Trial Outcomes & Findings for Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant (NCT NCT02065869)

NCT ID: NCT02065869

Last Updated: 2023-09-29

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

187 participants

Primary outcome timeframe

180 days after transplant

Results posted on

2023-09-29

Participant Flow

Participant milestones

Participant milestones
Measure	BPX-501 T Cells and Rimiducid 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 BPX-501 T cells: 1x10E6 cells/kg infused on Day 0 Rimiducid: 0.4mg/kg administered IV to treat GVHD
Overall Study STARTED	184
Overall Study Patients Receiving Rivogenlecleucel (BPX-501 Safety Population)	171
Overall Study ITT Population	142
Overall Study Patients Receiving at Least 1 Dose of Rimiducid	16
Overall Study COMPLETED	149
Overall Study NOT COMPLETED	35

Reasons for withdrawal

Reasons for withdrawal
Measure	BPX-501 T Cells and Rimiducid 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 BPX-501 T cells: 1x10E6 cells/kg infused on Day 0 Rimiducid: 0.4mg/kg administered IV to treat GVHD
Overall Study Death	7
Overall Study Disease relapse	16
Overall Study Lost to Follow-up	4
Overall Study No rivogenlecleucel cells infused (non-evaluable)	7
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BPX-501 T Cells and Rimiducid n=184 Participants 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 BPX-501 T cells: 1x10E6 cells/kg infused on Day 0 Rimiducid: 0.4mg/kg administered IV to treat GVHD
Age, Continuous	5.8 years n=5 Participants
Sex: Female, Male Female	83 Participants n=5 Participants
Sex: Female, Male Male	101 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	10 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	162 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	11 Participants n=5 Participants

PRIMARY outcome

Timeframe: 180 days after transplant

Population: ITT population: All patients treated with HSCT who received 1×10\^6 cells/kg BPX-501

Outcome measures

Outcome measures
Measure	BPX-501 T Cells and Rimiducid n=142 Participants 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 BPX-501 T cells: 1x10E6 cells/kg infused on Day 0 Rimiducid: 0.4mg/kg administered IV to treat GVHD
Event-free Survival (EFS) at 180 Days After Transplant	90.8 Event-Free Survival Estimate (%) Interval 84.6 to 94.5

Adverse Events

BPX-501

Serious events: 52 serious events

Other events: 140 other events

Deaths: 7 deaths

Rimiducid

Serious events: 7 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

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

Serious adverse events
Measure	BPX-501 n=171 participants at risk BPX-501 (rivogenlecleucel): TCR alpha beta depleted graft infusion with addback of BPX-501 T cells BPX-501 T cells: 1x10E6 cells/kg infused on Day 0	Rimiducid n=16 participants at risk 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 Rimiducid: 0.4mg/kg administered IV to treat GVHD
Gastrointestinal disorders Parotid gland enlargement	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	6.2% 1/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Anaemia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Autoimmune haemolytic anaemia	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Cytopenia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Haemolytic anaemia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Histiocytosis haematophagic	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Neutropenia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Thrombocytopenia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Blood and lymphatic system disorders Thrombotic microangiopathy	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Cardiac disorders Pericardial effusion	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Cardiac disorders Pneumopericardium	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Eye disorders Diplopia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Gastrointestinal disorders Diarrhoea	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Gastrointestinal disorders Gastrointestinal haemorrhage	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	6.2% 1/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Gastrointestinal disorders Haematemesis	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Gastrointestinal disorders Pancreatitis	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Gastrointestinal disorders Vomiting	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
General disorders Device damage	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
General disorders Malaise	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
General disorders Medical device complication	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
General disorders Pyrexia	4.1% 7/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Immune system disorders Acute graft versus host disease	2.3% 4/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	12.5% 2/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Adenovirus infection	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Bacterial sepsis	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Cytomegalovirus infection	1.8% 3/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Device related infection	1.8% 3/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Encephalitis	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Escherichia infection	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Gastroenteritis rotavirus	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Infection	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Klebsiella infection	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Pharyngitis	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Pseudomonas infection	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Respiratory tract infection	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Sepsis	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	6.2% 1/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Septic shock	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	6.2% 1/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Staphylococcal bacteraemia	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	6.2% 1/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Staphylococcal infection	0.58% 1/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Staphylococcal sepsis	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)
Infections and infestations Varicella zoster virus infection	1.2% 2/171 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)	0.00% 0/16 • Within 180 days post BPX-501 or 30 days post Rimiducid Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT). Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)