Trial Outcomes & Findings for Fludarabine Phosphate and Total-Body Irradiation Before Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Chronic Lymphocytic Leukemia or Small Lymphocytic Leukemia (NCT NCT00060424)
NCT ID: NCT00060424
Last Updated: 2017-12-08
Results Overview
Number of patients surviving 18 months post-transplant.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
At 18 months
Results posted on
2017-12-08
Participant Flow
Participant milestones
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Fludarabine Phosphate and Total-Body Irradiation Before Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Chronic Lymphocytic Leukemia or Small Lymphocytic Leukemia
Baseline characteristics by cohort
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 Participants
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At 18 monthsNumber of patients surviving 18 months post-transplant.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 Participants
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Overall Survival
|
15 Participants
|
SECONDARY outcome
Timeframe: 18 monthsNumber of patients with relapsed disease post-transplant. Relapse/progression is defined as 1) Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, 2) circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, or 3) lymph node Biopsy Richter's transformation.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 Participants
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Rate of Relapse
|
8 Participants
|
SECONDARY outcome
Timeframe: aGVHD: 100 days after transplant; cGVHD: 1 Year after transplant.Number of patients who developed acute/chronic GVHD post-transplant. aGVHD Stages Skin: a maculopapular eruption involving \< 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation Liver: bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 3 skin and/or stage 1 gut involvement and/or stage 1 liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 Participants
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Acute Grade II-IV GVHD and Chronic (Extensive) GVHD
Acute GVHD
|
10 Participants
|
|
Acute Grade II-IV GVHD and Chronic (Extensive) GVHD
Chronic extensive GVHD
|
10 Participants
|
SECONDARY outcome
Timeframe: 18 monthsNumber of infections patients experienced, by infection type.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 Participants
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Rate and Types of Infections
Other
|
5 infections
|
|
Rate and Types of Infections
Viral
|
27 infections
|
|
Rate and Types of Infections
Fungal
|
13 infections
|
|
Rate and Types of Infections
Fever of unknown origin
|
6 infections
|
|
Rate and Types of Infections
Bacterial
|
53 infections
|
SECONDARY outcome
Timeframe: At 200 daysDefined as death before day +200 not related to progression of disease.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 Participants
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Transplant-related Mortality
|
2 Participants
|
Adverse Events
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
Serious events: 7 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 participants at risk
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Immune system disorders
Death following progression of GVHD
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Immune system disorders
Death following disease progression post transplant
|
9.5%
2/21 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Cardiac Arrhythmia and Seizure
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Death: Sepsis/Renal failure/ with history of GVHD
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Severe abnormal pain due to gut GVH
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Acute Pulmonary Embolism
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Perforated sigmoid diverticulitis
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
Other adverse events
| Measure |
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
n=21 participants at risk
NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV on days -4 to -2 and TBI on day 0. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine PO every 12 hours on days -3 to 180 with taper on day 56 and mycophenolate mofetil PO every 12 hours on days 0-27.
Cyclosporine: Given PO
Fludarabine Phosphate: Given IV
Hematopoietic Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant
Total-Body Irradiation: Undergo TBI
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
47.6%
10/21 • Number of events 10 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
9.5%
2/21 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Minimal hydronephosis
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Surgical and medical procedures
Cholecystectomy
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Acute Pulmonary Embolism
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
9.5%
2/21 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Hypotension
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
9.5%
2/21 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Increased creatinine
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Nervous system disorders
Neurotoxicity
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Renal failure
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Blood and lymphatic system disorders
Tumor lysis syndrome
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Typhlitis & Bowel Perforation
|
4.8%
1/21 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place