Trial Outcomes & Findings for Busulfan Safety/Efficacy as Conditioning Prior to Hematopoietic Cell Transplantation (HCT) (NCT NCT00361140)

NCT ID: NCT00361140

Last Updated: 2017-03-03

Results Overview

The number of participants dead due to causes unrelated to relapse within the first 100 days post transplant.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

72 participants

Primary outcome timeframe

100 days

Results posted on

2017-03-03

Participant Flow

BMT population recruited from 08/01/2005 through 09/09/2012

candidates not meeting eligibility criteria excluded.

Participant milestones

Participant milestones
Measure	AUC Level 1 Busulfan targeted area under the curve, level 1: 6000 +/- 600 uM-min	AUC Level 2 Busulfan targeted area under the curve, level 2: 7500 +/- 750 uM-min	AUC Level 3 Busulfan targeted area under the curve, level 3: 9000 +/- 900 uM-min
Overall Study STARTED	40	29	3
Overall Study COMPLETED	40	29	3
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Busulfan Safety/Efficacy as Conditioning Prior to Hematopoietic Cell Transplantation (HCT)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AUC Level 1 n=40 Participants Busulfan targeted area under the curve, level 1: 6000 +/- 600 uM-min	AUC Level 2 n=29 Participants Busulfan targeted area under the curve, level 2: 7500 +/- 750 uM-min	AUC Level 3 n=3 Participants Busulfan targeted area under the curve, level 3: 9000 +/- 900 uM-min	Total n=72 Participants Total of all reporting groups
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Female	16 Participants n=5 Participants	17 Participants n=7 Participants	3 Participants n=5 Participants	36 Participants n=4 Participants
Sex: Female, Male Male	24 Participants n=5 Participants	12 Participants n=7 Participants	0 Participants n=5 Participants	36 Participants n=4 Participants
Region of Enrollment United States	40 participants n=5 Participants	29 participants n=7 Participants	3 participants n=5 Participants	72 participants n=4 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	40 Participants n=5 Participants	29 Participants n=7 Participants	3 Participants n=5 Participants	72 Participants n=4 Participants

PRIMARY outcome

Timeframe: 100 days

Population: Intent to treat

The number of participants dead due to causes unrelated to relapse within the first 100 days post transplant.

Outcome measures

Outcome measures
Measure	AUC 6000 n=40 Participants Busulfan AUC Level 1: 6000 +/- 600 uM-min Fludarabine 40 mg/m\^2 I.V. over 1 hour	AUC 7500 n=29 Participants Busulfan AUC Level 2: 7500 +/- 750 uM-min Fludarabine 40 mg/m\^2 I.V. over 1 hour	AUC 9000 n=3 Participants Busulfan AUC Level 3: 9000 +/- 900 uM-min Fludarabine 40 mg/m\^2 I.V. over 1 hour
Non-relapse Mortality	3 participants	4 participants	2 participants

SECONDARY outcome

Timeframe: 100 days

Population: Intent to treat

The number of subjects with severe VOD / SOS; severity staged according to criteria set forth by McDonald G.B., Hinds M.S., Fisher L.D., et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med. 1993;118:255-267. Assessed within the first 100 days post transplant.

Outcome measures

Outcome measures
Measure	AUC 6000 n=40 Participants Busulfan AUC Level 1: 6000 +/- 600 uM-min Fludarabine 40 mg/m\^2 I.V. over 1 hour	AUC 7500 n=29 Participants Busulfan AUC Level 2: 7500 +/- 750 uM-min Fludarabine 40 mg/m\^2 I.V. over 1 hour	AUC 9000 n=3 Participants Busulfan AUC Level 3: 9000 +/- 900 uM-min Fludarabine 40 mg/m\^2 I.V. over 1 hour
Severe Venous Occlusive Disease (VOD)/ Sinusoidal Obstructive Syndrome (SOS)	0 participants	1 participants	2 participants

Adverse Events

AUC Level 1

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

AUC Level 2

Serious events: 13 serious events

Other events: 0 other events

Deaths: 0 deaths

AUC Level 3

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Adverse event data not reported

Additional Information

Dr. Teresa Field

Moffitt Cancer Center

Phone: (813) 745-7202

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	AUC Level 1 n=40 participants at risk Busulfan targeted area under the curve, level 1: 6000 +/- 600 uM-min	AUC Level 2 n=29 participants at risk Busulfan targeted area under the curve, level 2: 7500 +/- 750 uM-min	AUC Level 3 n=3 participants at risk Busulfan targeted area under the curve, level 3: 9000 +/- 900 uM-min
Cardiac disorders Cardiac ischemia/infarction	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Gastrointestinal disorders Hemorrhage, GI - Stomach	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	33.3% 1/3 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Gastrointestinal disorders Nausea	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
General disorders Death not associated with CTCAE term - Death NOS	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Hepatobiliary disorders Cholecystitis	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Hepatobiliary disorders Hepatobiliary/Pancreas - Other, VOD	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Hepatobiliary disorders Liver dysfunction/failure (clinical)	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	33.3% 1/3 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils - Bladder (urinary)	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils - Blood	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils - Lung (pneumonia)	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	10.3% 3/29 • Number of events 4 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils - Stomach	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Metabolism and nutrition disorders AST, SGOT - high	2.5% 1/40 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/29 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Nervous system disorders Cognitive disturbance	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Psychiatric disorders Mental status	2.5% 1/40 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/29 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome (ARDS)	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Respiratory, thoracic and mediastinal disorders Hemorrhage, pulmonary/upper respiratory - Lung	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - Other, edema	0.00% 0/40 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	3.4% 1/29 • Number of events 1 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.	0.00% 0/3 Anticipated "other than serious" Allogeneic Hematopoietic Cell Transplantation (HCT) adverse events were not tabulated.