Not provided
Not provided
Not provided
Not provided
Funding, portfolio re-prioritization
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| single-arm Phase II: 3 x 10E6 BPX-501 cell/kg | Experimental | 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 |
|
| phase 3 Arm A: Dose Determined in phase 2 group (never completed) | Experimental | αβ T cell and CD19+ B cell-depleted, related haploidentical hematopoietic stem cell transplantation (haplo-HSCT) plus rivogenlecleucel |
|
| phase 3 Arm B: dose determined in the phase 2 group (never completed) | Active Comparator | haplo-HSCT followed by post-transplant cyclophosphamide (PTCy) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rivogenlecleucel | Biological | Biological: T cells transduced with caspase 9 safety switch |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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:
| 100 days |
Not provided
Not provided
Inclusion Criteria:
Signed informed consent
Meeting institutional criteria to undergo allogenic HSCT
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
Eastern Cooperative Oncology Group (ECOG) performance status: 0-2
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Bellicum Pharmaceuticals | Bellicum Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| TriStar Bone Marrow Transplant, LLC | Nashville | Tennessee | 37203 | United States | ||
| Methodist Healthcare System of San Antonio Clinical Trials Office |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Phase II, Cohort 1 (Only Study Arm That the Only Participant Enrolled Into) | αβ T-cell and CD19+ B-cell-depleted haploidentical stem cell transplantation plus rivogenlecleucel (BPX-501) dose: 3x 10^6 cells/kg Rimiducid only to be administered to inactivate rivogenlecleucel in the event of GVHD not responsive to standard of care treatment; (study terminated after enrollment of the first patient total study participant number is 1) ---------------------- rivogenlecleucel: Biological: T cells transduced with caspase 9 safety switch rimiducid: administered to inactivate rivogenlecleucel in the event of GVHD haplo-HSCT: treatment for disease |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 5, 2019 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| rimiducid | Drug | administered to inactivate rivogenlecleucel in the event of GVHD |
|
|
| Cyclophosphamide | Drug | GVHD prophylaxis |
|
|
| haplo-HSCT | Procedure | treatment for disease |
|
| San Antonio |
| Texas |
| 78229 |
| United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
only 1 patient was enrolled in this study
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Phase II, Cohort 1 | αβ T-cell and CD19+ B-cell-depleted haploidentical stem cell transplantation plus rivogenlecleucel Rimiducid will be administered to inactivate rivogenlecleucel in the event of GVHD not responsive to standard of care treatment rivogenlecleucel: Biological: T cells transduced with caspase 9 safety switch rimiducid: administered to inactivate rivogenlecleucel in the event of GVHD haplo-HSCT: treatment for disease |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||||
| history of AML in the second or subsequent complete remission | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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:
| Study terminated early after graft failure of the first participant. | Posted | Count of Participants | Participants | 100 days |
|
|
|
from enrolment to 43 days after receiving BPX-501 (subject passed away at this point, study had only enrolled 1 patient)
All AEs were reported. CTCAE version 5.0 was used for grading.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Phase II, Cohort 1 | αβ T-cell and CD19+ B-cell-depleted haploidentical stem cell transplantation plus rivogenlecleucel Rimiducid will be administered to inactivate rivogenlecleucel in the event of GVHD not responsive to standard of care treatment rivogenlecleucel: Biological: T cells transduced with caspase 9 safety switch rimiducid: administered to inactivate rivogenlecleucel in the event of GVHD haplo-HSCT: treatment for disease | 1 | 1 | 1 | 1 | 1 | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Transplant failure | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Viral sepsis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Brain hypoxia | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cellulitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Contact for Clinical Trial Information | Bellicum | (832) 384-1100 | clinicaltrials@bellicum.com |
| Jul 31, 2023 |
| Prot_SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C423866 | AP 1903 reagent |
| D003520 | Cyclophosphamide |
| ID | Term |
|---|---|
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|