Trial Outcomes & Findings for Adoptive TReg Cell for Suppression of aGVHD After UCB HSCT for Heme Malignancies (NCT NCT02991898)

NCT ID: NCT02991898

Last Updated: 2020-09-29

Results Overview

Count of patients who survived 2 years post intervention

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2020-09-29

Participant Flow

Participant milestones

Measure	Treg Infusion The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Adoptive TReg Cell for Suppression of aGVHD After UCB HSCT for Heme Malignancies

Baseline characteristics by cohort

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Count of patients who survived 2 years post intervention

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants Survived	1 Participants

SECONDARY outcome

Timeframe: Assessed weekly until day 100, then day 180, 360

Probability of grade II-IV aGVHD

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants With Grade II-IV aGVHD	2 Participants

SECONDARY outcome

Timeframe: 6 months

Evaluated with descriptive statistics and plots or cumulative incidence curves if enough evaluable patients are available for time-to-event endpoints.

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants Experiencing Treatment Related Mortality (TRM)	0 Participants

SECONDARY outcome

Timeframe: 1 year

Evaluated with descriptive statistics and plots or cumulative incidence curves if enough evaluable patients are available for time-to-event endpoints.

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants Who Experienced Relapse	1 Participants

SECONDARY outcome

Timeframe: 1 year

Evaluated with descriptive statistics and plots or cumulative incidence curves if enough evaluable patients are available for time-to-event endpoints.

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants With Incidence of Bacterial, Viral and Fungal Infections Bacterial	1 Participants
Number of Participants With Incidence of Bacterial, Viral and Fungal Infections Viral	2 Participants
Number of Participants With Incidence of Bacterial, Viral and Fungal Infections Fungal	0 Participants

SECONDARY outcome

Timeframe: 14 days

Population: Samples were not analyzed, and so data not available for reporting.

The proportion of patients with detectable Treg cells at day 14 post infusion

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Assessed at Day 4, weekly for 8 weeks

Population: Samples were not analyzed, and so data not available for reporting.

The proportion of patients with immune reconstitution. Continuous endpoints will be described by medians, ranges and interquartile ranges as well as means and standard deviations if normally distributed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 48 hours post infusion

Incidence of Adverse Events

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants Experiencing Treg Cell Infusion Toxicity	2 Participants

SECONDARY outcome

Timeframe: 24 hours post infusion

Population: Samples were not analyzed, and so data not available for reporting.

Length of Treg survival after infusion of Treg.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day +100

The incidence of chimerism in patients treated

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Percentage of Donor Cell Chimerism	72 percentage of donor cells Interval 50.0 to 100.0

SECONDARY outcome

Timeframe: 1 year

The probability of survival, one year post-treatment

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants Survived One Year Post-transplant	2 Participants

SECONDARY outcome

Timeframe: Day 42

The incidence of neutrophil recovery, that is return of neutrophil counts to ≥ 5 X 10^8/L in treated patients

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants With Neutrophil Recovery	2 Participants

SECONDARY outcome

Timeframe: 1 year

The incidence of platelet recovery (return of platelet counts to > 20,000/μL) in treated patients

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants With Platelet Recovery	2 Participants

SECONDARY outcome

Timeframe: 1 year

The incidence of chronic GVHD in treated patients after one year

Outcome measures

Measure	Treg Infusion n=3 Participants The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Number of Participants With Chronic GVHD	0 Participants

Adverse Events

Treg Infusion

Serious events: 2 serious events

Other events: 2 other events

Deaths: 2 deaths

Serious adverse events

Measure	Treg Infusion n=3 participants at risk The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Vascular disorders Capillary leak syndrome	33.3% 1/3 • Number of events 1 • 1 week
Vascular disorders Cytokine release syndrome	33.3% 1/3 • Number of events 1 • 1 week
Immune system disorders Graft Versus Host Disease	66.7% 2/3 • Number of events 2 • 1 week

Other adverse events

Measure	Treg Infusion n=3 participants at risk The Treg cell infusion is given no sooner than 1 hour, but within 24 hours after the 2nd cord blood infusion
Cardiac disorders Hypertension	33.3% 1/3 • Number of events 7 • 1 week
General disorders Chills	33.3% 1/3 • Number of events 2 • 1 week
General disorders Edema Limbs	33.3% 1/3 • Number of events 4 • 1 week
General disorders Fatigue	33.3% 1/3 • Number of events 2 • 1 week
General disorders Febrile neutropenia	33.3% 1/3 • Number of events 3 • 1 week
General disorders Fever	33.3% 1/3 • Number of events 2 • 1 week

Additional Information

Claudio Brunstein

Masonic Cancer Center, University of Minnesota

Phone: 612-625-3918

Email: bruns072@umn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place