Trial Outcomes & Findings for T-Regulatory Cell Kinetics, Stem Cell Transplantation, REGKINE (NCT NCT00578461)
NCT ID: NCT00578461
Last Updated: 2016-04-22
Results Overview
The investigative intent is to determine the changes in numbers and function of the regulatory cell population using the best methods to measure this cell population. The frequency of T cells will be summarized at baseline and each time point of follow-up.
Recruitment status
TERMINATED
Study phase
NA
Target enrollment
26 participants
Primary outcome timeframe
1 Year
Results posted on
2016-04-22
Participant Flow
Participant milestones
| Measure |
Stem Cell Transplant
All patients will receive stem cell transplantation conditioning, GVHD prevention and stem cell infusion.
Conditioning includes: Ara C, Cyclophosphamide, MESNA, TBI-Total Body Irradiation. Ara C: 3000 mg/m\^2 - pts will receive via IV every 12 hours for 6 doses starting at 20:00 hours on day -8.Mesna: 45 mg/kg; divided into 5 doses-will be administered 15 minutes prior to Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide.
Cyclophosphamide: 45 mg/kg; IV once daily on day -7 and day -6 starting at 1400 hours.TBI-Total Body Irradiation: Total dose 12 Gy, will be delivered in 8 fractions of 150 cGy, each, two fractions per day beginning day -4 to day -1. Stem cell Infusion is on day 0.
|
|---|---|
|
Overall Study
STARTED
|
26
|
|
Overall Study
COMPLETED
|
26
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
T-Regulatory Cell Kinetics, Stem Cell Transplantation, REGKINE
Baseline characteristics by cohort
| Measure |
Stem Cell Transplant
n=26 Participants
All patients will receive stem cell transplantation conditioning, GVHD prevention and stem cell infusion.
|
|---|---|
|
Age, Continuous
|
9.7 years
STANDARD_DEVIATION 4.5 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
26 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 YearPopulation: Only 20 of the 26 patients enrolled were included in this analysis as only 20 patients have Treg values at 1 year.
The investigative intent is to determine the changes in numbers and function of the regulatory cell population using the best methods to measure this cell population. The frequency of T cells will be summarized at baseline and each time point of follow-up.
Outcome measures
| Measure |
Stem Cell Transplant
n=20 Participants
All patients will be receiving a stem cell transplant on study. Conditioning includes: Ara C, Cyclophosphamide, MESNA, TBI-Total Body Irradiation.
|
|---|---|
|
Median Percentage of Treg Cells at 1 Year Post Transplant
|
4.1 percentage of total CD4+ cells
Interval 3.1 to 6.4
|
Adverse Events
Stem Cell Transplant
Serious events: 15 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Stem Cell Transplant
n=26 participants at risk
All patients will receive stem cell transplantation conditioning, GVHD prevention and stem cell infusion.
Conditioning includes: Ara C, Cyclophosphamide, MESNA, TBI-Total Body Irradiation. Ara C: 3000 mg/m\^2 - pts will receive via IV every 12 hours for 6 doses starting at 20:00 hours on day -8.Mesna: 45 mg/kg; divided into 5 doses-will be administered 15 minutes prior to Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide. Cyclophosphamide: 45 mg/kg; IV once daily on day -7 and day -6 starting at 1400 hours.TBI-Total Body Irradiation: Total dose 12 Gy, will be delivered in 8 fractions of 150 cGy, each, two fractions per day beginning day -4 to day -1. Stem cell Infusion is on day 0.
|
|---|---|
|
Immune system disorders
Allergic reaction/hypersensitivity (including drug fever)
|
3.8%
1/26 • Number of events 1
|
|
Cardiac disorders
Hypertension
|
3.8%
1/26 • Number of events 1
|
|
Cardiac disorders
Pericardial effusion (non-malignant)
|
7.7%
2/26 • Number of events 3
|
|
Vascular disorders
Coagulation - Other: Severe coagulopathy
|
3.8%
1/26 • Number of events 1
|
|
General disorders
Fever
|
3.8%
1/26 • Number of events 2
|
|
General disorders
Death
|
3.8%
1/26 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
3.8%
1/26 • Number of events 1
|
|
Gastrointestinal disorders
Dehydration
|
3.8%
1/26 • Number of events 1
|
|
Hepatobiliary disorders
Liver dysfunction/failure (clinical)
|
7.7%
2/26 • Number of events 3
|
|
Infections and infestations
Febrile neutropenia
|
3.8%
1/26 • Number of events 1
|
|
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Catheter-related
|
3.8%
1/26 • Number of events 2
|
|
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Vein
|
3.8%
1/26 • Number of events 1
|
|
Infections and infestations
Infection - Other: only clinical signs of infection
|
3.8%
1/26 • Number of events 1
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Blood
|
7.7%
2/26 • Number of events 2
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Catheter-related
|
19.2%
5/26 • Number of events 6
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Nose
|
3.8%
1/26 • Number of events 1
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Upper airway NOS
|
3.8%
1/26 • Number of events 1
|
|
Metabolism and nutrition disorders
AST, SGOT
|
3.8%
1/26 • Number of events 1
|
|
General disorders
Pain - Abdomen NOS
|
3.8%
1/26 • Number of events 1
|
|
General disorders
Pain - Head/headache
|
3.8%
1/26 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.8%
1/26 • Number of events 1
|
|
Renal and urinary disorders
Renal failure
|
11.5%
3/26 • Number of events 4
|
|
Vascular disorders
Portal vein flow
|
3.8%
1/26 • Number of events 1
|
Other adverse events
| Measure |
Stem Cell Transplant
n=26 participants at risk
All patients will receive stem cell transplantation conditioning, GVHD prevention and stem cell infusion.
Conditioning includes: Ara C, Cyclophosphamide, MESNA, TBI-Total Body Irradiation. Ara C: 3000 mg/m\^2 - pts will receive via IV every 12 hours for 6 doses starting at 20:00 hours on day -8.Mesna: 45 mg/kg; divided into 5 doses-will be administered 15 minutes prior to Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide. Cyclophosphamide: 45 mg/kg; IV once daily on day -7 and day -6 starting at 1400 hours.TBI-Total Body Irradiation: Total dose 12 Gy, will be delivered in 8 fractions of 150 cGy, each, two fractions per day beginning day -4 to day -1. Stem cell Infusion is on day 0.
|
|---|---|
|
Vascular disorders
PTT
|
7.7%
2/26 • Number of events 3
|
|
Gastrointestinal disorders
Diarrhea
|
15.4%
4/26 • Number of events 4
|
|
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam) - Oral cavity
|
11.5%
3/26 • Number of events 3
|
|
Gastrointestinal disorders
Nausea
|
7.7%
2/26 • Number of events 2
|
|
Gastrointestinal disorders
Vomiting
|
15.4%
4/26 • Number of events 5
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Catheter-related
|
11.5%
3/26 • Number of events 3
|
|
Metabolism and nutrition disorders
ALT, SGPT
|
7.7%
2/26 • Number of events 2
|
|
Metabolism and nutrition disorders
AST, SGOT
|
15.4%
4/26 • Number of events 6
|
|
Metabolism and nutrition disorders
Bilirubin (hyperbilirubinemia)
|
7.7%
2/26 • Number of events 3
|
|
Metabolism and nutrition disorders
Calcium, serum-low (hypocalcemia)
|
7.7%
2/26 • Number of events 2
|
|
Metabolism and nutrition disorders
GGT
|
7.7%
2/26 • Number of events 2
|
|
Metabolism and nutrition disorders
Glucose, serum-high (hyperglycemia)
|
7.7%
2/26 • Number of events 2
|
|
Metabolism and nutrition disorders
Sodium, serum-low (hyponatremia)
|
7.7%
2/26 • Number of events 2
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place