Trial Outcomes & Findings for Phase 1-2 MAHCT w/ TCell Depleted Graft w/ Simultaneous Infusion Conventional and Regulatory T Cell (NCT NCT01660607)
NCT ID: NCT01660607
Last Updated: 2025-08-19
Results Overview
GvHD-free is defined as no GvHD symptoms, and relapse free survival is defined as survival at 12 months without relapse.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
68 participants
Primary outcome timeframe
12 months
Results posted on
2025-08-19
Participant Flow
Participant milestones
| Measure |
Arm/Group Title Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 2, Phase 1: Mid Dose Treg + Tcon (Dose Escalation)
Participants receive low-dose Treg (3e6 cells/kg), Tcon (1e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Cohort 3, Phase I: High Dose Treg + Tcon (Dose Escalation)
Participants receive high-dose Treg (3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Cohort 3A, Phase I: High Dose Treg + Tcon (Dose Escalation)
Participants receive high-dose Treg (1e6 cells/kg), Tcon (1e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 1 Treg + Tcon With Immunosuppression (Cohort 2A)
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 1 Treg + Tcon Without Immunosuppression (Cohort 2A)
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 2 Treg + Tcon With Immunosuppression (Cohort 2A)
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation. Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graftversus- host disease. Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement. Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation
|
|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
1
|
5
|
0
|
6
|
0
|
0
|
12
|
12
|
32
|
|
Overall Study
Day +28 Completed
|
1
|
5
|
0
|
6
|
0
|
0
|
12
|
12
|
32
|
|
Overall Study
12-Month Follow-Up Completed
|
1
|
5
|
0
|
6
|
0
|
0
|
10
|
10
|
26
|
|
Overall Study
24-Month Follow-Up Completed
|
1
|
5
|
0
|
6
|
0
|
0
|
10
|
10
|
24
|
|
Overall Study
COMPLETED
|
0
|
2
|
0
|
3
|
0
|
0
|
10
|
10
|
24
|
|
Overall Study
NOT COMPLETED
|
1
|
3
|
0
|
3
|
0
|
0
|
2
|
2
|
8
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 1-2 MAHCT w/ TCell Depleted Graft w/ Simultaneous Infusion Conventional and Regulatory T Cell
Baseline characteristics by cohort
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2 , Stage 1 Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 1 Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation. Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graftversus- host disease. Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement. Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation
|
Total
n=68 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|
|
Age, Customized
< 30
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
11 Participants
n=30 Participants
|
16 Participants
n=3 Participants
|
|
Age, Customized
30 - 39
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
4 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
2 Participants
n=30 Participants
|
10 Participants
n=3 Participants
|
|
Age, Customized
40 - 49
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=31 Participants
|
8 Participants
n=30 Participants
|
18 Participants
n=3 Participants
|
|
Age, Customized
50 - 59
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
3 Participants
n=31 Participants
|
5 Participants
n=30 Participants
|
17 Participants
n=3 Participants
|
|
Age, Customized
equal or less than 60
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
6 Participants
n=30 Participants
|
7 Participants
n=3 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=31 Participants
|
16 Participants
n=30 Participants
|
30 Participants
n=3 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
9 Participants
n=31 Participants
|
16 Participants
n=30 Participants
|
38 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=31 Participants
|
7 Participants
n=30 Participants
|
14 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
11 Participants
n=7 Participants
|
9 Participants
n=31 Participants
|
25 Participants
n=30 Participants
|
54 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
9 Participants
n=30 Participants
|
12 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
1 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
1 Participants
n=3 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
10 Participants
n=7 Participants
|
8 Participants
n=31 Participants
|
17 Participants
n=30 Participants
|
43 Participants
n=3 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
6 Participants
n=30 Participants
|
11 Participants
n=3 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=99 Participants
|
5 participants
n=107 Participants
|
6 participants
n=206 Participants
|
12 participants
n=7 Participants
|
12 participants
n=31 Participants
|
32 participants
n=30 Participants
|
68 participants
n=3 Participants
|
PRIMARY outcome
Timeframe: 12 monthsGvHD-free is defined as no GvHD symptoms, and relapse free survival is defined as survival at 12 months without relapse.
Outcome measures
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
|
Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
|---|---|---|---|---|---|---|
|
GvHD Free Relapse Free Survival (GRFS)
|
1 Participants
|
1 Participants
|
0 Participants
|
9 Participants
|
7 Participants
|
26 Participants
|
SECONDARY outcome
Timeframe: 28 daysDose-limiting Toxicity (DLT) was assessed as: * Absolute neutrophil count \<500/µL, to 28 day * Cytokine release syndrome/acute infusion reactions as CTCAE Grade 3 to 5 * Grade 3 to 4 acute GvHD. GvHD was staged as follows: * 1: Skin: rash \<25%. Liver: bilirubin (BIL) 2-3mg/dL. Gut: diarrhea (DIA) 500-1000 mL/day * 2: Skin: rash 25-50%. Liver: BIL 3-6mg/dL. Gut: DIA 1001-1500 mL/day * 3: Skin: rash \> 50%. Liver: BIL 6-15mg/dL. Gut: DIA \>1501-2000 mL/day * 4: Skin: generalized erythroderma. Liver: BIL \>15mg/dL. Gut: DIA \>2001 mL/day GvHD was graded as follows. * 1: Skin Stage 1-2; No Liver stage; No Gut stage * 2: Skin Stage 1-3 ; Liver Stage 1; +/- Gut Stage 1 * 3: Skin Stage 2-3, Liver Stage 2-4; +/- Gut Stage 2-3 * 4: Skin Stage 2-4; Liver Stage 2-4; +/- Gut Stage 2-4 The outcome is reported as the number of participants who received both Treg and Tcon cell infusions and had DLT events, per treatment level.
Outcome measures
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
|
Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
|---|---|---|---|---|---|---|
|
Number of Participants With Dose-Limiting Toxicity (DLT) Within 28 Days
Total DLT Events
|
1 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Dose-Limiting Toxicity (DLT) Within 28 Days
Grade 3 Acute GvHD
|
1 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
1 Participants
|
|
Number of Participants With Dose-Limiting Toxicity (DLT) Within 28 Days
Grade 4 Acute GvHD
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Dose-Limiting Toxicity (DLT) Within 28 Days
Grade 3-5 CRS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Dose-Limiting Toxicity (DLT) Within 28 Days
ANC <500
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearOverall Survival (OS) at 1 year was assessed as the number of participants per treatment level that received the hematopoietic cell transplant (HCT), and remained alive 12 months later.
Outcome measures
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
|
Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
|---|---|---|---|---|---|---|
|
Number of Participants With Overall Survival (OS) at 1 Year
|
0 Participants
|
2 Participants
|
6 Participants
|
11 Participants
|
10 Participants
|
28 Participants
|
SECONDARY outcome
Timeframe: 2 yearsIncidence and severity of chronic GvHD was assessed in participants who received the hematopoietic cell transplant (HCT) at 24 months.
Outcome measures
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
|
Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
|---|---|---|---|---|---|---|
|
Number of Participants With Severity of Chronic Graft-vs-Host Disease (cGvHD) at 24 Months
Milld cGvHD
|
0 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
6 Participants
|
|
Number of Participants With Severity of Chronic Graft-vs-Host Disease (cGvHD) at 24 Months
Moderate cGvHD
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
7 Participants
|
|
Number of Participants With Severity of Chronic Graft-vs-Host Disease (cGvHD) at 24 Months
Severe cGvHD
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 24 monthsThe outcome is reported as the number of serious infections per treatment level, in participants who received the hematopoietic cell transplant (HCT).
Outcome measures
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
|
Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
|---|---|---|---|---|---|---|
|
Number of Participants With Incidence of Serious Infections
|
0 Participants
|
1 Participants
|
6 Participants
|
3 Participants
|
0 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 2 yearsDuring Phase 2, stage 1, concomitant single-agent immunosuppression was assessed as in participants receiving fresh Treg cells.
Outcome measures
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 Participants
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 Participants
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
|
Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 Participants
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
|
Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 Participants
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
|
|---|---|---|---|---|---|---|
|
Number of Participants Receiving Concomitant Single-Agent Immunosuppression
|
0 Participants
|
1 Participants
|
6 Participants
|
12 Participants
|
12 Participants
|
32 Participants
|
Adverse Events
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
Serious events: 1 serious events
Other events: 0 other events
Deaths: 1 deaths
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
Serious events: 2 serious events
Other events: 4 other events
Deaths: 3 deaths
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
Serious events: 6 serious events
Other events: 6 other events
Deaths: 2 deaths
Phase 2, Staage 1 Treg + Tcon With Immunosuppression (Cohort 2A)
Serious events: 2 serious events
Other events: 11 other events
Deaths: 3 deaths
Phase 2, Stage 1 Treg + Tcon Without Immunosuppression (Cohort 2A)
Serious events: 6 serious events
Other events: 11 other events
Deaths: 2 deaths
Phase 2, Stage 2 Treg + Tcon With Immunosuppression (Cohort 2A)
Serious events: 10 serious events
Other events: 25 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 participants at risk
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 participants at risk
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 participants at risk
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Staage 1 Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 participants at risk
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 1 Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 participants at risk
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 2 Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 participants at risk
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation. Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graftversus- host disease. Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement. Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation
|
|---|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
100.0%
6/6 • Number of events 6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 3 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
CML relapse
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
Graft failure
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Ascites
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Epigastric pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Mucositis oral
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
33.3%
2/6 • Number of events 2 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Acute GI and Liver GVHD
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
General disorders
Fever
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Hepatobiliary disorders
Acute cholecystitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Hepatobiliary disorders
Gallbladder obstruction
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Hepatobiliary disorders
Sinusoidal obstruction syndrome
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Hepatobiliary disorders
Liver GVHD
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Bacteremia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Cytomegalovirus infection reactivation
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Epstein-Barr Infection reactivation
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Herpes simplex reactivation
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 3 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Sepsis
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Rhizomucor/aspergillus/rhizopus
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Hemorrhagic cystitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Transaminitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Nervous system disorders
Syncope
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Nervous system disorders
Vasovagal reaction
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Nervous system disorders
Altered mental status
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure secondary to pneumonia
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Renal and urinary disorders
Urinary tract obstruction
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Renal and urinary disorders
Renal failure
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Renal and urinary disorders
Renal insufficiency
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
6.2%
2/32 • Number of events 2 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Rash maculopapular
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Skin GVHD
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
16.7%
2/12 • Number of events 2 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Vascular disorders
Hypotension
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Vascular disorders
Hematoma of the bladder
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Vascular disorders
Orthostatic hypertension
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Investigations
Creatinine increased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Investigations
Elevated INR
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Investigations
Transminitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
100.0%
1/1 • Number of events 1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
Other adverse events
| Measure |
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=1 participants at risk
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (3e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
n=5 participants at risk
Participants receive low-dose Treg (1e6 cells/kg) and Tcon (1e6 cells/kg) with CD34+ HSPC.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
|
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
n=6 participants at risk
Participants receive low-dose Treg (up to 3e6 cells/kg), Tcon (3e6 cells/kg), and CD34+ HSPC following a conditioning regimen.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Staage 1 Treg + Tcon With Immunosuppression (Cohort 2A)
n=12 participants at risk
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 1 Treg + Tcon Without Immunosuppression (Cohort 2A)
n=12 participants at risk
Participants receive Treg and Tcon therapy without immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation.
Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graft-versus-host disease.
Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement.
Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation.
|
Phase 2, Stage 2 Treg + Tcon With Immunosuppression (Cohort 2A)
n=32 participants at risk
Participants receive Treg and Tcon therapy with immunosuppressive drugs following myeloablative conditioning.
CD34+ Hematopoietic Progenitor Cells (HSPC): Purified CD34+ hematopoietic progenitor cells used in transplantation. Regulatory T-Cells (Treg): Highly purified CD4+CD25+CD127-FoxP3+ regulatory T cells to reduce graftversus- host disease. Conventional T-Cells (Tcon): Conventional CD3+ T cells used for immune reconstitution and graft enhancement. Myeloablative Conditioning Regimen: Chemotherapy or total body irradiation used before hematopoietic cell transplantation
|
|---|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/1 • Up to 2 years
|
80.0%
4/5 • Number of events 5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
58.3%
7/12 • Number of events 8 • Up to 2 years
|
66.7%
8/12 • Number of events 9 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
25.0%
3/12 • Number of events 3 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
White blood cell decreased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 2 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Blood and lymphatic system disorders
pancytopenia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Cardiac disorders
Sinus tachycardia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
Cardiac disorders
Hypotension
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Cardiac disorders
Hypertension
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Cardiac disorders
chest pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Eye disorders
Periorbital Edema (retinal hemorrhage)
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Gastrointestinal disorders
Mucositis oral
|
0.00%
0/1 • Up to 2 years
|
40.0%
2/5 • Number of events 2 • Up to 2 years
|
66.7%
4/6 • Number of events 8 • Up to 2 years
|
33.3%
4/12 • Number of events 6 • Up to 2 years
|
33.3%
4/12 • Number of events 5 • Up to 2 years
|
34.4%
11/32 • Number of events 11 • Up to 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/1 • Up to 2 years
|
40.0%
2/5 • Number of events 2 • Up to 2 years
|
83.3%
5/6 • Number of events 6 • Up to 2 years
|
41.7%
5/12 • Number of events 5 • Up to 2 years
|
16.7%
2/12 • Number of events 2 • Up to 2 years
|
15.6%
5/32 • Number of events 5 • Up to 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
83.3%
5/6 • Number of events 7 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
25.0%
3/12 • Number of events 3 • Up to 2 years
|
15.6%
5/32 • Number of events 6 • Up to 2 years
|
|
Gastrointestinal disorders
Abdominal distention
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Gastrointestinal disorders
Ascites
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
33.3%
2/6 • Number of events 2 • Up to 2 years
|
16.7%
2/12 • Number of events 2 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
Gastrointestinal disorders
Vomiting (Emesis)
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
50.0%
3/6 • Number of events 4 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Rectal hemorrhoids
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
Gastrointestinal disorders
Gastric hemorrhage
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
GI GVHD
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
6.2%
2/32 • Number of events 2 • Up to 2 years
|
|
Gastrointestinal disorders
Rectal Pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
constipation
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Gastrointestinal disorders
Anorexia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
General disorders
Fever
|
0.00%
0/1 • Up to 2 years
|
40.0%
2/5 • Number of events 2 • Up to 2 years
|
33.3%
2/6 • Number of events 2 • Up to 2 years
|
16.7%
2/12 • Number of events 5 • Up to 2 years
|
33.3%
4/12 • Number of events 5 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
General disorders
Dysuria
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
33.3%
4/12 • Number of events 5 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 4 • Up to 2 years
|
|
General disorders
Fatigue
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
50.0%
3/6 • Number of events 3 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
General disorders
Chills
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
General disorders
Epistaxis
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
General disorders
Hypothermia
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
General disorders
Limb edema
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Hepatobiliary disorders
Blast crisis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
33.3%
2/6 • Number of events 2 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 2 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Oral candidiasis (Thrush)
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Streptococcus mitis bacteremia
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 2 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
E coli bacteremia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Lung Infection
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Sinusitis
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Colonic fistula
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Clostridioides difficile
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
Tooth infection
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
rhinovirus
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
BK virus
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
HSV1
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Coag negative staphylococcus aureus bacteremia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Epstein-Barr virus
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
parainfluenza
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
fungal pneumonia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
invasive fungal infection
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Herpes simplex reactivation
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 2 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Pneumonia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Folliculitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
HHV6 Viremia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/1 • Up to 2 years
|
40.0%
2/5 • Number of events 2 • Up to 2 years
|
33.3%
2/6 • Number of events 3 • Up to 2 years
|
25.0%
3/12 • Number of events 3 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
33.3%
2/6 • Number of events 3 • Up to 2 years
|
25.0%
3/12 • Number of events 3 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Investigations
Alkaline phosphatase increased
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 2 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Metabolism and nutrition disorders
Hypernatremia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Neck Pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Other- Thigh pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Muscle spasm
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
Nervous system disorders
Headache
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
50.0%
3/6 • Number of events 6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
Nervous system disorders
Insomnia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
6.2%
2/32 • Number of events 2 • Up to 2 years
|
|
Nervous system disorders
Anosmia
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Nervous system disorders
dizziness
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Nervous system disorders
encephalopathy
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
33.3%
2/6 • Number of events 2 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
6.2%
2/32 • Number of events 2 • Up to 2 years
|
|
Psychiatric disorders
Hallucinations
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Psychiatric disorders
Depression
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/1 • Up to 2 years
|
40.0%
2/5 • Number of events 3 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Renal and urinary disorders
Cystitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Reproductive system and breast disorders
Vaginal pruritis
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Reproductive system and breast disorders
Vaginal bleeding
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Chylothorax
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Cough
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Hiccups
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Vascular disorders
Hematoma
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Vascular disorders
Generalized edema
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Vascular disorders
DVT
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
swelling
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
cyst
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Acute skin GVHD
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
6.2%
2/32 • Number of events 3 • Up to 2 years
|
|
Infections and infestations
Aspergillus
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Infections and infestations
bacteremia
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Infections and infestations
Cytomegalovirus (CMV) infection
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
16.7%
2/12 • Number of events 2 • Up to 2 years
|
16.7%
2/12 • Number of events 2 • Up to 2 years
|
9.4%
3/32 • Number of events 3 • Up to 2 years
|
|
General disorders
syncope
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Hepatobiliary disorders
Blood bilirubin increase
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Erythematous rash
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 1 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/1 • Up to 2 years
|
40.0%
2/5 • Number of events 3 • Up to 2 years
|
16.7%
1/6 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
12.5%
4/32 • Number of events 6 • Up to 2 years
|
|
Endocrine disorders
hyperparathyroidism
|
0.00%
0/1 • Up to 2 years
|
0.00%
0/5 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
8.3%
1/12 • Number of events 1 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/32 • Up to 2 years
|
|
Blood and lymphatic system disorders
platelet count decreased
|
0.00%
0/1 • Up to 2 years
|
20.0%
1/5 • Number of events 3 • Up to 2 years
|
0.00%
0/6 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
0.00%
0/12 • Up to 2 years
|
3.1%
1/32 • Number of events 1 • Up to 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place