A Phase 1/2 interventional study of BPX-501 T cells and Rimiducid in Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child and Lymphoma, Non-Hodgkin, sponsored by Bellicum Pharmaceuticals. Terminated at 4 sites in 2 countries. Open to participants aged 1 Month to 18 Years. Per ClinicalTrials.gov, last updated 2023-09-29.
Sponsored by Bellicum Pharmaceuticals · Phase 1/2, Interventional, and Treatment
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 I/II 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.
2,584 studies on the registry are indexed under Precursor Cell Lymphoblastic Leukemia-Lymphoma; 473 are open to participants now.
This study's enrollment of 187 is above the median of 40 across 1,952 interventional studies indexed under Precursor Cell Lymphoblastic Leukemia-Lymphoma.
Browse Precursor Cell Lymphoblastic Leukemia-Lymphoma studies →Bellicum Pharmaceuticals is the lead sponsor of 15 studies on the registry; none are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 5 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 · Drug: Rimiducid
1x10E6 cells/kg infused on Day 0
Also known as: Rivogenlecleucel
0.4mg/kg administered IV to treat GVHD
Also known as: AP1903
Event-free Survival (EFS) at 180 Days After Transplant
Events included transplant-related mortality (TRM) / non-relapse mortality (NRM), severe GvHD (acute Grades 2-4 organ or extensive chronic GvHD) and life-threatening infections (Grade 4). Time to the first event only is represented in the primary endpoint, if a subsequent event occurred in the same patient this was not captured in this outcome. There were no protocol-specified primary endpoints for Phase I of the study.
Time frame: 180 days after transplant
| Milestone | BPX-501 T Cells and Rimiducid |
|---|---|
| Started | 184 |
| Patients receiving rivogenlecleucel (bpx-501 safety population) | 171 |
| Itt population | 142 |
| Patients receiving at least 1 dose of rimiducid | 16 |
| Completed | 149 |
| Not completed | 35 |
| Withdrew: Death | 7 |
| Withdrew: Disease relapse | 16 |
| Withdrew: Lost to follow-up | 4 |
| Withdrew: No rivogenlecleucel cells infused (non-evaluable) | 7 |
| Withdrew: Withdrawal by subject | 1 |
Events included transplant-related mortality (TRM) / non-relapse mortality (NRM), severe GvHD (acute Grades 2-4 organ or extensive chronic GvHD) and life-threatening infections (Grade 4). Time to the first event only is represented in the primary endpoint, if a subsequent event occurred in the same patient this was not captured in this outcome. There were no protocol-specified primary endpoints for Phase I of the study.
| Event-Free Survival Estimate (%) | BPX-501 T Cells and Rimiducid |
|---|---|
| Event-free Survival (EFS) at 180 Days After Transplant | 90.8 (84.6 to 94.5) |
Collected over Within 180 days post BPX-501 or 30 days post Rimiducid. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BPX-501 | 7/171 (4.1%) | 52/171 (30.4%) | 140/171 (81.9%) |
| Rimiducid | 0/16 (0%) | 7/16 (43.8%) | 9/16 (56.3%) |
| Event | BPX-501 | Rimiducid |
|---|---|---|
| Acute graft versus host diseaseImmune system disorders | 4/171 | 2/16 |
| Gastrointestinal haemorrhageGastrointestinal disorders | 1/171 | 1/16 |
| Parotid gland enlargementGastrointestinal disorders | 1/171 | 1/16 |
| SepsisInfections and infestations | 1/171 | 1/16 |
| Septic shockInfections and infestations | 1/171 | 1/16 |
| Staphylococcal bacteraemiaInfections and infestations | 1/171 | 1/16 |
| Central nervous system lesionNervous system disorders | 1/171 | 1/16 |
| EncephalopathyNervous system disorders | 1/171 | 1/16 |
| Pulmonary hypertensionRespiratory, thoracic and mediastinal disorders | 1/171 | 1/16 |
| Respiratory distressRespiratory, thoracic and mediastinal disorders | 1/171 | 1/16 |
| Event | BPX-501 | Rimiducid |
|---|---|---|
| Acute graft versus host diseaseInfections and infestations | 48/171 | 3/16 |
| Cytomegalovirus infectionInfections and infestations | 34/171 | 0/16 |
| PyrexiaGeneral disorders | 33/171 | 0/16 |
| RashSkin and subcutaneous tissue disorders | 21/171 | 0/16 |
| DiarrhoeaGastrointestinal disorders | 20/171 | 0/16 |
| VomitingGastrointestinal disorders | 20/171 | 0/16 |
| PruritusSkin and subcutaneous tissue disorders | 12/171 | 0/16 |
| Adenovirus infectionInfections and infestations | 11/171 | 0/16 |
| SepsisInfections and infestations | 1/171 | 1/16 |
| Septic shockInfections and infestations | 1/171 | 1/16 |
All enrolled patients iwho received HSCT (HSCT safety population)
| Age, Continuous(years) | BPX-501 T Cells and Rimiducid |
|---|---|
| Mean | 5.8 (0.1 to 18.1) |
| Sex: Female, Male(Participants) | BPX-501 T Cells and Rimiducid |
|---|---|
| Female | 83 |
| Male | 101 |
| Race (NIH/OMB)(Participants) | BPX-501 T Cells and Rimiducid |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 10 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 162 |
| More than one race | 1 |
| Unknown or Not Reported | 11 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Apr 2014 to primary completion in Jun 2020, 74 months.
Results were first posted in Jan 2023, 2 years 7 months after primary completion. A results delay was requested in Nov 2020.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Rivogenlecleucel
Precursor Cell Lymphoblastic Leukemia-Lymphoma →
Bellicum Pharmaceuticals