Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine (NCT NCT00987480)
NCT ID: NCT00987480
Last Updated: 2018-07-10
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
45 participants
Primary outcome timeframe
2 years
Results posted on
2018-07-10
Participant Flow
This is a multicenter site and MSK is the site responsible for data collection and analysis for all sites. 11 participants were enrolled at Memorial Sloan Kettering Cancer Center. The remaining 34 participants were enrolled at the participating institutions. This makes a total of 45 participants enrolled and treated on this study.
Participant milestones
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
|
|---|---|
|
Overall Study
STARTED
|
45
|
|
Overall Study
COMPLETED
|
45
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
Baseline characteristics by cohort
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
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|---|---|
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Age, Continuous
|
8.2 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
25 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
40 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
36 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
45 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
|
|---|---|
|
Successful Neutrophil Engraftment
Achieved neutrophil engraftment
|
44 Participants
|
|
Successful Neutrophil Engraftment
Did not achieve neutrophil engraftment
|
1 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
|
|---|---|
|
The Incidence of Early Transplant Related Mortality
|
0 Participants
|
PRIMARY outcome
Timeframe: 100 daysOutcome measures
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
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|---|---|
|
The Incidence of Acute GvHD
|
6.7 percentage of participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
|
|---|---|
|
The Incidence of Chronic GvHD
Developed limited chronic GVHD
|
3 Participants
|
|
The Incidence of Chronic GvHD
Did not develop limited chronic GVHD
|
42 Participants
|
SECONDARY outcome
Timeframe: 3 yearsOverall Survival is defined as time from date of transplant to event (death from any cause) or last follow-up.
Outcome measures
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
|
|---|---|
|
Overall Survival at 3 Years
|
80 percentage of participants
|
SECONDARY outcome
Timeframe: 3 yearsDefined as time from date of transplant to relapse, graft rejection or graft failure, or death. Primary non-engraftment is diagnosed when the participants fails to achieve an ANC \>/= 500/ul at any time in the first 28 days post-transplant. For participants with MDS or AML, relapse will be analyzed as to type and genetic origin of the MDS/leukemic cells. These will be defined by an increasing number of blasts in the marrow over 5% by the presence of circulating peripheral blasts, or by the presence of blasts in any extramedullary site. Cytogenetic analysis of the marrow and/or peripheral blood will also be obtained for the diagnosis of relapse.
Outcome measures
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 Participants
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.
Busulfan, fludarabine, \& cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.
|
|---|---|
|
Disease-free Survival at 3 Years
|
77.8 percentage of participants
|
Adverse Events
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
Serious events: 11 serious events
Other events: 31 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 participants at risk
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease
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|---|---|
|
Investigations
Bilirubin (hyperbilirubinemia)
|
2.2%
1/45 • 1 year
|
|
Blood and lymphatic system disorders
Blood/Bone Marrow, Other
|
2.2%
1/45 • 1 year
|
|
Cardiac disorders
Cardiopulmonary arrest, cause unknkown
|
2.2%
1/45 • 1 year
|
|
Psychiatric disorders
Confusion
|
2.2%
1/45 • 1 year
|
|
General disorders
Death NOS
|
6.7%
3/45 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
2.2%
1/45 • 1 year
|
|
Gastrointestinal disorders
Gastrointestinal, other
|
2.2%
1/45 • 1 year
|
|
Gastrointestinal disorders
Hemorrhage, Oral cavity
|
2.2%
1/45 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, Respiratory tract NOS
|
2.2%
1/45 • 1 year
|
|
Gastrointestinal disorders
Hemorrhage, Stoma (GI)
|
2.2%
1/45 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
15.6%
7/45 • 1 year
|
|
Gastrointestinal disorders
Ileus, GI (func obstruction of bowel)
|
2.2%
1/45 • 1 year
|
|
Infections and infestations
Inf norm ANC/gr1/2 neut-Blood
|
2.2%
1/45 • 1 year
|
|
Infections and infestations
Inf norm ANC/gr1/2 neut-Meningitis(meninges)
|
2.2%
1/45 • 1 year
|
|
Infections and infestations
Inf norm ANC/gr1/2 neut-Myositis infection(muscle)
|
2.2%
1/45 • 1 year
|
|
Infections and infestations
Infection w/ Gr 3/4 neut, Blood
|
2.2%
1/45 • 1 year
|
|
Infections and infestations
Infection, other
|
8.9%
4/45 • 1 year
|
|
Gastrointestinal disorders
Mucositis (Clin exam)- Oral cavity
|
2.2%
1/45 • 1 year
|
|
Nervous system disorders
Neurology - Other (specify)
|
2.2%
1/45 • 1 year
|
|
Nervous system disorders
Neuropathy: motor
|
2.2%
1/45 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/pulm infiltrates
|
4.4%
2/45 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulm/upp respiratory - Other (spec)
|
2.2%
1/45 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
2.2%
1/45 • 1 year
|
|
Nervous system disorders
Seizure
|
2.2%
1/45 • 1 year
|
Other adverse events
| Measure |
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
n=45 participants at risk
This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease
|
|---|---|
|
Investigations
Aspartate aminotransferase increased
|
6.7%
3/45 • 1 year
|
|
Investigations
Bicarbonate, serum-low
|
6.7%
3/45 • 1 year
|
|
Investigations
Blood bilirubin increased
|
6.7%
3/45 • 1 year
|
|
Metabolism and nutrition disorders
Sodium, high (hypernatremia)
|
6.7%
3/45 • 1 year
|
|
Metabolism and nutrition disorders
Glucose, low (hypoglycemia)
|
8.9%
4/45 • 1 year
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
8.9%
4/45 • 1 year
|
|
Gastrointestinal disorders
Mucositis (Clin exam)- Pharynx
|
8.9%
4/45 • 1 year
|
|
Metabolism and nutrition disorders
Phosphate, low (hypophosphatemia)
|
8.9%
4/45 • 1 year
|
|
Investigations
Alanine aminotransferase increased
|
11.1%
5/45 • 1 year
|
|
Infections and infestations
Infection, other
|
11.1%
5/45 • 1 year
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
13.3%
6/45 • 1 year
|
|
Investigations
INR
|
13.3%
6/45 • 1 year
|
|
Metabolism and nutrition disorders
Magnesium, high (hypermagnesemia)
|
13.3%
6/45 • 1 year
|
|
Metabolism and nutrition disorders
Magnesium, low (hypomagnesemia)
|
13.3%
6/45 • 1 year
|
|
Metabolism and nutrition disorders
Trglycrde, high (hypertriglyceridemia)
|
13.3%
6/45 • 1 year
|
|
Investigations
Alkaline phosphatase
|
15.6%
7/45 • 1 year
|
|
Metabolism and nutrition disorders
Potassium, high (hyperkalemia)
|
15.6%
7/45 • 1 year
|
|
Investigations
PTT
|
15.6%
7/45 • 1 year
|
|
Metabolism and nutrition disorders
Albumin, low (hypoalbuminemia)
|
17.8%
8/45 • 1 year
|
|
Investigations
Creatinine
|
17.8%
8/45 • 1 year
|
|
Investigations
Hemoglobin
|
17.8%
8/45 • 1 year
|
|
Vascular disorders
Hypertension
|
17.8%
8/45 • 1 year
|
|
Investigations
Leukocytes (total WBC)
|
17.8%
8/45 • 1 year
|
|
Blood and lymphatic system disorders
Lymphopenia
|
17.8%
8/45 • 1 year
|
|
Investigations
Neutrophils/granulocytes (ANC/AGC)
|
17.8%
8/45 • 1 year
|
|
Investigations
Platelets
|
17.8%
8/45 • 1 year
|
|
Metabolism and nutrition disorders
Sodium, low (hyponatremia)
|
20.0%
9/45 • 1 year
|
|
Investigations
AST, SGOT
|
22.2%
10/45 • 1 year
|
|
Metabolism and nutrition disorders
Potassium, low (hypokalemia)
|
22.2%
10/45 • 1 year
|
|
Investigations
ALT, SGPT
|
24.4%
11/45 • 1 year
|
|
Investigations
Bilirubin (hyperbilirubinemia)
|
24.4%
11/45 • 1 year
|
|
Metabolism and nutrition disorders
Glucose, high (hyperglycemia)
|
31.1%
14/45 • 1 year
|
|
Gastrointestinal disorders
Mucositis (Clin exam)- Oral cavity
|
33.3%
15/45 • 1 year
|
Additional Information
Dr. Farid Boulad
Memorial Sloan Kettering Cancer Center
Phone: 212-639-2429
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place