Trial Outcomes & Findings for Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant (NCT NCT01118013)
NCT ID: NCT01118013
Last Updated: 2017-03-24
Results Overview
EFS was defined as the date of transplant to date of progression or develop myelodysplasia after autologous transplant. EFS was estimated using the Kaplan Meier method.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Duration of study (up to 5.5 years)
Results posted on
2017-03-24
Participant Flow
Between May 2010 and March 2012, 6 participants were recruited.
Participant milestones
| Measure |
Treatment
Participants will receive:
Busulfan test dose of 25 mg/m\^2 IV over 45 minutes between days -14 and -9; Fludarabine 30 mg/m\^2/day IV over 30 minutes on days -7 to -3; Busulfan IV x 4 days on days -6 through -3 (dosage based on AUC of 4000 mmol/min based on pharmacokinetics determined from test dose); Allopurinol was given at discretion of treating physician; Rabbit antithymocyte globulin 1.5 mg/kg/day IV on days -6 and -5; Peripheral Blood Stem Cell Transplant (PBST) on Day 0 and +1; and methotrexate 5 mg/m\^2/day IV on days +1, +3 and +6. G-CSF of 5mcg/kg/day SQ began daily on day +7 continuing until ANC \> 1000/mL for 3 consecutive days.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant
Baseline characteristics by cohort
| Measure |
Treatment
n=6 Participants
Participants will receive:
Busulfan test dose of 25 mg/m\^2 IV over 45 minutes between days -14 and -9; Fludarabine 30 mg/m\^2/day IV over 30 minutes on days -7 to -3; Busulfan IV x 4 days on days -6 through -3 (dosage based on AUC of 4000 mmol/min based on pharmacokinetics determined from test dose); Allopurinol was given at discretion of treating physician; Rabbit antithymocyte globulin 1.5 mg/kg/day IV on days -6 and -5; Peripheral Blood Stem Cell Transplant (PBST) on Day 0 and +1; and methotrexate 5 mg/m\^2/day IV on days +1, +3 and +6. G-CSF of 5mcg/kg/day SQ began daily on day +7 continuing until ANC \> 1000/mL for 3 consecutive days.
|
|---|---|
|
Age, Continuous
|
67 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 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
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Duration of study (up to 5.5 years)Population: Due to study termination, data were not collected and the outcome measure was not analyzed.
EFS was defined as the date of transplant to date of progression or develop myelodysplasia after autologous transplant. EFS was estimated using the Kaplan Meier method.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 2 yearsPopulation: Due to study termination, data were not collected and the outcome measure was not analyzed.
EFS distributions between CALGB-100002 and this study will be compared using the two-sample log-rank test.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 5.5 yearsPopulation: Due to study termination, data were not collected and the outcome measure was not analyzed.
Complete response (CR) rate is reported as the percentage of participants who achieved a CR.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 5.5 yearsPopulation: Due to study termination, data were not collected and the outcome measure was not analyzed.
Overall survival (OS) was defined as the transplant from registration to death of any cause. Surviving patients were censored at the date of last follow-up. The median OS with 95% confidence interval (CI) was estimated using the Kaplan Meier method.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 year post transplantPopulation: Due to study termination, data were not collected and the outcome measure was not analyzed.
Percent of participants who have an opportunistic (viral, bacterial and fungal) infection in the first year following transplant.
Outcome measures
Outcome data not reported
Adverse Events
Treatment
Serious events: 3 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment
n=6 participants at risk
Participants will receive:
Busulfan test dose of 25 mg/m\^2 IV over 45 minutes between days -14 and -9; Fludarabine 30 mg/m\^2/day IV over 30 minutes on days -7 to -3; Busulfan IV x 4 days on days -6 through -3 (dosage based on AUC of 4000 mmol/min based on pharmacokinetics determined from test dose); Allopurinol was given at discretion of treating physician; Rabbit antithymocyte globulin 1.5 mg/kg/day IV on days -6 and -5; Peripheral Blood Stem Cell Transplant (PBST) on Day 0 and +1; and methotrexate 5 mg/m\^2/day IV on days +1, +3 and +6. G-CSF of 5mcg/kg/day SQ began daily on day +7 continuing until ANC \> 1000/mL for 3 consecutive days.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
16.7%
1/6 • Number of events 1
|
|
Cardiac disorders
Sinus tachycardia
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
Cataract
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
Flashing lights
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
Watering eyes
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • Number of events 1
|
|
General disorders
Death NOS
|
16.7%
1/6 • Number of events 1
|
|
General disorders
Edema limbs
|
16.7%
1/6 • Number of events 1
|
|
General disorders
Fatigue
|
33.3%
2/6 • Number of events 2
|
|
General disorders
Fever
|
16.7%
1/6 • Number of events 1
|
|
General disorders
Malaise
|
16.7%
1/6 • Number of events 1
|
|
General disorders
Non-cardiac chest pain
|
16.7%
1/6 • Number of events 1
|
|
General disorders
Pain
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
Infections and infestations - Other, specify
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
Upper respiratory infection
|
16.7%
1/6 • Number of events 1
|
|
Investigations
Creatinine increased
|
33.3%
2/6 • Number of events 2
|
|
Investigations
Neutrophil count decreased
|
16.7%
1/6 • Number of events 1
|
|
Investigations
Platelet count decreased
|
33.3%
2/6 • Number of events 2
|
|
Investigations
Weight loss
|
16.7%
1/6 • Number of events 1
|
|
Investigations
White blood cell decreased
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Anorexia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
33.3%
2/6 • Number of events 2
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hyponatremia
|
16.7%
1/6 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
1/6 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
2/6 • Number of events 2
|
|
Nervous system disorders
Ataxia
|
16.7%
1/6 • Number of events 1
|
|
Nervous system disorders
Dysgeusia
|
16.7%
1/6 • Number of events 1
|
|
Nervous system disorders
Tremor
|
16.7%
1/6 • Number of events 1
|
|
Psychiatric disorders
Agitation
|
16.7%
1/6 • Number of events 1
|
|
Psychiatric disorders
Anxiety
|
16.7%
1/6 • Number of events 1
|
|
Psychiatric disorders
Delirium
|
16.7%
1/6 • Number of events 1
|
|
Renal and urinary disorders
Acute kidney injury
|
16.7%
1/6 • Number of events 1
|
|
Renal and urinary disorders
Hematuria
|
16.7%
1/6 • Number of events 1
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
16.7%
1/6 • Number of events 1
|
|
Vascular disorders
Hypotension
|
16.7%
1/6 • Number of events 1
|
Other adverse events
| Measure |
Treatment
n=6 participants at risk
Participants will receive:
Busulfan test dose of 25 mg/m\^2 IV over 45 minutes between days -14 and -9; Fludarabine 30 mg/m\^2/day IV over 30 minutes on days -7 to -3; Busulfan IV x 4 days on days -6 through -3 (dosage based on AUC of 4000 mmol/min based on pharmacokinetics determined from test dose); Allopurinol was given at discretion of treating physician; Rabbit antithymocyte globulin 1.5 mg/kg/day IV on days -6 and -5; Peripheral Blood Stem Cell Transplant (PBST) on Day 0 and +1; and methotrexate 5 mg/m\^2/day IV on days +1, +3 and +6. G-CSF of 5mcg/kg/day SQ began daily on day +7 continuing until ANC \> 1000/mL for 3 consecutive days.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
6/6 • Number of events 18
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
50.0%
3/6 • Number of events 3
|
|
Cardiac disorders
Cardiac disorders - Other, specify
|
33.3%
2/6 • Number of events 2
|
|
Cardiac disorders
Heart failure
|
16.7%
1/6 • Number of events 1
|
|
Cardiac disorders
Pericardial effusion
|
33.3%
2/6 • Number of events 3
|
|
Cardiac disorders
Sinus bradycardia
|
16.7%
1/6 • Number of events 1
|
|
Cardiac disorders
Sinus tachycardia
|
16.7%
1/6 • Number of events 3
|
|
Cardiac disorders
Ventricular arrhythmia
|
16.7%
1/6 • Number of events 1
|
|
Cardiac disorders
Ventricular tachycardia
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
Blurred vision
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
Cataract
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
Dry eye
|
16.7%
1/6 • Number of events 2
|
|
Eye disorders
Watering eyes
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
Constipation
|
50.0%
3/6 • Number of events 4
|
|
Gastrointestinal disorders
Diarrhea
|
66.7%
4/6 • Number of events 7
|
|
Gastrointestinal disorders
Dry mouth
|
50.0%
3/6 • Number of events 6
|
|
Gastrointestinal disorders
Dysphagia
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
Enterocolitis
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
Mucositis oral
|
100.0%
6/6 • Number of events 6
|
|
Gastrointestinal disorders
Nausea
|
50.0%
3/6 • Number of events 6
|
|
Gastrointestinal disorders
Oral pain
|
50.0%
3/6 • Number of events 3
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
3/6 • Number of events 5
|
|
General disorders
Chills
|
33.3%
2/6 • Number of events 2
|
|
General disorders
Edema limbs
|
50.0%
3/6 • Number of events 4
|
|
General disorders
Fatigue
|
66.7%
4/6 • Number of events 12
|
|
General disorders
Fever
|
50.0%
3/6 • Number of events 8
|
|
General disorders
Pain
|
33.3%
2/6 • Number of events 3
|
|
Infections and infestations
Catheter related infection
|
33.3%
2/6 • Number of events 2
|
|
Infections and infestations
Infections and infestations - Other, specify
|
50.0%
3/6 • Number of events 7
|
|
Infections and infestations
Lung infection
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
Meningitis
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
Mucosal infection
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
Sepsis
|
33.3%
2/6 • Number of events 2
|
|
Infections and infestations
Upper respiratory infection
|
33.3%
2/6 • Number of events 2
|
|
Injury, poisoning and procedural complications
Bruising
|
16.7%
1/6 • Number of events 1
|
|
Investigations
Activated partial thromboplastin time prolonged
|
33.3%
2/6 • Number of events 2
|
|
Investigations
Alanine aminotransferase increased
|
83.3%
5/6 • Number of events 13
|
|
Investigations
Alkaline phosphatase increased
|
50.0%
3/6 • Number of events 5
|
|
Investigations
Aspartate aminotransferase increased
|
83.3%
5/6 • Number of events 13
|
|
Investigations
Blood bilirubin increased
|
33.3%
2/6 • Number of events 2
|
|
Investigations
Cholesterol high
|
16.7%
1/6 • Number of events 1
|
|
Investigations
Creatinine increased
|
100.0%
6/6 • Number of events 16
|
|
Investigations
GGT increased
|
16.7%
1/6 • Number of events 1
|
|
Investigations
INR increased
|
33.3%
2/6 • Number of events 2
|
|
Investigations
Investigations - Other, specify
|
33.3%
2/6 • Number of events 3
|
|
Investigations
Lymphocyte count decreased
|
16.7%
1/6 • Number of events 1
|
|
Investigations
Neutrophil count decreased
|
100.0%
6/6 • Number of events 13
|
|
Investigations
Platelet count decreased
|
100.0%
6/6 • Number of events 18
|
|
Investigations
Weight loss
|
66.7%
4/6 • Number of events 5
|
|
Investigations
White blood cell decreased
|
33.3%
2/6 • Number of events 8
|
|
Metabolism and nutrition disorders
Anorexia
|
66.7%
4/6 • Number of events 9
|
|
Metabolism and nutrition disorders
Dehydration
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
100.0%
6/6 • Number of events 13
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
16.7%
1/6 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
50.0%
3/6 • Number of events 7
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
50.0%
3/6 • Number of events 7
|
|
Metabolism and nutrition disorders
Hypokalemia
|
33.3%
2/6 • Number of events 9
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
50.0%
3/6 • Number of events 6
|
|
Metabolism and nutrition disorders
Hyponatremia
|
66.7%
4/6 • Number of events 10
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
33.3%
2/6 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Avascular necrosis
|
16.7%
1/6 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
33.3%
2/6 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
50.0%
3/6 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
1/6 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Trismus
|
16.7%
1/6 • Number of events 2
|
|
Nervous system disorders
Dizziness
|
33.3%
2/6 • Number of events 3
|
|
Nervous system disorders
Dysgeusia
|
33.3%
2/6 • Number of events 4
|
|
Nervous system disorders
Headache
|
16.7%
1/6 • Number of events 2
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
33.3%
2/6 • Number of events 2
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
33.3%
2/6 • Number of events 2
|
|
Psychiatric disorders
Confusion
|
33.3%
2/6 • Number of events 2
|
|
Psychiatric disorders
Depression
|
16.7%
1/6 • Number of events 4
|
|
Psychiatric disorders
Insomnia
|
33.3%
2/6 • Number of events 3
|
|
Renal and urinary disorders
Acute kidney injury
|
16.7%
1/6 • Number of events 1
|
|
Renal and urinary disorders
Chronic kidney disease
|
16.7%
1/6 • Number of events 1
|
|
Renal and urinary disorders
Hematuria
|
16.7%
1/6 • Number of events 1
|
|
Renal and urinary disorders
Urinary incontinence
|
16.7%
1/6 • Number of events 1
|
|
Reproductive system and breast disorders
Genital edema
|
16.7%
1/6 • Number of events 1
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders - Other, specify
|
16.7%
1/6 • Number of events 1
|
|
Reproductive system and breast disorders
Scrotal pain
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Apnea
|
16.7%
1/6 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
33.3%
2/6 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
2/6 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
83.3%
5/6 • Number of events 8
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
33.3%
2/6 • Number of events 5
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
33.3%
2/6 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnea
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
33.3%
2/6 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
33.3%
2/6 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
33.3%
2/6 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
66.7%
4/6 • Number of events 5
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
33.3%
2/6 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
33.3%
2/6 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
16.7%
1/6 • Number of events 1
|
|
Surgical and medical procedures
Surgical and medical procedures - Other, specify
|
16.7%
1/6 • Number of events 1
|
|
Vascular disorders
Hematoma
|
16.7%
1/6 • Number of events 1
|
|
Vascular disorders
Hypertension
|
66.7%
4/6 • Number of events 9
|
|
Vascular disorders
Hypotension
|
66.7%
4/6 • Number of events 6
|
|
Vascular disorders
Thromboembolic event
|
16.7%
1/6 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60