Trial Outcomes & Findings for Use of T-allo10 in Hematopoietic Stem Cell Transplantation (HSCT) for Blood Disorders (NCT NCT03198234)

NCT ID: NCT03198234

Last Updated: 2024-07-18

Results Overview

Number of participants experiencing TEAEs. Assessments of TEAE will include laboratory abnormalities, changes in vital signs, and changes in physical examination related to the infusion of T-allo10 cells in order to assess the tolerability of T-allo10.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

5 participants

Primary outcome timeframe

Time of T-allo10 cell infusion until 28 days following the infusion.

Results posted on

2024-07-18

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Overall Study STARTED	3	2
Overall Study COMPLETED	3	1
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 3 Participants receive 9 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Overall Study Relapse of primary disease	0	1	0

Baseline Characteristics

Use of T-allo10 in Hematopoietic Stem Cell Transplantation (HSCT) for Blood Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant).	Cohort 2 n=2 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant).	Total n=5 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Diagnosis Acute Myeloid Leukemia (AML)	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Diagnosis Acute Lymphoid Leukemia (ALL)	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Time of T-allo10 cell infusion until 28 days following the infusion.

Population: One participant who had no post-transplant data is excluded from analysis.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Participants Experiencing Treatment Emergent Adverse Events (TEAE)	1 Participants	0 Participants

PRIMARY outcome

Timeframe: Time of T-allo10 cell infusion until 28 days following the infusion.

Population: One participant who had no post-transplant data is excluded from analysis.

Number of participants experiencing TEAEs related to infusion, by severity graded according to the CTCAE grading system, from grade 1 (least severe) to grade 5 (death). Assessments of TEAE will include laboratory abnormalities, changes in vital signs, and changes in physical examination following infusion of T-allo10 cells in order to assess the safety of T-allo10.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Severity of Treatment Emergent Adverse Events (TEAE) Grade 1	1 Participants	0 Participants
Severity of Treatment Emergent Adverse Events (TEAE) >=Grade 2	0 Participants	0 Participants

PRIMARY outcome

Timeframe: +42 days post HSCT

Population: One participant who had no post-transplant data is excluded from analysis.

Stem cell engraftment is evaluated by clinical laboratory studies including absolute neutrophil count above 500/mm3 for three consecutive days, hematopoiesis at bone marrow examination, with cellularity \>5 % and donor chimerism \>90% by short tandem repeat (STR) analysis for the presence of donor cells, and minimal residual disease (MRD) assay \< 0.1%.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Participants Who Achieved Stem Cell Engraftment After Hematopoietic Stem Cell Transplant (HSCT).	3 Participants	1 Participants

PRIMARY outcome

Timeframe: By Day -2

Feasibility defined by the rate of successful manufacture of the T-allo10 product to satisfy the targeted dose level and meet the required release specifications. Number of products meeting specifications out of total products manufactured is reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Products Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=2 Products Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Successful T-allo10 Products Manufactured for Patients Enrolled	3 Products	0 Products

SECONDARY outcome

Timeframe: Study visits through Day +100

Population: One participant who had no post-transplant data is excluded from analysis.

The number of patients who experienced grade III and IV acute GvHD at Day +100 following infusion of Tallo10 cells, assessed using the Modified Keystone scale administered by an independent evaluator on study visits through Day +100

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Participants Who Experienced Grade III and/or IV Acute GvHD	2 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: After Day +100 through Day +365

Population: One participant who had no post-transplant data is excluded from analysis.

The number of participants who experienced chronic GvHD and severity will be assessed by an independent evaluator. Outcome is reported as the highest level of chronic GVHD reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Patients Who Developed Chronic GvHD Mild	2 Participants	0 Participants
Number of Patients Who Developed Chronic GvHD Moderate	1 Participants	1 Participants
Number of Patients Who Developed Chronic GvHD Severe	0 Participants	0 Participants
Number of Patients Who Developed Chronic GvHD Overall Incidence	3 Participants	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to Day 365

Population: One participant who had no post-transplant data is excluded from analysis.

Immune reconstitution will be evaluated by clinical laboratory studies of CD3+ T cells, assessed by the number of days to reach \>200/microliter CD3+ T cells.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Days to Reach Immune Reconstitution	22 Days Interval 21.0 to 28.0	56 Days Interval 56.0 to 56.0

OTHER_PRE_SPECIFIED outcome

Timeframe: At Day +365

Population: One participant who had no post-transplant data is excluded from analysis.

Disease free survival is defined as the absence of minimal residual disease in the bone marrow. The investigators will use bone marrow aspirate examination, minimal residual disease (MRD) assay, and donor chimerism by STR analysis to evaluate disease free survival.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)	Cohort 2 n=1 Participants Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant)
Number of Participants Who Experienced Disease Free Survival	3 Participants	1 Participants

Adverse Events

Cohort 1

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

Cohort 2

Serious events: 2 serious events

Other events: 2 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1 n=3 participants at risk Participants receive 1 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant).	Cohort 2 n=2 participants at risk Participants receive 3 X 10\^6/kg (± 10%) T-allo10 cells infused intravenously on Day -1 (day before transplant).
Renal and urinary disorders Acute kidney injury	33.3% 1/3 • Adverse events were recorded up to 1 year post-transplant.	0.00% 0/2 • Adverse events were recorded up to 1 year post-transplant.
Respiratory, thoracic and mediastinal disorders Cough	33.3% 1/3 • Adverse events were recorded up to 1 year post-transplant.	0.00% 0/2 • Adverse events were recorded up to 1 year post-transplant.
Vascular disorders Duodenal hematoma	33.3% 1/3 • Adverse events were recorded up to 1 year post-transplant.	0.00% 0/2 • Adverse events were recorded up to 1 year post-transplant.
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/3 • Adverse events were recorded up to 1 year post-transplant.	50.0% 1/2 • Adverse events were recorded up to 1 year post-transplant.
Blood and lymphatic system disorders Febrile neutropenia	33.3% 1/3 • Adverse events were recorded up to 1 year post-transplant.	0.00% 0/2 • Adverse events were recorded up to 1 year post-transplant.
General disorders Fever	0.00% 0/3 • Adverse events were recorded up to 1 year post-transplant.	100.0% 2/2 • Adverse events were recorded up to 1 year post-transplant.
Gastrointestinal disorders Nausea	0.00% 0/3 • Adverse events were recorded up to 1 year post-transplant.	50.0% 1/2 • Adverse events were recorded up to 1 year post-transplant.
Investigations Neutrophil count decreased	0.00% 0/3 • Adverse events were recorded up to 1 year post-transplant.	50.0% 1/2 • Adverse events were recorded up to 1 year post-transplant.
Infections and infestations Sepsis	33.3% 1/3 • Adverse events were recorded up to 1 year post-transplant.	0.00% 0/2 • Adverse events were recorded up to 1 year post-transplant.
Infections and infestations Viremia: RSV	33.3% 1/3 • Adverse events were recorded up to 1 year post-transplant.	0.00% 0/2 • Adverse events were recorded up to 1 year post-transplant.

Other adverse events

Additional Information

Rajni Agarwal, MD

Stanford

Phone: 650-725-9250

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place