Trial Outcomes & Findings for Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers (NCT NCT01135329)

NCT ID: NCT01135329

Last Updated: 2018-07-03

Results Overview

Percentage of participants achieving full-donor chimerism in unsorted peripheral blood.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

Day 60

Results posted on

2018-07-03

Participant Flow

Participant milestones

Participant milestones
Measure
BMT Allogenic Transplantation
Fludarabine, Busulfan, Cyclophosphamide, Tacrolimus,: Fludarabine 30 mg/m2 IV once a day for 4 days Busulfan 0.8 mg/kg IV every 12 hours for 4 days Cyclophosphamide 50 mg/kg IV daily for 2 days
Overall Study
STARTED
15
Overall Study
COMPLETED
15
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
BMT Allogenic Transplantation
n=15 Participants
Fludarabine, Busulfan, Cyclophosphamide, Tacrolimus,: Fludarabine 30 mg/m2 IV once a day for 4 days Busulfan 0.8 mg/kg IV every 12 hours for 4 days Cyclophosphamide 50 mg/kg IV daily for 2 days
Age, Continuous
49 years
n=99 Participants
Age, Categorical
<=18 years
1 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
12 Participants
n=99 Participants
Age, Categorical
>=65 years
2 Participants
n=99 Participants
Sex/Gender, Customized
Female
6 participants
n=99 Participants
Sex/Gender, Customized
Male
9 participants
n=99 Participants
Region of Enrollment
United States
15 participants
n=99 Participants

PRIMARY outcome

Timeframe: Day 60

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants achieving full-donor chimerism in unsorted peripheral blood.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 60

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants achieving full-donor chimerism in CD3+ sorted peripheral blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants alive

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants alive without disease relapse or progression.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants experiencing disease relapse or progression

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants who died due to BMT-related reasons

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.

Percentage of participants experiencing acute and chronic GVHD. Acute GVHD is graded by Przepiorka criteria. Chronic GVHD is graded by NIH consensus criteria and Seattle criteria.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 60

Percentage of participants who failed to engraft.

Outcome measures

Outcome measures
Measure
BMT Allogenic Transplantation
n=15 Participants
Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil.
Graft Failure
8 Participants

Adverse Events

Transplant

Serious events: 11 serious events
Other events: 7 other events
Deaths: 9 deaths

Serious adverse events

Serious adverse events
Measure
Transplant
n=15 participants at risk
Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil.
Infections and infestations
Infection
46.7%
7/15 • Number of events 7 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Gastrointestinal disorders
Diarrhea
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
General disorders
Fever
13.3%
2/15 • Number of events 2 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Blood and lymphatic system disorders
Febrile neutropenia
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Respiratory, thoracic and mediastinal disorders
Hypoxia
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
General disorders
Chills
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Metabolism and nutrition disorders
Dehydration
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Respiratory, thoracic and mediastinal disorders
Respiratory failure
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Infections and infestations
Sepsis
13.3%
2/15 • Number of events 2 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.

Other adverse events

Other adverse events
Measure
Transplant
n=15 participants at risk
Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil.
Gastrointestinal disorders
nausea
6.7%
1/15 • Number of events 2 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
General disorders
fever
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Nervous system disorders
tremor
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Psychiatric disorders
hallucinations
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Investigations
Elevated liver enzymes
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Investigations
Elevated total bilirubin
6.7%
1/15 • Number of events 1 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Respiratory, thoracic and mediastinal disorders
Hypoxia
13.3%
2/15 • Number of events 2 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Infections and infestations
Infection
26.7%
4/15 • Number of events 4 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
Infections and infestations
Febrile neutropenia
26.7%
4/15 • Number of events 4 • 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.

Additional Information

Yvette Kasamon

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Phone: 410-955-8839

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place