickle cell disease, Diamond-Blackfan anemia.
e. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML).
Note: Subjects will be eligible if they meet either item 5 OR item 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
- A minimum genotypic identical match of 5/10 is required.
- The donor and recipient must be identical, as determined by high resolution typing, on 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 informed consent by the patient or the patient's parent or guardian for patients who are minors
Exclusion Criteria:
- Greater than active Grade II acute GvHD or chronic extensive GvHD due to a previous allograft at the time of screening
- Patient receiving an immunosuppressive treatment for GvHD treatment due to a previous allograft at the time of screening
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance <30ml/min/1.73m2)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
- Current clinically active infectious disease (including positive HIV serology or viral RNA)
- Serious concurrent uncontrolled medical disorder
- Pregnant or breast feeding female patient
- Lack of parents'/guardian's informed consent for children who are minors.

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): NCT02065869
Italy | |
IRCCS Ospedale Pediatrico Bambino Gesù | |
Roma, Italy, 00161 | |
United Kingdom | |
Royal Free London NHS Foundation Trust | |
London, United Kingdom, NW3 2QG | |
Institute of Child Health & Great Ormond Street Hospital | |
London, United Kingdom, WC1N 1EH | |
Great North Children's Hospital | |
Newcastle Upon Tyne, United Kingdom, NE1 4LP |
Study Director: | Bellicum Pharmaceuticals | Bellicum Pharmaceuticals, Inc. |
Responsible Party: | Bellicum Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT02065869 |
Other Study ID Numbers: |
BP-004 |
First Posted: | February 19, 2014 Key Record Dates |
Last Update Posted: | November 17, 2020 |
Last Verified: | November 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 |
ALL AML hematologic neoplasms hematologic malignancies |
primary immune deficiences hemoglobinopathy congenital cytopenia anemia |
Leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma Lymphoma, Non-Hodgkin Leukemia, Myeloid, Acute Osteopetrosis Anemia Myelodysplastic Syndromes Thalassemia Fanconi Anemia Hemoglobinopathies Anemia, Sickle Cell Anemia, Diamond-Blackfan Anemia, Aplastic Neoplasms by Histologic Type Neoplasms |
Hematologic Diseases Bone Marrow Diseases Leukemia, Lymphoid Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Genetic Diseases, Inborn Lymphoma Anemia, Hypoplastic, Congenital DNA Repair-Deficiency Disorders Metabolic Diseases Red-Cell Aplasia, Pure |