Trial Outcomes & Findings for Stem Cell Transplantation To Treat High Risk Multiple Myeloma With Reduced Toxicity Myeloablative Conditioning Regimen (NCT NCT00615589)
NCT ID: NCT00615589
Last Updated: 2016-04-04
Results Overview
The primary objective is overall survival, one year from the time of transplant.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
1 Year
Results posted on
2016-04-04
Participant Flow
Participant milestones
| Measure |
Flu-Bu4
Fludarabine Busulfan chemotherapy regimen (Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Stem Cell Transplantation To Treat High Risk Multiple Myeloma With Reduced Toxicity Myeloablative Conditioning Regimen
Baseline characteristics by cohort
| Measure |
Flu-Bu4
n=22 Participants
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Age, Continuous
|
54 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 1 YearThe primary objective is overall survival, one year from the time of transplant.
Outcome measures
| Measure |
Flu-Bu4
n=22 Participants
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
The Percentage of Patients Alive 1 Year Post Transplant
|
61 percentage of patients
Interval 43.0 to 87.0
|
SECONDARY outcome
Timeframe: 1 YearOne of the secondary outcomes that will be measured is progression free survival at 1 Year. Progressive Disease (PD) is defined as a \>25% increase in serum monoclonal paraprotein, a \>25% increase in 24-hour urinary light chain excretion, a \>25% increase in plasma cells in bone marrow aspirate, an increase in the size or the development of new bone lesions/soft tissue plasmacytomas, or the development of hypercalcemia.
Outcome measures
| Measure |
Flu-Bu4
n=22 Participants
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
The Percentage of Patients Free From Progression at 1 Year
|
40 percentage of patients
Interval 23.0 to 67.0
|
SECONDARY outcome
Timeframe: 100 days, one-yearOutcome measures
| Measure |
Flu-Bu4
n=22 Participants
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Percentage of Patients With Treatment Related Mortality (TRM)
|
9 percentage of patients
Interval 2.0 to 33.0
|
SECONDARY outcome
Timeframe: 100 days, 2 yearsIncidence of acute (Stage II-IV and Stage III-IV) and chronic GVHD (any stage) were analyzed. Acute GVHD Grading: Stage II - Skin, 25-50% BSA (Body Surface Area); Liver, 3.1-6mg/dl bilirubin; Gut, 1000-1500ml/day diarrhea Stage III - Skin, generalized erythroderma; Liver, 6.1-15mg/dl bilirubin; Gut, \>1500ml/day diarrhea Stage IV - Skin, bullae; Liver, \>15mg/dl bilirubin; Gut, pain +/- ileus
Outcome measures
| Measure |
Flu-Bu4
n=22 Participants
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)
Grade II-IV Acute GVHD
|
48 percentage of participants
Interval 29.0 to 72.0
|
|
Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)
Grade III-IV Acute GVHD
|
23 percentage of participants
Interval 10.0 to 47.0
|
|
Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)
Chronic GVHD
|
55 percentage of participants
Interval 34.0 to 78.0
|
SECONDARY outcome
Timeframe: 3 yearsNon relapse mortality, defined as the percentage of deaths not attributable to disease relapse or progression at 1 year and at 3 years.
Outcome measures
| Measure |
Flu-Bu4
n=22 Participants
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Non Relapse Mortality (NRM) at 1 Year and 3 yearsThe Percentage of Deaths Not Attributable to Disease Relapse or Progression
NRM at 1 Year
|
19 percentage of deaths
Interval 7.0 to 44.0
|
|
Non Relapse Mortality (NRM) at 1 Year and 3 yearsThe Percentage of Deaths Not Attributable to Disease Relapse or Progression
NRM at 3 Years
|
29 percentage of deaths
Interval 13.0 to 55.0
|
Adverse Events
Flu-Bu4
Serious events: 13 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Flu-Bu4
n=22 participants at risk
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Immune system disorders
Allergy/Immunology - Other
|
4.5%
1/22 • Number of events 1
|
|
Blood and lymphatic system disorders
Blood/Bone Marrow - Other
|
9.1%
2/22 • Number of events 2
|
|
Cardiac disorders
Cardiac General - Other
|
4.5%
1/22 • Number of events 1
|
|
Cardiac disorders
Hypotension
|
9.1%
2/22 • Number of events 2
|
|
General disorders
Fever
|
9.1%
2/22 • Number of events 2
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Death, disease progression
|
4.5%
1/22 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
9.1%
2/22 • Number of events 2
|
|
Gastrointestinal disorders
Diarrhea
|
9.1%
2/22 • Number of events 2
|
|
Vascular disorders
Hemorrhage, bladder
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Febrile neutropenia
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Infection with Grade 3 or 4 neutrophils
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Infection - Other
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Infection, blood
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Infection, lung
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Infection, upper airway
|
4.5%
1/22 • Number of events 1
|
|
Infections and infestations
Infection, UTI
|
4.5%
1/22 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
4.5%
1/22 • Number of events 1
|
|
Nervous system disorders
Somnolence/depressed level of consciousness
|
4.5%
1/22 • Number of events 1
|
|
General disorders
Pain, headache
|
4.5%
1/22 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
13.6%
3/22 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other
|
4.5%
1/22 • Number of events 1
|
|
Renal and urinary disorders
Renal failure
|
9.1%
2/22 • Number of events 2
|
Other adverse events
| Measure |
Flu-Bu4
n=22 participants at risk
Fludarabine Busulfan chemotherapy regimen(Flu-Bu4), followed by allogeneic stem cell transplant from best available, matched donor.
Fludarabine/Busulfan x 4 days:
Fludarabine: 40 mg/m2/day in NS, administered IV over 30 minutes on days -5, -4, -3, and -2 pre-transplant.
Busulfan: 3.2 mg/kg IV daily in NS over 4 hours on days -5, -4, -3, and -2.
The Fludarabine shall be administered prior to the Busulfan each day.
|
|---|---|
|
Blood and lymphatic system disorders
Blood/Bone Marrow - Other
|
9.1%
2/22 • Number of events 2
|
|
Blood and lymphatic system disorders
Platelets
|
9.1%
2/22 • Number of events 2
|
|
Cardiac disorders
Sinus bradycardia
|
9.1%
2/22 • Number of events 2
|
|
Cardiac disorders
Sinus tachycardia
|
36.4%
8/22 • Number of events 9
|
|
Cardiac disorders
Supraventricular extrasystoles
|
9.1%
2/22 • Number of events 2
|
|
Cardiac disorders
Hypertension
|
9.1%
2/22 • Number of events 2
|
|
Cardiac disorders
Hypotension
|
27.3%
6/22 • Number of events 6
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
22.7%
5/22 • Number of events 5
|
|
General disorders
Fever
|
9.1%
2/22 • Number of events 2
|
|
Psychiatric disorders
Insomnia
|
18.2%
4/22 • Number of events 5
|
|
Metabolism and nutrition disorders
Weight gain
|
9.1%
2/22 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin - Other
|
27.3%
6/22 • Number of events 8
|
|
Skin and subcutaneous tissue disorders
Pruritus/itching
|
9.1%
2/22 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
27.3%
6/22 • Number of events 8
|
|
Gastrointestinal disorders
Diarrhea
|
13.6%
3/22 • Number of events 3
|
|
Gastrointestinal disorders
Dry mouth/salivary gland (xerostomia)
|
18.2%
4/22 • Number of events 4
|
|
Gastrointestinal disorders
Gastrointestinal - Other (Specify)
|
13.6%
3/22 • Number of events 3
|
|
Gastrointestinal disorders
Heartburn/dyspepsia
|
13.6%
3/22 • Number of events 3
|
|
Gastrointestinal disorders
Incontinence, anal
|
9.1%
2/22 • Number of events 2
|
|
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam)
|
13.6%
3/22 • Number of events 3
|
|
Gastrointestinal disorders
Taste alteration (dysgeusia)
|
27.3%
6/22 • Number of events 7
|
|
Gastrointestinal disorders
Hemorrhage, GI
|
9.1%
2/22 • Number of events 2
|
|
Renal and urinary disorders
Hemorrhage, GU
|
9.1%
2/22 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary/upper respiratory
|
13.6%
3/22 • Number of events 3
|
|
Infections and infestations
Infection - Other (Specify)
|
22.7%
5/22 • Number of events 5
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils
|
13.6%
3/22 • Number of events 5
|
|
General disorders
Edema: head and neck
|
9.1%
2/22 • Number of events 2
|
|
General disorders
Edema: limb
|
22.7%
5/22 • Number of events 5
|
|
General disorders
Edema: trunk/genital
|
9.1%
2/22 • Number of events 2
|
|
Investigations
ALT, SGPT (serum glutamic pyruvic transaminase)
|
9.1%
2/22 • Number of events 2
|
|
Investigations
Albumin, serum-low (hypoalbuminemia)
|
9.1%
2/22 • Number of events 2
|
|
Investigations
Alkaline phosphatase
|
9.1%
2/22 • Number of events 2
|
|
Metabolism and nutrition disorders
Calcium, serum-low (hypocalcemia)
|
9.1%
2/22 • Number of events 2
|
|
Metabolism and nutrition disorders
Potassium, serum-high (hyperkalemia)
|
9.1%
2/22 • Number of events 2
|
|
Metabolism and nutrition disorders
Potassium, serum-low (hypokalemia)
|
9.1%
2/22 • Number of events 2
|
|
Metabolism and nutrition disorders
Sodium, serum-low (hyponatremia)
|
9.1%
2/22 • Number of events 2
|
|
Nervous system disorders
Confusion
|
9.1%
2/22 • Number of events 2
|
|
Nervous system disorders
Dizziness
|
27.3%
6/22 • Number of events 7
|
|
Psychiatric disorders
Mood alteration, anxiety
|
9.1%
2/22 • Number of events 2
|
|
Nervous system disorders
Neurology - Other
|
13.6%
3/22 • Number of events 4
|
|
Nervous system disorders
Somnolence/depressed level of consciousness
|
9.1%
2/22 • Number of events 2
|
|
Nervous system disorders
Tremor
|
31.8%
7/22 • Number of events 7
|
|
Eye disorders
Dry eye syndrome
|
9.1%
2/22 • Number of events 2
|
|
Eye disorders
Vision-blurred vision
|
13.6%
3/22 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Pain, back
|
13.6%
3/22 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Pain, bone
|
9.1%
2/22 • Number of events 2
|
|
Nervous system disorders
Pain, headache
|
22.7%
5/22 • Number of events 6
|
|
Musculoskeletal and connective tissue disorders
Pain, neck
|
9.1%
2/22 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Pain, pelvis
|
18.2%
4/22 • Number of events 4
|
|
General disorders
Pain - Other
|
18.2%
4/22 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.6%
3/22 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
22.7%
5/22 • Number of events 5
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
22.7%
5/22 • Number of events 9
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other
|
9.1%
2/22 • Number of events 2
|
|
Renal and urinary disorders
Bladder spasms
|
9.1%
2/22 • Number of events 3
|
|
Renal and urinary disorders
Renal failure
|
9.1%
2/22 • Number of events 2
|
|
Renal and urinary disorders
Urinary frequency/urgency
|
9.1%
2/22 • Number of events 2
|
|
Renal and urinary disorders
Urinary retention (including neurogenic bladder)
|
9.1%
2/22 • Number of events 2
|
|
General disorders
Syndromes - Other
|
9.1%
2/22 • Number of events 3
|
Additional Information
Dr. Attaphol Pawarode, M.D.
University of Michigan Comprehensive Cancer Center
Phone: 734-936-8785
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place