Trial Outcomes & Findings for Busulfan Plus Clofarabine Followed by Allogeneic Hematopoietic Stem Cell Transplantation (NCT NCT00990249)
NCT ID: NCT00990249
Last Updated: 2018-08-07
Results Overview
NRM was defined as death from any cause other than disease progression or relapse and reported as percentage of participant deaths. Treatment related deaths after transplant are defined either by deaths which could not be attributed to disease relapse or progression or by deaths without previous relapse or progression. For TRM at day 100, Bayesian method of Thall, Simon, and Estey used to perform interim monitoring.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
120 participants
Primary outcome timeframe
100 Days
Results posted on
2018-08-07
Participant Flow
Recruitment Period: October 01, 2009 to July 13, 2015. All recruitment done at The University of Texas MD Anderson Cancer Center.
Participant milestones
| Measure |
Busulfan + Clofarabine + Stem Cell Transplant
Busulfan test dose 32 mg/m\^2 intravenous (IV) Day -8; following doses Days -6 to -3 derived from pharmacokinetic (PK) testing done up to 11 times over 11 hours after test dose. Clofarabine 40 mg/m\^2 IV daily Day -6 through Day -3. Thymoglobulin 0.5 mg/kg Day -3, 1.5 mg/kg Day -2 \& 2.0 mg/kg Day -1 for HLA nonidentical or unrelated donors. Stem cell infusion on Day 0.
|
|---|---|
|
Overall Study
STARTED
|
120
|
|
Overall Study
COMPLETED
|
107
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Busulfan + Clofarabine + Stem Cell Transplant
Busulfan test dose 32 mg/m\^2 intravenous (IV) Day -8; following doses Days -6 to -3 derived from pharmacokinetic (PK) testing done up to 11 times over 11 hours after test dose. Clofarabine 40 mg/m\^2 IV daily Day -6 through Day -3. Thymoglobulin 0.5 mg/kg Day -3, 1.5 mg/kg Day -2 \& 2.0 mg/kg Day -1 for HLA nonidentical or unrelated donors. Stem cell infusion on Day 0.
|
|---|---|
|
Overall Study
Disease Progression
|
8
|
|
Overall Study
Non-Compliance
|
1
|
|
Overall Study
Death
|
2
|
|
Overall Study
Not treated
|
2
|
Baseline Characteristics
Busulfan Plus Clofarabine Followed by Allogeneic Hematopoietic Stem Cell Transplantation
Baseline characteristics by cohort
| Measure |
Busulfan + Clofarabine + Stem Cell Transplant
n=120 Participants
Busulfan test dose 32 mg/m\^2 IV Day -8; following doses Days -6 to -3 derived from PK testing. Clofarabine 40 mg/m\^2 IV daily Day -6 through Day -3. Thymoglobulin 0.5 mg/kg Day -3, 1.5 mg/kg Day -2 \& 2.0 mg/kg Day -1 for HLA nonidentical or unrelated donors. Stem cell infusion on Day 0.
|
|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
117 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
38 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
47 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
73 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
86 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
8 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
91 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
12 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
120 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 100 DaysPopulation: Only 107 participants were evaluated.
NRM was defined as death from any cause other than disease progression or relapse and reported as percentage of participant deaths. Treatment related deaths after transplant are defined either by deaths which could not be attributed to disease relapse or progression or by deaths without previous relapse or progression. For TRM at day 100, Bayesian method of Thall, Simon, and Estey used to perform interim monitoring.
Outcome measures
| Measure |
Busulfan + Clofarabine + Stem Cell Transplant
n=107 Participants
Busulfan test dose 32 mg/m\^2 IV Day -8; following doses Days -6 to -3 derived from PK testing. Clofarabine 40 mg/m\^2 IV daily Day -6 through Day -3. Thymoglobulin 0.5 mg/kg Day -3, 1.5 mg/kg Day -2 \& 2.0 mg/kg Day -1 for HLA nonidentical or unrelated donors. Stem cell infusion on Day 0.
|
|---|---|
|
Treatment-Related Mortality (TRM) Defined as Non Relapse Mortality (NRM)
|
10 percentage of participants
|
Adverse Events
Busulfan + Clofarabine + Stem Cell Transplant
Serious events: 3 serious events
Other events: 118 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Busulfan + Clofarabine + Stem Cell Transplant
n=120 participants at risk
Busulfan test dose 32 mg/m\^2 IV Day -8; following doses Days -6 to -3 derived from PK testing. Clofarabine 40 mg/m\^2 IV daily Day -6 through Day -3. Thymoglobulin 0.5 mg/kg Day -3, 1.5 mg/kg Day -2 \& 2.0 mg/kg Day -1 for HLA nonidentical or unrelated donors. Stem cell infusion on Day 0.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Diffuse alveolar hemorrhage (DAH)
|
0.83%
1/120 • Number of events 1 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Infections and infestations
Infection/Sepsis
|
0.83%
1/120 • Number of events 1 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
0.83%
1/120 • Number of events 1 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
Other adverse events
| Measure |
Busulfan + Clofarabine + Stem Cell Transplant
n=120 participants at risk
Busulfan test dose 32 mg/m\^2 IV Day -8; following doses Days -6 to -3 derived from PK testing. Clofarabine 40 mg/m\^2 IV daily Day -6 through Day -3. Thymoglobulin 0.5 mg/kg Day -3, 1.5 mg/kg Day -2 \& 2.0 mg/kg Day -1 for HLA nonidentical or unrelated donors. Stem cell infusion on Day 0.
|
|---|---|
|
Investigations
Bilirubin
|
31.7%
38/120 • Number of events 38 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Investigations
Transaminitis
|
75.8%
91/120 • Number of events 91 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Vascular disorders
Veno-occlusive disease (VOD)
|
5.0%
6/120 • Number of events 6 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Gastrointestinal disorders
Diarrhea
|
46.7%
56/120 • Number of events 56 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Gastrointestinal disorders
Nausea
|
56.7%
68/120 • Number of events 68 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Gastrointestinal disorders
Mucositis
|
87.5%
105/120 • Number of events 105 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Investigations
Creatinine
|
9.2%
11/120 • Number of events 11 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Skin and subcutaneous tissue disorders
Skin Rash
|
18.3%
22/120 • Number of events 22 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Nervous system disorders
Headache
|
5.0%
6/120 • Number of events 6 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Vascular disorders
Hypertension (HTN)
|
13.3%
16/120 • Number of events 16 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Cardiac disorders
Cardiac
|
3.3%
4/120 • Number of events 4 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Blood and lymphatic system disorders
Fluid Overload
|
37.5%
45/120 • Number of events 45 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
8.3%
10/120 • Number of events 10 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath (SOB)
|
1.7%
2/120 • Number of events 2 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Diffuse Alveolar Hemorrahge (DAH)
|
0.83%
1/120 • Number of events 1 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Infections and infestations
Infection
|
76.7%
92/120 • Number of events 248 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Blood and lymphatic system disorders
Fever
|
35.8%
43/120 • Number of events 47 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
|
Blood and lymphatic system disorders
Neutropenic Fever
|
52.5%
63/120 • Number of events 65 • The specific period was from the start of preparative regiment up to Day 100 for the collection of adverse events.
|
Additional Information
Partow Kebriaei, MD/Professor, Stem Cell Transplantation
The University of Texas (UT) MD Anderson Cancer Center
Phone: 713-792-7734
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place