Trial Outcomes & Findings for Efficacy and Safety of a Donor Lymphocyte Preparation Depleted of Functional Host Alloreactive T-cells (ATIR) in Patients Undergoing a Peripheral Blood Stem Cell Transplant From a Related, Haploidentical Donor (NCT NCT00967343)
NCT ID: NCT00967343
Last Updated: 2021-06-14
Results Overview
TRM is defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide)
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
40 participants
Primary outcome timeframe
6, 12 and 24 months after the transplantation
Results posted on
2021-06-14
Participant Flow
Participant milestones
| Measure |
ATIR
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Overall Study
STARTED
|
40
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
38
|
Reasons for withdrawal
| Measure |
ATIR
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Overall Study
Study termination by sponsor
|
12
|
|
Overall Study
Death
|
24
|
|
Overall Study
Relapse
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Efficacy and Safety of a Donor Lymphocyte Preparation Depleted of Functional Host Alloreactive T-cells (ATIR) in Patients Undergoing a Peripheral Blood Stem Cell Transplant From a Related, Haploidentical Donor
Baseline characteristics by cohort
| Measure |
ATIR
n=40 Participants
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Age, Continuous
|
45 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 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
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
36 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
|
|
Hematologic malignancy
Acute lymphatic leukemia
|
5 Participants
n=5 Participants
|
|
Hematologic malignancy
Acute myeloid leukemia
|
22 Participants
n=5 Participants
|
|
Hematologic malignancy
Chronic myeloid leukemia
|
1 Participants
n=5 Participants
|
|
Hematologic malignancy
Myelodysplastic syndrome
|
3 Participants
n=5 Participants
|
|
Hematologic malignancy
Myeloproliferative syndrome
|
1 Participants
n=5 Participants
|
|
Hematologic malignancy
Other acute leukemia
|
2 Participants
n=5 Participants
|
|
Hematologic malignancy
Other leukemia
|
3 Participants
n=5 Participants
|
|
Hematologic malignancy
Plasma cell disorder
|
3 Participants
n=5 Participants
|
|
Donor HLA compatibility (HLA-A, -B, -DR)
Donor HLA compatibility (HLA-A, -B, -DR): 3/6
|
27 Participants
n=5 Participants
|
|
Donor HLA compatibility (HLA-A, -B, -DR)
Donor HLA compatibility (HLA-A, -B, -DR): 4/6
|
13 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6, 12 and 24 months after the transplantationTRM is defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide)
Outcome measures
| Measure |
ATIR
n=40 Participants
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Transplant Related Mortality
TRM at 6 months post HSCT
|
33 Kaplan-Meier estimates (%)
|
|
Transplant Related Mortality
TRM at 24 months post HSCT
|
71 Kaplan-Meier estimates (%)
|
|
Transplant Related Mortality
TRM at 12 months post HSCT
|
56 Kaplan-Meier estimates (%)
|
SECONDARY outcome
Timeframe: Up to 24 months after the transplantationGVHD was graded according to standard criteria as referred to in the reference module (Filipovich et al. 2005; Przepiorka et al. 1995).
Outcome measures
| Measure |
ATIR
n=40 Participants
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Incidence and Severity Graft-versus-host Disease (GVHD)
Grade II acute GVHD (moderate)
|
7.5 Percentage of participants
|
|
Incidence and Severity Graft-versus-host Disease (GVHD)
Grade III (moderate) or IV (severe) acute GVHD
|
12.5 Percentage of participants
|
|
Incidence and Severity Graft-versus-host Disease (GVHD)
Severe chronic GVHD
|
5 Percentage of participants
|
SECONDARY outcome
Timeframe: Up to 24 months after the transplantationPopulation: Data were not collected, premature termination of study
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 24 months after the transplantationPopulation: Data not collected, premature termination of study
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 24 months after the transplantationPopulation: Data were not collected, premature termination of study
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 24 months after the transplantationPopulation: Data were not collected, premature termination of study
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6, 12, and 24 months after the transplantationOutcome measures
| Measure |
ATIR
n=40 Participants
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Overall Survival
OS 6 months after the HSCT
|
65 Kaplan-Meier estimates (%)
|
|
Overall Survival
OS 12 months after the HSCT
|
33 Kaplan-Meier estimates (%)
|
|
Overall Survival
OS 24 months after the HSCT
|
22 Kaplan-Meier estimates (%)
|
Adverse Events
ATIR
Serious events: 9 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ATIR
n=40 participants at risk
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Immune system disorders
Acute graft versus host disease
|
7.5%
3/40
|
|
Immune system disorders
Chronic graft versus host disease
|
2.5%
1/40
|
|
Infections and infestations
Bacterial sepsis
|
2.5%
1/40
|
|
Infections and infestations
Encephalitis
|
2.5%
1/40
|
|
Infections and infestations
JC virus infection
|
2.5%
1/40
|
|
Infections and infestations
Urinary tract infection
|
2.5%
1/40
|
|
Infections and infestations
Viral infection
|
2.5%
1/40
|
|
Gastrointestinal disorders
Abdominal pain
|
2.5%
1/40
|
|
Gastrointestinal disorders
Ascites
|
2.5%
1/40
|
|
Gastrointestinal disorders
Diarrhoea
|
2.5%
1/40
|
|
Gastrointestinal disorders
Nausea
|
2.5%
1/40
|
|
Hepatobiliary disorders
Hepatitis failure
|
2.5%
1/40
|
|
Hepatobiliary disorders
Hepatitis toxic
|
2.5%
1/40
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.5%
1/40
|
|
General disorders
Pain
|
2.5%
1/40
|
|
General disorders
Pyrexia
|
2.5%
1/40
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.5%
1/40
|
Other adverse events
| Measure |
ATIR
n=40 participants at risk
Donor lymphocyte preparation depleted of host functional alloreactive T-cells: Single intravenous infusion with 2x10E6 T-cells/kg
|
|---|---|
|
Immune system disorders
Acute graft versus host disease
|
10.0%
4/40
|
|
Infections and infestations
Cytomegalovirus infection
|
5.0%
2/40
|
|
Gastrointestinal disorders
abdominal pain
|
5.0%
2/40
|
|
Gastrointestinal disorders
Diarrhoea
|
10.0%
4/40
|
|
Gastrointestinal disorders
Vomiting
|
7.5%
3/40
|
|
Gastrointestinal disorders
Nausea
|
7.5%
3/40
|
|
Gastrointestinal disorders
Dry mouth
|
5.0%
2/40
|
Additional Information
Andrew Sandler, MD / Chief Medical Officer
Kiadis Pharma Netherlands B.V.
Phone: +1 206 779 9213
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place