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Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer

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ClinicalTrials.gov Identifier: NCT00134017
Recruitment Status : Completed
First Posted : August 24, 2005
Results First Posted : August 31, 2018
Last Update Posted : August 31, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Chronic Myeloproliferative Disorders
Leukemia
Lymphoma
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Interventions Drug: Busulfan
Drug: Cyclophosphamide
Enrollment 142
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Period Title: Overall Study
Started 142
Completed 142
Not Completed 0
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Number of Baseline Participants 142
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 142 participants
<=18 years
16
  11.3%
Between 18 and 65 years
123
  86.6%
>=65 years
3
   2.1%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 142 participants
47
(21 to 66)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 142 participants
Female
66
  46.5%
Male
76
  53.5%
1.Primary Outcome
Title Percentage of Participants Who Develop Acute Graft-versus-host Disease (GVHD)
Hide Description Percentage of participants who developed grades II-IV and grades III-IV acute GVHD. Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade I (skin involvement stages 1+ to 2+, with no liver or GI involvement), Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. GVHD data was collected on 15 additional participants for a total of 132. GVHD data was not collected on the remaining 10 participants.
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:
Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Number of Participants Analyzed 132
Measure Type: Count of Participants
Unit of Measure: Participants
Grades II-IV acute GVHD
49
  37.1%
Grades III-IV acute GVHD
10
   7.6%
2.Secondary Outcome
Title Days to Engraftment
Hide Description Median number of days to neutrophil and platelet engraftment.
Time Frame Up to one year
Hide Outcome Measure Data
Hide Analysis Population Description
The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. Engraftment data was not collected on the remaining 25 participants. Recipients of related-donor (n=78) and unrelated-donor (n=39) transplants were reported separately.
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:
Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Number of Participants Analyzed 117
Median (Full Range)
Unit of Measure: days
Neutrophil engraftment, related donors Number Analyzed 78 participants
23
(2 to 100)
Neutrophil engraftment, unrelated donors Number Analyzed 39 participants
25
(2 to 100)
Platelet engraftment, related donors Number Analyzed 78 participants
31
(5 to 354)
Platelet engraftment, unrelated donors Number Analyzed 39 participants
35
(5 to 354)
3.Secondary Outcome
Title Chimerism
Hide Description Number of patients who achieved 100% donor chimerism.
Time Frame Day 30, Day 60
Hide Outcome Measure Data
Hide Analysis Population Description
The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. Chimerism data was not collected on the remaining 25 participants. One participant died prior to Day 30 and was not analyzed for this outcome.
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:
Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Number of Participants Analyzed 116
Measure Type: Count of Participants
Unit of Measure: Participants
Day 30
99
  85.3%
Day 60
101
  87.1%
4.Secondary Outcome
Title Non-relapse Mortality
Hide Description Percentage of participants who died for BMT-related reasons.
Time Frame Day 100, 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. NRM data was not collected on the remaining 25 participants.
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:
Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Number of Participants Analyzed 117
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Day 100
6
(2 to 13)
2 years
15
(8 to 24)
5.Secondary Outcome
Title Relapse
Hide Description Percentage of participants who developed relapse or progressive disease.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The study data was analyzed early after 117 participants had been treated. This was done in order to publish the data and establish a new local standard of care at our institution. Relapse data was not collected on the remaining 25 participants.
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:
Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis.
Overall Number of Participants Analyzed 117
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
44
(34 to 53)
Time Frame Up to one year
Adverse Event Reporting Description

Adverse events were tracked systematically for 30-60 days depending on exact discharge date from the initial hospitalization.

Only the following adverse events were collected: all graft failures; deaths in the first sixty days; unexpected grade 4-5 events.

 
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description Myeloablative bone marrow transplant with a busulfan (Bu), cyclophosphamide (Cy), preparative regimen and post-transplant cyclophosphamide (PTCy) as GVHD prophylaxis
All-Cause Mortality
Bone Marrow Transplant
Affected / at Risk (%)
Total   66/142 (46.48%)    
Hide Serious Adverse Events
Bone Marrow Transplant
Affected / at Risk (%) # Events
Total   20/142 (14.08%)    
Hepatobiliary disorders   
Veno-occlusive disease  1  4/142 (2.82%)  4
Immune system disorders   
Graft failure  1  6/142 (4.23%)  6
Graft versus host disease  1  1/142 (0.70%)  1
Multiorgan failure  1  1/142 (0.70%)  1
Infections and infestations   
Aspergillus pneumonia  1  1/142 (0.70%)  1
Septic shock  1  1/142 (0.70%)  1
Respiratory, thoracic and mediastinal disorders   
Pulmonary failure  1  7/142 (4.93%)  7
Pulmonary hemorrhage  1  1/142 (0.70%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Bone Marrow Transplant
Affected / at Risk (%) # Events
Total   0/142 (0.00%)    
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Leo Luznik, MD
Organization: Johns Hopkins University
Phone: 4105027732
EMail: luznile@jhmi.edu
Layout table for additonal information
Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT00134017    
Other Study ID Numbers: J0373
P01CA015396 ( U.S. NIH Grant/Contract )
P30CA006973 ( U.S. NIH Grant/Contract )
NA_00034100 ( Other Identifier: JHMIRB )
First Submitted: August 22, 2005
First Posted: August 24, 2005
Results First Submitted: July 31, 2018
Results First Posted: August 31, 2018
Last Update Posted: August 31, 2018