Trial Outcomes & Findings for Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases (NCT NCT02231710)

Results Overview

To determine the safety (as defined by non-responsive Grade III-IV GVHD to rimiducid) of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled rimiducid infusion on Day 7. this outcome measure is reported as number of patients who experienced the AE of Grade III-IV GVHD that was not non-responsive to rimiducid (safety switch) administration.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

1 participants

Primary outcome timeframe

24 months

Results posted on

2024-03-25

Participant Flow

Participant milestones

Participant milestones
Measure	BPX-501 and Rimiducid Single administration of BPX-501 T cells (5 x 10\^6 cell/kg), post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 BPX-501 and Rimiducid: Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 NOTE: This study enrolled a single patient and was terminated early due to lack of enrolment
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	BPX-501 and Rimiducid Single administration of BPX-501 T cells (5 x 10\^6 cell/kg), post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 BPX-501 and Rimiducid: Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 NOTE: This study enrolled a single patient and was terminated early due to lack of enrolment
Overall Study Physician Decision	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BPX-501 and Rimiducid n=1 Participants Single administration of BPX-501 T cells (5 x 10\^6 cell/kg), post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 BPX-501 and Rimiducid: Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 NOTE: This study enrolled a single patient and was terminated early due to lack of enrolment
Age, Continuous	12 years n=1 Participants
Sex: Female, Male Female	0 Participants n=1 Participants
Sex: Female, Male Male	1 Participants n=1 Participants
Region of Enrollment United States	1 participants n=1 Participants
sickle cell disease	1 Participants n=1 Participants

PRIMARY outcome

Timeframe: 24 months

Population: One subject was dosed with 5 x 106 cells of BPX-501 T cells and 0.4 mg/kg of rimiducid on Day 7. No efficacy data was gathered for this patient and patient was withdrawn from the Study by investigator's physician. The study was terminated early by Sponsor due to lack of enrolment

To determine the safety (as defined by non-responsive Grade III-IV GVHD to rimiducid) of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled rimiducid infusion on Day 7. this outcome measure is reported as number of patients who experienced the AE of Grade III-IV GVHD that was not non-responsive to rimiducid (safety switch) administration.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 28

Population: One subject was dosed with 5 x 106 cells of BPX-501 T cells and 0.4 mg/kg of rimiducid on Day 7. No efficacy data was gathered for this patient and patient was withdrawn from the Study by investigator's physician. The study was terminated early by Sponsor due to lack of enrolment

Determine the engraftment rate (defined as \>50% donor CD3 chimerism) on day 28 after HCT with HLA-haploidentical CD34+ selected PBSC grafts per dose cohort of BPX 501 T cells followed by Rimiducid infusion on Day 7. NOTE: only one patient was enrolled who received the dose of 5x 10\^6cell/kg dose of BPX-501

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 24

To determine the incidence and severity of acute and chronic GVHD

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 24

Measure immune reconstitution

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 200

Determine the risk for severe infections

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 24

Incidence of graft rejection

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 24

Time to resolution of acute and chronic GvHD following administration of Rimiducid

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 24

Rate of high grade toxicity

Outcome measures

Outcome data not reported

Adverse Events

BPX-501 and Rimiducid

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BPX-501 and Rimiducid n=1 participants at risk Single administration of BPX-501 T cells (5 x 10\^6 cell/kg), post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 BPX-501 and Rimiducid: Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 NOTE: This study enrolled a single patient who discontinued the study due to investigating physicians decision. The study was terminated early due to lack of enrolment.
Blood and lymphatic system disorders haemolysis	100.0% 1/1 • Number of events 1 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)

Other adverse events

Other adverse events
Measure	BPX-501 and Rimiducid n=1 participants at risk Single administration of BPX-501 T cells (5 x 10\^6 cell/kg), post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 BPX-501 and Rimiducid: Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 NOTE: This study enrolled a single patient who discontinued the study due to investigating physicians decision. The study was terminated early due to lack of enrolment.
Blood and lymphatic system disorders Febrile Neutropenia	100.0% 1/1 • Number of events 1 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)
Blood and lymphatic system disorders Disseminated Intravascular Coagulation	100.0% 1/1 • Number of events 1 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)
Investigations Platelet Count Decreased	100.0% 1/1 • Number of events 22 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)
Investigations Haematocrit Decreased	100.0% 1/1 • Number of events 8 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)
Investigations Activated Partial Thromboplastin Time abnormality	100.0% 1/1 • Number of events 1 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)
Immune system disorders Hypersensitivity	100.0% 1/1 • Number of events 1 • was intended for 24 months Adverse events were collected via questioning or subject-reporting and were subsequently coded in tabular form using the MedDRA Version 21.0 adverse event dictionary and CTCAE designations (in lieu of Grade 1-4 classification)

Additional Information

Rivogenlecleucel Study Team

Bellicum Pharmaceuticals

Phone: (832) 384-1100

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place