Trial Outcomes & Findings for Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies (NCT NCT00943319)

Last Updated: 2019-02-04

Results Overview

Maximally tolerated area under the curve of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion. The number reported will be an Area Under the Curve (AUC) measure reported in µmol-min/L.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

50 participants

Primary outcome timeframe

5 years

Results posted on

2019-02-04

Participant Flow

Participant milestones

Participant milestones
Measure	Busulfan and Fludarabine Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Overall Study STARTED	50
Overall Study COMPLETED	49
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Busulfan and Fludarabine Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Overall Study Death	1

Baseline Characteristics

Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Busulfan and Fludarabine n=50 Participants Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	46 Participants n=5 Participants
Age, Categorical >=65 years	4 Participants n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	31 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 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	3 Participants n=5 Participants
Race (NIH/OMB) White	39 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	5 Participants n=5 Participants
Region of Enrollment United States	50 participants n=5 Participants

PRIMARY outcome

Timeframe: 5 years

Population: The 36 patients refer to the phase I portion of the study only.

Outcome measures

Outcome measures
Measure	Busulfan and Fludarabine n=36 Participants Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Maximum Tolerated Dose	6800 mmol-min/L

SECONDARY outcome

Timeframe: 5 years

Overall Survival measured as median survival in days

Outcome measures

Outcome measures
Measure	Busulfan and Fludarabine n=50 Participants Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Overall Survival	161 days Interval 121.0 to 305.0

SECONDARY outcome

Timeframe: 5 years

Disease Free Survival measured by median survival time in days

Outcome measures

Outcome measures
Measure	Busulfan and Fludarabine n=50 Participants Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Disease Free Survival	172 days Interval 85.0 to 436.0

Adverse Events

Busulfan and Fludarabine

Serious events: 1 serious events

Other events: 1 other events

Deaths: 45 deaths

Serious adverse events

Serious adverse events
Measure	Busulfan and Fludarabine n=49 participants at risk Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Blood and lymphatic system disorders Sepsis	2.0% 1/49 • Number of events 1 • 10 years

Other adverse events

Other adverse events
Measure	Busulfan and Fludarabine n=49 participants at risk Intravenous busulfan (Busulfan®) in combination with fludarabine Busulfan: Daily intravenous dosing to target AVC Fludarabine: Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose. Campath: All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times). Stem Cell Transplant: Infusion of bone marrow and donors(related/ unrelated).
Nervous system disorders Mental Status change	2.0% 1/49 • Number of events 1 • 10 years

Additional Information

Dr. Andrew Artz

UChicago

Phone: 773-834-8980

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place