A Phase 2/3 interventional study of rivogenlecleucel and rimiducid in Acute Myeloid Leukemia and Myelodysplastic Syndromes, sponsored by Bellicum Pharmaceuticals. Terminated at 2 sites in United States. Open to participants aged 12 Years to 70 Years. Per ClinicalTrials.gov, last updated 2023-09-29.
Sponsored by Bellicum Pharmaceuticals · Phase 2/3, Interventional, and Treatment
This study compares the safety and effectiveness of giving rivogenlecleucel (BPX-501 T cells) to patients with AML or MDS post haploidentical hematopoietic stem cell transplant compared to post-transplant cyclophosphamide.
In the Phase 2 portion, participants will undergo αβ T cell and CD19+ B cell depleted haploidentical HSCT followed by an infusion of a fixed dose of rivogenlecleucel (BPX-501 T cells) per kg. These participants will be evaluated for prespecified dose limiting toxicities (DLTs) for a 100-day dose limiting toxicity window.
Following completion of the Phase 2 portion, participants will be enrolled and randomized to one of two treatment arms in the Phase 3 portion.
Pediatric patients ages 12-17 will also be included in US only.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's enrollment of 1 is below the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute 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.
Signed
Meeting institutional criteria to undergo allogenic
Age 18-70 y/o (12-70 y/o in US only)
Patients with AML or MDS as defined below:
AML Patients Patients with intermediate to adverse AML as defined by ELN (Dohner, 2017).
MDS Patients
Lack of suitable conventional donor (i.e. HLA 10/10 related or unrelated donor)
At least a 5/10 genotypic identical haplotype match
The donor and recipient must be identical, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1, and HLA-DQB1
Patients with adequate organ function
(ECOG) performance status: 0-2
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.
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.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
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.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
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.
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.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
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.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Determining the safety of maximum allowable Dose for BPX-501 starting at 3 x 10E6 cells/kg Rimiducid will be administered to inactivate rivogenlecleucel in the event of GVHD not responsive to standard of care treatment
Biological: rivogenlecleucel · Drug: rimiducid · Procedure: haplo-HSCT
αβ T cell and CD19+ B cell-depleted, related haploidentical hematopoietic stem cell transplantation (haplo-HSCT) plus rivogenlecleucel
Biological: rivogenlecleucel · Drug: rimiducid · Procedure: haplo-HSCT
haplo-HSCT followed by post-transplant cyclophosphamide (PTCy) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)
Drug: Cyclophosphamide · Procedure: haplo-HSCT
Biological: T cells transduced with caspase 9 safety switch
Also known as: BPX-501 T cells
administered to inactivate rivogenlecleucel in the event of GVHD
Also known as: AP1903
GVHD prophylaxis
Also known as: Cytoxan
treatment for disease
Number of Subjects Experiencing 3 or More Dose Limiting Toxicities [Phase 2] Within a 100-day DLT Window After Receiving BPX-501
If any of the following adverse events that occur within the DLT window they will be considered a DLT: * Grade III or IV acute GVHD attributable to rivogenlecleucel and non-responsive to \> 1 dose of rimiducid treatment (plus standard doses (at least 1 mg/kg) of methylprednisone or dose equivalent of other corticosteroids, and/or calcineurin inhibitor) within 14 days * Grade 3-4 neurologic events attributable to rivogenlecleucel * Death due to any cause other than underlying disease * Any CTCAE Grade 3-5 adverse events related to rivogenlecleucel (including allergic reactions, infusion reactions, and any other related adverse reactions whether expected or unexpected). in case 3 or more DLTs are observed with 3 x 10E6 dose, another cohort would have been enrolled to receive the 1 x 10E6 cell dose (never happened as study terminated early)
Time frame: 100 days
| Milestone | Phase II, Cohort 1 (Only Study Arm That the Only Participant Enrolled Into) |
|---|---|
| Started | 1 |
| Completed | 0 |
| Not completed | 1 |
| Withdrew: Death | 1 |
If any of the following adverse events that occur within the DLT window they will be considered a DLT: * Grade III or IV acute GVHD attributable to rivogenlecleucel and non-responsive to \> 1 dose of rimiducid treatment (plus standard doses (at least 1 mg/kg) of methylprednisone or dose equivalent of other corticosteroids, and/or calcineurin inhibitor) within 14 days * Grade 3-4 neurologic events attributable to rivogenlecleucel * Death due to any cause other than underlying disease * Any CTCAE Grade 3-5 adverse events related to rivogenlecleucel (including allergic reactions, infusion reactions, and any other related adverse reactions whether expected or unexpected). in case 3 or more DLTs are observed with 3 x 10E6 dose, another cohort would have been enrolled to receive the 1 x 10E6 cell dose (never happened as study terminated early)
| Participants | Phase II, Cohort 1 |
|---|---|
| Number of Subjects Experiencing 3 or More Dose Limiting Toxicities [Phase 2] Within a 100-day DLT Window After Receiving BPX-501 | 1 |
Collected over from enrolment to 43 days after receiving BPX-501 (subject passed away at this point, study had only enrolled 1 patient). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase II, Cohort 1 | 1/1 (100%) | 1/1 (100%) | 1/1 (100%) |
| Event | Phase II, Cohort 1 |
|---|---|
| Transplant failureInjury, poisoning and procedural complications | 1/1 |
| Viral sepsisInfections and infestations | 1/1 |
| Brain hypoxiaNervous system disorders | 1/1 |
| Event | Phase II, Cohort 1 |
|---|---|
| CellulitisInfections and infestations | 1/1 |
only 1 patient was enrolled in this study
| Age, Categorical(Participants) | Phase II, Cohort 1 |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 1 |
| >=65 years | 0 |
| Age, Continuous(years) | Phase II, Cohort 1 |
|---|---|
| Median | 28 (28 to 28) |
| Sex: Female, Male(Participants) | Phase II, Cohort 1 |
|---|---|
| Female | 0 |
| Male | 1 |
| Ethnicity (NIH/OMB)(Participants) | Phase II, Cohort 1 |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 0 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Phase II, Cohort 1 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 1 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Phase II, Cohort 1 |
|---|---|
| United States | 1 |
| history of AML in the second or subsequent complete remission(Participants) | Phase II, Cohort 1 |
|---|---|
| Count of participants | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
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This study is terminated, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Rivogenlecleucel
Bellicum Pharmaceuticals