Trial Outcomes & Findings for Allogeneic Hematopoietic Stem Cell Transplantation With Reduced Intensity Pre-transplant Conditioning for the Treatment of High-risk Hematological Malignancies (V3) (NCT NCT01139164)
NCT ID: NCT01139164
Last Updated: 2018-07-11
Results Overview
Number of subject deaths prior to day 100.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
78 participants
Primary outcome timeframe
8 years
Results posted on
2018-07-11
Participant Flow
Participant milestones
| Measure |
Group 1: Regimen A
Regimen A: Chronic Lymphocytic Leukemia/Chronic Prolymphocytic Leukemia/Multiple Myeloma
|
Group 2: Regimen B
Other Malignancies not addressed in regimens A and C
|
Group 3: Regimen C
B-Cell Lymphomas
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
67
|
7
|
|
Overall Study
COMPLETED
|
4
|
65
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
0
|
Reasons for withdrawal
| Measure |
Group 1: Regimen A
Regimen A: Chronic Lymphocytic Leukemia/Chronic Prolymphocytic Leukemia/Multiple Myeloma
|
Group 2: Regimen B
Other Malignancies not addressed in regimens A and C
|
Group 3: Regimen C
B-Cell Lymphomas
|
|---|---|---|---|
|
Overall Study
Withdrawn prior to study start
|
0
|
2
|
0
|
Baseline Characteristics
Allogeneic Hematopoietic Stem Cell Transplantation With Reduced Intensity Pre-transplant Conditioning for the Treatment of High-risk Hematological Malignancies (V3)
Baseline characteristics by cohort
| Measure |
Group 1: Regimen A
n=4 Participants
Regimen A: Chronic Lymphocytic Leukemia/Chronic Prolymphocytic Leukemia/Multiple Myeloma
|
Group 2: Regimen B
n=67 Participants
Other Malignancies not addressed in regimens A and C
|
Group 3: Regimen C
n=7 Participants
B-Cell Lymphomas
|
Total
n=78 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
44 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
54 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
24 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
27 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
43 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
51 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
67 participants
n=7 Participants
|
7 participants
n=5 Participants
|
78 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 8 yearsPopulation: For regimen B, only the subjects who participated in study intervention were analyzed for outcome measures.
Number of subject deaths prior to day 100.
Outcome measures
| Measure |
Group 1: Regimen A
n=4 Participants
Regimen A: Chronic Lymphocytic Leukemia/Chronic Prolymphocytic Leukemia/Multiple Myeloma
|
Group 2: Regimen B
n=65 Participants
Other Malignancies not addressed in regimens A and C
|
Group 3: Regimen C
n=7 Participants
B-Cell Lymphomas
|
|---|---|---|---|
|
To Determine the Treatment-related Mortality Rate of Allogeneic Stem Cell Transplants Using Reduced-intensity Conditioning Regimens Within 1st 100-days.
|
0 participants
|
18 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Day +100Population: The outcome measure data below only accounts for the number of subjects who met the engraftment criteria prior at day +30; the chimerism data was not collected. For regimen B, only the subjects who participated in study intervention were analyzed for outcome measures.
To determine the engraftment rate of allogeneic stem cell transplants using reduced-intensity conditioning regimens. It will be measured as the proportion of subjects meeting criteria for engraftment before day +30 and full donor chimerism demonstrated before or at day +100. Engraftment is defined by maintenance of ANC \> 500/mm3 for at least 3 consecutive days and platelet count \> 20,000/mm3 for 3 consecutive days in absence of platelet transfusion. These criteria must have been met before Day +30. Chimerism is the pressence of donor cells and will be analyzed by FISH for sex-mismatched donor-recipient pairs and VNTR analysis for sex-mathced pairs. Chimerism by Day +100 will be documented for this outcome
Outcome measures
| Measure |
Group 1: Regimen A
n=4 Participants
Regimen A: Chronic Lymphocytic Leukemia/Chronic Prolymphocytic Leukemia/Multiple Myeloma
|
Group 2: Regimen B
n=65 Participants
Other Malignancies not addressed in regimens A and C
|
Group 3: Regimen C
n=7 Participants
B-Cell Lymphomas
|
|---|---|---|---|
|
To Determine the Engraftment Rate of Allogeneic Stem Cell Transplants
|
3 participants
|
46 participants
|
6 participants
|
SECONDARY outcome
Timeframe: 100 daysPopulation: This study terminated early; funding ran out before the analysis could be completed.
To determine the morbidity, including the pattern and severity of complications, of allogeneic stem cell transplants using reduced-intensity conditioning regimens. The average number of days spent in the hospital until day +100 will be reported as a surrogate for morbidity and complications.
Outcome measures
Outcome data not reported
Adverse Events
Group 1: Regimen A
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Group 2: Regimen B
Serious events: 32 serious events
Other events: 0 other events
Deaths: 0 deaths
Group 3: Regimen C
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group 1: Regimen A
n=4 participants at risk
Regimen A: Chronic Lymphocytic Leukemia/Chronic Prolymphocytic Leukemia/Multiple Myeloma
|
Group 2: Regimen B
n=67 participants at risk
Other Malignancies not addressed in regimens A and C
|
Group 3: Regimen C
n=7 participants at risk
B-Cell Lymphomas
|
|---|---|---|---|
|
Nervous system disorders
altered mental status
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Infections and infestations
GVHD
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
22.4%
15/67 • Number of events 15 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
3.0%
2/67 • Number of events 2 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Vascular disorders
hypotension
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Respiratory, thoracic and mediastinal disorders
respiratory arrest
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
3.0%
2/67 • Number of events 2 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Gastrointestinal disorders
gastrointestinal hemorrhage
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
3.0%
2/67 • Number of events 2 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Infections and infestations
sepsis
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Gastrointestinal disorders
diarrhea
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Gastrointestinal disorders
nausea
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
3.0%
2/67 • Number of events 2 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Nervous system disorders
anoxic brain damage
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Respiratory, thoracic and mediastinal disorders
aspiration
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Infections and infestations
TA-TMA
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
Infections and infestations
febrile neutropenia
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
|
General disorders
fever
|
0.00%
0/4 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
1.5%
1/67 • Number of events 1 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
0.00%
0/7 • Start of treatment until end of study
repotable adverse events included: any deaths occuring on study, any grade IV non-hematological toxicity until 1 year post transplant or 6 months after the last donor lymphocyte infusion (whichever is latest), severe (grade III or IV) acute graft versus host disease (GVHD) any unexpected adverse events
|
Other adverse events
Adverse event data not reported
Additional Information
Dr. Robert K Stuart
Medical University of South Carolina
Phone: 843-792-4271
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place