Trial Outcomes & Findings for Study of Haplo-HSCT + Rivogenlecleucel vs Haplo-HSCT + Post Transplant Cyclophosphamide in Patients With AML or MDS (NCT NCT03699475)

Last Updated: 2023-09-29

Results Overview

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)

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

1 participants

Primary outcome timeframe

100 days

Results posted on

2023-09-29

Participant Flow

Participant milestones

Participant milestones
Measure	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
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	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
Overall Study Death	1

Baseline Characteristics

Study of Haplo-HSCT + Rivogenlecleucel vs Haplo-HSCT + Post Transplant Cyclophosphamide in Patients With AML or MDS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phase II, Cohort 1 n=1 Participants 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
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	28 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants
history of AML in the second or subsequent complete remission	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days

Population: Study terminated early after graft failure of the first participant.

Outcome measures

Outcome measures
Measure	Phase II, Cohort 1 n=1 Participants 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
Number of Subjects Experiencing 3 or More Dose Limiting Toxicities [Phase 2] Within a 100-day DLT Window After Receiving BPX-501	1 Participants

Adverse Events

Phase II, Cohort 1

Serious events: 1 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Phase II, Cohort 1 n=1 participants at risk 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
Injury, poisoning and procedural complications Transplant failure	100.0% 1/1 • Number of events 1 • 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.
Infections and infestations Viral sepsis	100.0% 1/1 • Number of events 1 • 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.
Nervous system disorders Brain hypoxia	100.0% 1/1 • Number of events 1 • 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.

Other adverse events

Other adverse events
Measure	Phase II, Cohort 1 n=1 participants at risk 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
Infections and infestations Cellulitis	100.0% 1/1 • Number of events 2 • 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.

Additional Information

Contact for Clinical Trial Information

Bellicum

Phone: (832) 384-1100

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place