Trial Outcomes & Findings for Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders (NCT NCT00856388)
NCT ID: NCT00856388
Last Updated: 2019-11-13
Results Overview
Day 100 Treatment Related Mortality An exact 95% confidence interval will be provided.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
62 participants
Primary outcome timeframe
First 100 days
Results posted on
2019-11-13
Participant Flow
Participant milestones
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Overall Study
STARTED
|
62
|
|
Overall Study
COMPLETED
|
61
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Overall Study
Other
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1
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Baseline Characteristics
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
Baseline characteristics by cohort
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=62 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
43 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
17 Participants
n=5 Participants
|
|
Age, Continuous
|
54.6 years
STANDARD_DEVIATION 14.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
29 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
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33 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: First 100 daysPopulation: All treated and eligible patients
Day 100 Treatment Related Mortality An exact 95% confidence interval will be provided.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=61 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Day 100 TRM
|
8.44 percentage of participants
Interval 3.08 to 17.29
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SECONDARY outcome
Timeframe: Days 30Population: All treated and eligible patients
Median Time to ANC Engraftment Summarized using standard descriptive statistics along with corresponding 95% confidence intervals.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=61 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
|
Median Time to ANC Engraftment
|
16.5 Days
Interval 0.0 to 26.0
|
SECONDARY outcome
Timeframe: Day 100Population: All treated and eligible patients
Median Time to Platelet Engraftment Summarized using standard descriptive statistics along with corresponding 95% confidence intervals.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=61 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Median Time to Platelet Engraftment
|
17.0 Days
Interval 0.0 to 74.0
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SECONDARY outcome
Timeframe: Day 30Population: All treated and eligible patients, who were able to complete testing
Rate of Complete Donor Chimerism - Blood Summarized using standard descriptive statistics.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=44 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Rate of Complete Donor Chimerism - Blood
|
89 percentage of participants
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SECONDARY outcome
Timeframe: Day 100Population: All treated and eligible patients, who were able to complete testing
Rate of Complete Donor Chimerism - Blood Summarized using standard descriptive statistics.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=49 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Rate of Complete Donor Chimerism - Blood
|
94 percentage of participants
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SECONDARY outcome
Timeframe: Up to day 100Population: All treated and eligible patients
Acute GVHD grade III-IV Summarized using standard descriptive statistics along with corresponding 95% confidence intervals.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=61 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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Acute GVHD Grade III-IV
|
27 percentage of participants
Interval 16.0 to 38.0
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SECONDARY outcome
Timeframe: Up to 4.5 yearsPopulation: All treated and eligible who survived to day 100 and were eligible to get chronic GVHD
1 yr Extensive Chronic GVHD Summarized using standard descriptive statistics along with corresponding 95% confidence intervals.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=53 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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1 yr Extenstive Chronic GVHD
|
60 percentage of participants
Interval 45.0 to 73.0
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SECONDARY outcome
Timeframe: Up to 4.5 yearsPopulation: All treated and eligible patients
3 yr Overall Survival estimated using the Kaplan-Meier method.
Outcome measures
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=61 Participants
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
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|---|---|
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3 yr Overall Survival
|
46.0 percentage of participants
Interval 33.0 to 57.0
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Adverse Events
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
Serious events: 34 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)
n=62 participants at risk
Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -2 and melphalan\* IV over 30 minutes on day -2. Patients then undergo total-body irradiation on day -1 and allogeneic stem cell transplantation on day 0.
Note: \*Patients with chromosomal breakage syndromes, such as Fanconi anemia or dyskeratosis congenita, receive anti-thymocyte globulin IV over 4 hours on day -4 to -2 instead of melphalan.
fludarabine phosphate: Given IV
melphalan: Given IV
total-body irradiation: Undergo total-body irradiation
allogeneic hematopoietic stem cell transplantation: Undergo allogeneic stem cell transplantation
anti-thymocyte globulin: Given IV
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|---|---|
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Blood and lymphatic system disorders
Febrile neutropenia
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1.6%
1/62 • Number of events 1
|
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Cardiac disorders
Atrial tachycardia
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1.6%
1/62 • Number of events 1
|
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Cardiac disorders
Cardiac disorder
|
3.2%
2/62 • Number of events 3
|
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Cardiac disorders
Cardio-respiratory arrest
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1.6%
1/62 • Number of events 1
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Gastrointestinal disorders
Abdominal pain
|
1.6%
1/62 • Number of events 1
|
|
General disorders
Death
|
8.1%
5/62 • Number of events 5
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General disorders
Disease progression
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1.6%
1/62 • Number of events 1
|
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General disorders
Multi-organ failure
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1.6%
1/62 • Number of events 1
|
|
General disorders
Pain
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1.6%
1/62 • Number of events 1
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General disorders
Pyrexia
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8.1%
5/62 • Number of events 6
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Immune system disorders
Graft versus host disease
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12.9%
8/62 • Number of events 9
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Infections and infestations
Bacteraemia
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3.2%
2/62 • Number of events 2
|
|
Infections and infestations
Cellulitis
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1.6%
1/62 • Number of events 1
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|
Infections and infestations
Infection
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3.2%
2/62 • Number of events 2
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|
Metabolism and nutrition disorders
Dehydration
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1.6%
1/62 • Number of events 1
|
|
Nervous system disorders
Cerebral haemorrhage
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1.6%
1/62 • Number of events 1
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|
Nervous system disorders
Headache
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1.6%
1/62 • Number of events 1
|
|
Renal and urinary disorders
Anuria
|
1.6%
1/62 • Number of events 1
|
|
Renal and urinary disorders
Haematuria
|
3.2%
2/62 • Number of events 3
|
|
Renal and urinary disorders
Renal failure
|
3.2%
2/62 • Number of events 3
|
|
Renal and urinary disorders
Urinary tract disorder
|
1.6%
1/62 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
3.2%
2/62 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
1.6%
1/62 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
1.6%
1/62 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.6%
1/62 • Number of events 1
|
|
Surgical and medical procedures
Hospitalisation
|
19.4%
12/62 • Number of events 16
|
|
Vascular disorders
Hypotension
|
3.2%
2/62 • Number of events 2
|
Other adverse events
Adverse event data not reported
Additional Information
Senior Administrator, Compliance - Clinical Research Services
Roswell Park Cancer Institute
Phone: 716-845-2300
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place