Trial Outcomes & Findings for Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States (NCT NCT00997386)
NCT ID: NCT00997386
Last Updated: 2019-09-09
Results Overview
To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood) in peripheral blood by day +100 (i.e., 100 days after allogeneic PBSCT).
COMPLETED
PHASE2
16 participants
Day +100
2019-09-09
Participant Flow
Participant milestones
| Measure |
Busulfan, and Melphalan, and Alemtuzumab
Three drug regimen using busulfan, and melphalan, and alemtuzumab.
Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT).
Intravenous melphalan 100 mg/m2 on day -3.
Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
Busulfan, and Melphalan, and Alemtuzumab
Three drug regimen using busulfan, and melphalan, and alemtuzumab.
Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT).
Intravenous melphalan 100 mg/m2 on day -3.
Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.
|
|---|---|
|
Overall Study
Death
|
7
|
Baseline Characteristics
Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States
Baseline characteristics by cohort
| Measure |
Busulfan, and Melphalan, and Alemtuzumab
n=16 Participants
Three drug regimen using busulfan, and melphalan, and alemtuzumab.
Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT).
Intravenous melphalan 100 mg/m2 on day -3.
Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
16 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day +100Population: Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment
To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood) in peripheral blood by day +100 (i.e., 100 days after allogeneic PBSCT).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day +100Population: Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment.
To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day +100Population: Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment
To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day +100Population: Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment
To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.
Outcome measures
Outcome data not reported
Adverse Events
Busulfan, and Melphalan, and Alemtuzumab
Serious adverse events
| Measure |
Busulfan, and Melphalan, and Alemtuzumab
n=16 participants at risk
Three drug regimen using busulfan, and melphalan, and alemtuzumab.
Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT).
Intravenous melphalan 100 mg/m2 on day -3.
Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.
|
|---|---|
|
Gastrointestinal disorders
Pneumatosis cystoides intestinalis
|
6.2%
1/16
|
|
General disorders
Fever
|
6.2%
1/16
|
|
Gastrointestinal disorders
Abdominal Pain
|
6.2%
1/16
|
Other adverse events
| Measure |
Busulfan, and Melphalan, and Alemtuzumab
n=16 participants at risk
Three drug regimen using busulfan, and melphalan, and alemtuzumab.
Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT).
Intravenous melphalan 100 mg/m2 on day -3.
Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Acidosis
|
6.2%
1/16
|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratory Distress Syndrome
|
12.5%
2/16
|
|
Blood and lymphatic system disorders
Hypoalbuminemia
|
12.5%
2/16
|
|
Blood and lymphatic system disorders
Alkaline phosphatase
|
12.5%
2/16
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Alkalosis
|
6.2%
1/16
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
6.2%
1/16
|
|
Metabolism and nutrition disorders
Anorexia
|
25.0%
4/16
|
|
Blood and lymphatic system disorders
Hyperbilirubinemia
|
12.5%
2/16
|
|
Renal and urinary disorders
Bladder spasms
|
6.2%
1/16
|
|
Blood and lymphatic system disorders
Blood/Bone marrow - other
|
31.2%
5/16
|
|
Blood and lymphatic system disorders
Hypocalcemia
|
6.2%
1/16
|
|
Cardiac disorders
Cardiac arrhythmia
|
12.5%
2/16
|
|
Cardiac disorders
Cardiac - general
|
18.8%
3/16
|
|
Gastrointestinal disorders
Infectious colitis
|
6.2%
1/16
|
|
General disorders
Confusion
|
12.5%
2/16
|
|
Gastrointestinal disorders
Constipation
|
37.5%
6/16
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
68.8%
11/16
|
|
Blood and lymphatic system disorders
Creatinine
|
62.5%
10/16
|
|
Skin and subcutaneous tissue disorders
Skin - other
|
37.5%
6/16
|
|
Gastrointestinal disorders
Diarrhea
|
93.8%
15/16
|
|
Gastrointestinal disorders
Abdominal bloating
|
12.5%
2/16
|
|
General disorders
Dizziness
|
31.2%
5/16
|
|
Eye disorders
Dry eye
|
25.0%
4/16
|
|
Skin and subcutaneous tissue disorders
Dry mouth
|
25.0%
4/16
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
12.5%
2/16
|
|
Nervous system disorders
Dysphagia
|
25.0%
4/16
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
62.5%
10/16
|
|
Blood and lymphatic system disorders
Edema
|
56.2%
9/16
|
|
Gastrointestinal disorders
Esophagitis
|
6.2%
1/16
|
|
Nervous system disorders
Gait disturbance
|
18.8%
3/16
|
|
General disorders
Fatigue
|
87.5%
14/16
|
|
Blood and lymphatic system disorders
Neutropenia
|
68.8%
11/16
|
|
General disorders
Fever
|
62.5%
10/16
|
|
Blood and lymphatic system disorders
Flushing
|
12.5%
2/16
|
|
Gastrointestinal disorders
Gastrointestinal - other
|
68.8%
11/16
|
|
Endocrine disorders
Hyperglycemia
|
25.0%
4/16
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
25.0%
4/16
|
|
Gastrointestinal disorders
Dyspepsia
|
18.8%
3/16
|
|
Blood and lymphatic system disorders
Hemoglobin
|
68.8%
11/16
|
|
Blood and lymphatic system disorders
Bleeding - other
|
18.8%
3/16
|
|
Hepatobiliary disorders
Hepatobiliary/pancreas - other
|
6.2%
1/16
|
|
Gastrointestinal disorders
Hemorrhoids
|
6.2%
1/16
|
|
General disorders
Hypertension
|
68.8%
11/16
|
|
General disorders
Hypotension
|
25.0%
4/16
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
12.5%
2/16
|
|
Infections and infestations
Infection
|
68.8%
11/16
|
|
General disorders
Insomnia
|
50.0%
8/16
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC)
|
100.0%
16/16
|
|
Blood and lymphatic system disorders
Lymphatics - other
|
12.5%
2/16
|
|
Blood and lymphatic system disorders
Hypomagnesemia
|
93.8%
15/16
|
|
Psychiatric disorders
Anxiety
|
62.5%
10/16
|
|
Psychiatric disorders
Depression
|
18.8%
3/16
|
|
Skin and subcutaneous tissue disorders
Mucositis/stomatitis
|
87.5%
14/16
|
|
Metabolism and nutrition disorders
Metabolic - other
|
43.8%
7/16
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal - weakness/other
|
18.8%
3/16
|
|
Respiratory, thoracic and mediastinal disorders
Sinus reaction
|
6.2%
1/16
|
|
Gastrointestinal disorders
Nausea
|
100.0%
16/16
|
|
Nervous system disorders
Neurology - other
|
18.8%
3/16
|
|
Nervous system disorders
Neuropathy
|
12.5%
2/16
|
|
Blood and lymphatic system disorders
Neutrophils/Granulocytes
|
81.2%
13/16
|
|
General disorders
Pain
|
100.0%
16/16
|
|
Renal and urinary disorders
Kidney perforation
|
6.2%
1/16
|
|
Blood and lymphatic system disorders
Petichiae/Purpura
|
6.2%
1/16
|
|
Blood and lymphatic system disorders
Hypophosphatemia
|
6.2%
1/16
|
|
Blood and lymphatic system disorders
Platelets
|
68.8%
11/16
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/Pulmonary infiltrates
|
18.8%
3/16
|
|
General disorders
Prutitis
|
31.2%
5/16
|
|
Psychiatric disorders
Psychosis
|
12.5%
2/16
|
|
Cardiac disorders
Pulmonary hypertension
|
6.2%
1/16
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper respiratory - other
|
18.8%
3/16
|
|
Skin and subcutaneous tissue disorders
Rash
|
18.8%
3/16
|
|
Renal and urinary disorders
Renal failure
|
12.5%
2/16
|
|
Renal and urinary disorders
Renal/Genitourinary - other
|
31.2%
5/16
|
|
General disorders
Rigors/chills
|
100.0%
16/16
|
|
Nervous system disorders
Seizure
|
6.2%
1/16
|
|
Reproductive system and breast disorders
Sexual/reproductive function - other
|
6.2%
1/16
|
|
Blood and lymphatic system disorders
Hypernatremia
|
6.2%
1/16
|
|
Blood and lymphatic system disorders
Hyponatremia
|
12.5%
2/16
|
|
General disorders
Somnolence/depressed level of consciousness
|
6.2%
1/16
|
|
Cardiac disorders
Atrial fibrillation
|
18.8%
3/16
|
|
Cardiac disorders
Tachycardia
|
25.0%
4/16
|
|
General disorders
Syncope
|
6.2%
1/16
|
|
Gastrointestinal disorders
Dysgeusia
|
6.2%
1/16
|
|
Nervous system disorders
Tremor
|
12.5%
2/16
|
|
Renal and urinary disorders
Dysuria
|
6.2%
1/16
|
|
Renal and urinary disorders
Urticaria
|
25.0%
4/16
|
|
Reproductive system and breast disorders
Vaginal discharge
|
6.2%
1/16
|
|
Vascular disorders
Vascular - other
|
25.0%
4/16
|
|
Cardiac disorders
Ventricular arrhythmia
|
6.2%
1/16
|
|
Eye disorders
Blurred vision
|
6.2%
1/16
|
|
Gastrointestinal disorders
Dysarthria
|
6.2%
1/16
|
|
Gastrointestinal disorders
Vomiting
|
68.8%
11/16
|
|
Metabolism and nutrition disorders
Weight gain
|
12.5%
2/16
|
|
Metabolism and nutrition disorders
Weight loss
|
12.5%
2/16
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place