Trial Outcomes & Findings for Megadose CD34 Selected Progenitor Cells for Transplantation in Patients With Advanced Hematological Malignant Diseases (NCT NCT00038857)
NCT ID: NCT00038857
Last Updated: 2012-08-10
Results Overview
Absolute neutrophil engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 \* 10\^9/L. Baseline to Day 30 post transplant.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
29 participants
Primary outcome timeframe
Day 0 up to Day 30
Results posted on
2012-08-10
Participant Flow
Recruitment Period:October 1, 2001 to November 1, 2008. All participants recruited at UT MD Anderson Cancer Center.
Of the 29 participants enrolled, one participant was excluded from the trial and did not receive any treatment.
Participant milestones
| Measure |
CD34 PBPC
Melphalan 140 mg/m\^2 given IV for one day. Thiotepa 10 mg/kg given IV for one day. Fludarabine 40 mg/m\^2 given IV daily for four days. Rabbit ATG 1.5 mg/kg given IV daily for four days. Stem Cell Infusion of CD34+ Selected Cells given on Day 0.
|
|---|---|
|
Overall Study
STARTED
|
29
|
|
Overall Study
COMPLETED
|
28
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Megadose CD34 Selected Progenitor Cells for Transplantation in Patients With Advanced Hematological Malignant Diseases
Baseline characteristics by cohort
| Measure |
CD34 PBPC
n=29 Participants
Melphalan 140 mg/m\^2 given IV for one day. Thiotepa 10 mg/kg given IV for one day. Fludarabine 40 mg/m\^2 given IV daily for four days. Rabbit ATG 1.5 mg/kg given IV daily for four days. Stem Cell Infusion of CD34+ Selected Cells given on Day 0.
|
|---|---|
|
Age Continuous
|
36 years
STANDARD_DEVIATION 11 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
29 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 0 up to Day 30Population: Analysis per protocol.
Absolute neutrophil engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 \* 10\^9/L. Baseline to Day 30 post transplant.
Outcome measures
| Measure |
Melphalan + Thiotepa + Fludarabine + Rabbit ATG + CD34 PBPC
n=28 Participants
Melphalan 140 mg/m\^2 given for one day. Thiotepa 10 mg/kg given for one day. Fludarabine 40 mg/m\^2 given daily for four days. Rabbit ATG 1.5 mg/kg given daily for four days. Infusion of CD34+ Selected Cells given on Day 0.
|
|---|---|
|
Number of Participants With Absolute Neutrophil Count Engraftment
|
21 participant
|
Adverse Events
CD34 PBPC
Serious events: 21 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CD34 PBPC
n=28 participants at risk
Melphalan 140 mg/m\^2 given IV for one day. Thiotepa 10 mg/kg given IV for one day. Fludarabine 40 mg/m\^2 given IV daily for four days. Rabbit ATG 1.5 mg/kg given IV daily for four days. Stem Cell Infusion of CD34+ Selected Cells given on Day 0.
|
|---|---|
|
General disorders
Graft Failure
|
21.4%
6/28 • Number of events 6 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Infections and infestations
Infection
|
60.7%
17/28 • Number of events 17 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Renal and urinary disorders
Left Ureteral Obstruction
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Gastrointestinal disorders
Mucositis
|
7.1%
2/28 • Number of events 2 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Hepatobiliary disorders
Elevated alanine aminotransferase (ALT)
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Metabolism and nutrition disorders
Hypokalemia
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Fever
|
32.1%
9/28 • Number of events 9 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Disease Relapse
|
14.3%
4/28 • Number of events 4 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Graft vs Host Disease
|
7.1%
2/28 • Number of events 2 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
17.9%
5/28 • Number of events 5 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Blood and lymphatic system disorders
Hemolysis
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Nervous system disorders
Neuropathy
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Renal and urinary disorders
Renal Failure
|
7.1%
2/28 • Number of events 2 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Death
|
14.3%
4/28 • Number of events 4 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Cardiac disorders
Cardiac Failure
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Blood and lymphatic system disorders
Thrombotic Microangiopathy
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Respiratory, thoracic and mediastinal disorders
Diffuse Alveolar Hemorrhage
|
10.7%
3/28 • Number of events 3 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Nervous system disorders
Encephalopathy
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
Other adverse events
| Measure |
CD34 PBPC
n=28 participants at risk
Melphalan 140 mg/m\^2 given IV for one day. Thiotepa 10 mg/kg given IV for one day. Fludarabine 40 mg/m\^2 given IV daily for four days. Rabbit ATG 1.5 mg/kg given IV daily for four days. Stem Cell Infusion of CD34+ Selected Cells given on Day 0.
|
|---|---|
|
Blood and lymphatic system disorders
Low Platelets
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Cardiac disorders
Hypertension
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Fever
|
14.3%
4/28 • Number of events 4 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Gastrointestinal disorders
Diarrhea
|
35.7%
10/28 • Number of events 10 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Gastrointestinal disorders
Mucositis
|
39.3%
11/28 • Number of events 11 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Gastrointestinal disorders
Gastrointestinal Bleed
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Gastrointestinal disorders
Nausea
|
50.0%
14/28 • Number of events 14 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Renal and urinary disorders
Elevated Creatinine
|
7.1%
2/28 • Number of events 2 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Infections and infestations
BK Virus
|
10.7%
3/28 • Number of events 3 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Renal and urinary disorders
Hemorrhagic Cystitis
|
21.4%
6/28 • Number of events 6 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Hepatobiliary disorders
Elevated Alkaline Phosphatase
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Hepatobiliary disorders
Elevated Alanine Aminotransferase
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Hepatobiliary disorders
Elevated Bilirubin
|
7.1%
2/28 • Number of events 2 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Infections and infestations
Infecrtion
|
50.0%
14/28 • Number of events 14 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Neutropenic Fever
|
25.0%
7/28 • Number of events 7 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Respiratory, thoracic and mediastinal disorders
Bronchiolitis
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
10.7%
3/28 • Number of events 3 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Fluid Overload
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
General disorders
Graft versus Host Disease
|
25.0%
7/28 • Number of events 7 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
|
Eye disorders
Diplopia
|
3.6%
1/28 • Number of events 1 • 7 Years, 1 Month (October 1, 2001 to November 1, 2008)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place