Trial Outcomes & Findings for T-Reg Cell Kinetics, Stem Cell Transplant, REGALE (NCT NCT00578539)

NCT ID: NCT00578539

Last Updated: 2016-04-22

Results Overview

To define the biologic recovery and behavior of T regulatory cells for patients undergoing stem cell transplantation as specified in this protocol

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

24 participants

Primary outcome timeframe

1 year

Results posted on

2016-04-22

Participant Flow

Participant milestones

Participant milestones
Measure
Stem Cell Transplant
All patients will receive Ara C IV every 12 hours for 6 doses starting at 1400 hours on day -8. Cyclophosphamide IV once daily on day -7 and day -6 starting at 1400 hours. MESNA will be administered 15 minutes prior to each dose of Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide. Campath 1h will be given on day -4, day -3, day -2 and day-1. TBI (Total Body Irradiation) will be delivered in 8 fractions of 1.75 Gy in two fractions on day -4, day -3, day -2, and day -1. Stem cell Infusion are infused on day 0.
Overall Study
STARTED
24
Overall Study
COMPLETED
24
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

T-Reg Cell Kinetics, Stem Cell Transplant, REGALE

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Stem Cell Transplant
n=24 Participants
All patients will receive stem cell transplantation conditioning, GVHD prevention and stem cell infusion.
Age, Continuous
10.0 years
STANDARD_DEVIATION 4.9 • n=5 Participants
Age, Categorical
<=18 years
23 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
1 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Sex: Female, Male
Female
7 Participants
n=5 Participants
Sex: Female, Male
Male
17 Participants
n=5 Participants
Region of Enrollment
United States
24 participants
n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: Only 13 of the 24 patients enrolled were included in this analysis as only 13 patients have Treg values at 1 year.

To define the biologic recovery and behavior of T regulatory cells for patients undergoing stem cell transplantation as specified in this protocol

Outcome measures

Outcome measures
Measure
Stem Cell Transplant
n=13 Participants
All patients will receive Ara C IV every 12 hours for 6 doses starting at 1400 hours on day -8. Cyclophosphamide IV once daily on day -7 and day -6 starting at 1400 hours. MESNA will be administered 15 minutes prior to each dose of Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide. Campath 1h will be given on day -4, day -3, day -2 and day-1. TBI (Total Body Irradiation) will be delivered in 8 fractions of 1.75 Gy in two fractions on day -4, day -3, day -2, and day -1. Stem cell Infusion are infused on day 0.
Median Percentage of Treg Cells at 1 Year Post Transplant
6.9 percentage of total CD4+ cells
Interval 4.6 to 13.2

Adverse Events

Stem Cell Transplant

Serious events: 17 serious events
Other events: 13 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Stem Cell Transplant
n=24 participants at risk
All patients will receive Ara C IV every 12 hours for 6 doses starting at 1400 hours on day -8. Cyclophosphamide IV once daily on day -7 and day -6 starting at 1400 hours. MESNA will be administered 15 minutes prior to each dose of Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide. Campath 1h will be given on day -4, day -3, day -2 and day-1. TBI (Total Body Irradiation) will be delivered in 8 fractions of 1.75 Gy in two fractions on day -4, day -3, day -2, and day -1. Stem cell Infusion are infused on day 0.
Blood and lymphatic system disorders
Hemolysis (e.g., immune hemolytic anemia, drug-related hemolysis)
4.2%
1/24 • Number of events 1
Cardiac disorders
Cardiopulmonary arrest, cause unknown (non-fatal)
4.2%
1/24 • Number of events 2
Cardiac disorders
Pericardial effusion (non-malignant)
8.3%
2/24 • Number of events 2
General disorders
Death
4.2%
1/24 • Number of events 1
Skin and subcutaneous tissue disorders
Rash/desquamation
4.2%
1/24 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary/upper respiratory - Bronchopulmonary NOS
4.2%
1/24 • Number of events 1
Hepatobiliary disorders
Liver dysfunction/failure (clinical)
4.2%
1/24 • Number of events 1
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Bladder (urinary)
4.2%
1/24 • Number of events 1
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Blood
12.5%
3/24 • Number of events 4
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Bronchus
4.2%
1/24 • Number of events 1
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Lung (pneumonia)
4.2%
1/24 • Number of events 1
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Urinary tract NOS
4.2%
1/24 • Number of events 1
Infections and infestations
Infection - Other: Multiple viral infections
4.2%
1/24 • Number of events 1
Infections and infestations
Infection - Other: Probable fungal disease
4.2%
1/24 • Number of events 1
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Abdomen NOS
8.3%
2/24 • Number of events 2
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Bladder (urinary)
12.5%
3/24 • Number of events 6
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Blood
12.5%
3/24 • Number of events 4
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Catheter-related
8.3%
2/24 • Number of events 2
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Upper aerodigestive NOS
8.3%
2/24 • Number of events 2
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Upper airway NOS
8.3%
2/24 • Number of events 3
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Urinary tract NOS
4.2%
1/24 • Number of events 1
Metabolism and nutrition disorders
AST, SGOT
8.3%
2/24 • Number of events 2
Nervous system disorders
Neurology - Other: Aseptic meningitis
4.2%
1/24 • Number of events 1
Nervous system disorders
Neurology - Other: Cranial nerve VI palsy
4.2%
1/24 • Number of events 1
Nervous system disorders
Neurology - Other: Peripheral neuropathy
4.2%
1/24 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Hypoxia
4.2%
1/24 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Pleural effusion (non-malignant)
4.2%
1/24 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other: Acute respiratory failure
4.2%
1/24 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other: Respiratory distress: dyspnea + hypoxia
4.2%
1/24 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other: Respiratory failure
4.2%
1/24 • Number of events 1
Renal and urinary disorders
Cystitis
4.2%
1/24 • Number of events 1
Renal and urinary disorders
Renal failure
12.5%
3/24 • Number of events 3
Vascular disorders
Vascular - Other: Vaso Oclusive Disease
4.2%
1/24 • Number of events 1
General disorders
Pain - Abdomen NOS
4.2%
1/24 • Number of events 1

Other adverse events

Other adverse events
Measure
Stem Cell Transplant
n=24 participants at risk
All patients will receive Ara C IV every 12 hours for 6 doses starting at 1400 hours on day -8. Cyclophosphamide IV once daily on day -7 and day -6 starting at 1400 hours. MESNA will be administered 15 minutes prior to each dose of Cyclophosphamide and 3, 6, 9, and 12 hours after each dose of Cyclophosphamide. Campath 1h will be given on day -4, day -3, day -2 and day-1. TBI (Total Body Irradiation) will be delivered in 8 fractions of 1.75 Gy in two fractions on day -4, day -3, day -2, and day -1. Stem cell Infusion are infused on day 0.
Gastrointestinal disorders
Vomiting
12.5%
3/24 • Number of events 3
Infections and infestations
Infection - Other: CMV viremia
8.3%
2/24 • Number of events 2
Infections and infestations
Infection with Grade 3 or 4 neutrophils - Blood
8.3%
2/24 • Number of events 2
Metabolism and nutrition disorders
AST, SGOT
8.3%
2/24 • Number of events 2
Renal and urinary disorders
Renal failure
8.3%
2/24 • Number of events 2
Cardiac disorders
Hypotension
8.3%
2/24 • Number of events 2
Vascular disorders
PTT
8.3%
2/24 • Number of events 2
Gastrointestinal disorders
Diarrhea
8.3%
2/24 • Number of events 3
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam)
16.7%
4/24 • Number of events 5
Gastrointestinal disorders
Nausea
12.5%
3/24 • Number of events 3

Additional Information

Robert Krance, MD

Baylor College of Medicine

Phone: 832-824-4661

Results disclosure agreements

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