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High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors

Autologous Blood and Marrow Transplantation for Hematologic Malignancies and Selected Solid Tumors

Status
Completed
Phases
NA
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT00536601
Enrollment
174
Registered
2007-09-28
Start date
2006-06-29
Completion date
2018-07-09
Last updated
2021-04-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Burkitt Lymphoma, Childhood Diffuse Large Cell Lymphoma, Childhood Immunoblastic Large Cell Lymphoma, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor (PNET), Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Intraocular Lymphoma, Nodal Marginal Zone B-cell Lymphoma, Peripheral T-cell Lymphoma, Plasma Cell Neoplasm, Primary Systemic Amyloidosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Malignant Testicular Germ Cell Tumor, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Neuroblastoma, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Multiple Myeloma, Regional Neuroblastoma, Splenic Marginal Zone Lymphoma, Testicular Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific, Waldenström Macroglobulinemia

Brief summary

This pilot trial studies different high-dose chemotherapy regimens with or without total-body irradiation (TBI) to compare how well they work when given before autologous stem cell transplant (ASCT) in treating patients with hematologic cancer or solid tumors. Giving high-dose chemotherapy with or without TBI before ASCT stops the growth of cancer cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood or bone marrow and stored. More chemotherapy may be given to prepare for the stem cell transplant. The stem cells are then returned to the patient to replace the blood forming cells that were destroyed by the chemotherapy.

Detailed description

PRIMARY OBJECTIVES: I. Estimate the progression free survival (PFS) distribution for Hodgkin lymphoma (HL), non-Hodgkin lymphoma (NHL) and multiple myeloma (MM) for each disease-specific high dose therapy regimen. SECONDARY OBJECTIVES: I. Estimate the PFS distribution for amyloidosis, acute leukemia and selected solid tumors for each disease-specific high dose therapy regimen. II. Explore the role of risk factors in the outcome of all treated patients. III. Examine the high dose therapy regimen-related toxicity (RRT) and overall survival after bone marrow transplant (BMT). OUTLINE: Patients are assigned to conditioning regimens based on disease, age, and co-morbidities.

Interventions

PROCEDUREautologous hematopoietic stem cell transplantation

Undergo ASCT

DRUGetoposide

Given IV

DRUGcyclophosphamide

Given IV

DRUGcarmustine

Given IV

DRUGmelphalan

Given IV

DRUGbusulfan

Given IV

DRUGcarboplatin

Given IV

DRUGthiotepa

Given IV

RADIATIONtotal-body irradiation

Undergo TBI

Sponsors

National Cancer Institute (NCI)
CollaboratorNIH
Roswell Park Cancer Institute
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No

Inclusion criteria

* Histologically confirmed diagnosis of malignant hematologic disorders, amyloidosis or solid tumor malignancy * Recurrent or refractory disease or disease at high risk for recurrence * Hodgkin Disease (HL): Relapsed or refractory disease after chemotherapy with a minimum of one standard regimen * Non-Hodgkin Lymphoma (NHL): (Low, Intermediate or High Grade) Relapsed or refractory disease after chemotherapy with at least one standard regimen or first complete remission (CR) lymphoblastic or small, non-cleaved cell lymphoma at high risk of relapse by high International Prognostic Index (IPI) Score * Acute Myeloid Leukemia (AML): Low or High Risk disease in first or second CR or greater in patients in whom the risks of an allogeneic transplant outweigh the benefits * Acute Lymphoblastic Leukemia (ALL): Low or high risk disease in first or second CR in whom the risks of an allogeneic transplant outweigh the benefits * Multiple Myeloma (MM): Low or high risk in first or greater response (stable disease or better) or for responding patients at first progression * Other Malignant Lymphoproliferative Disorders: (chronic lymphocytic lymphoma \[CLL\], Waldenstroms macroglobulinemia, relapsed or refractory disease after first-line chemotherapy * Amyloidosis: primary or previously treated * Solid Tumors: Testicular cancer patients who have relapsed disease or primary progressive disease which is responding to salvage therapy; relapsed or advanced-stage newly diagnosed neuroblastoma (NBL) or small round blue cell tumors (SRBCT) in patients 30 years of age; other patients with solid tumors who have recurred following conventional treatment or are at high risk for relapse, and demonstrate chemosensitivity * Patients with malignancies who would be treated with an autologous stem cell transplant but have a syngeneic donor; a syngeneic donor would be considered to have the same risk as an autologous stem cell transplant patient * Performance status 0-2 (Karnofsky performance status \[KPS\] \>= 70%); patients with amyloidosis or MM with decreased KPS due to disease are eligible * Life expectancy \> 2 months * Pulmonary function tests; diffusing capacity of the lung for carbon monoxide (DLCO) or diffusing volume of the alveolar volume (DLVA) \>= 50% predicted; DLCO to be corrected for hemoglobin and/or alveolar ventilation * Cardiac ventricular ejection fraction \>= 50% by radionuclide ventriculogram or echocardiogram * Bilirubin \< 3 x normal * Alkaline phosphatase, serum glutamic oxaloacetic transaminase (SGOT) \< 3 x normal * Calculated creatinine clearance \< 40 cc/min by the modified Cockcroft-Gault formula for adults or the Schwartz formula for pediatrics * Glomerular filtration rate by renal scan for neuroblastoma patients, to determine dosing parameters * Positive cytomegalovirus (CMV) immunoglobulin M (IgM) and/or positive hepatitis serologies demonstrating infection will require an Infectious Disease consult and subsequent clearance * Any active infection will require an Infectious Disease consult and subsequent clearance * Peripheral Blood Counts of polymorphonuclear neutrophil (PMN) \> 1500/uL * Platelet (Plt) \> 75,000/uL * Prior to stem cell storage: * No radiation within three weeks before stem cell harvest * Bone marrow may be used in conjunction with blood progenitor cells * Hematologic Malignancy patients with human immunodeficiency virus (HIV) positivity but on appropriate anti-retroviral therapy may go autotransplant with the following laboratory tests; (CD4+ cell count \> 75 cells per microliter and HIV copy number \< 100,000 per microliter and with Infectious Disease clearance * Acute Leukemia, HL, NHL, MM and Solid Tumor patients must have received 2 cycles of chemotherapy followed by disease-specific restaging prior to mobilization and collection of stem cells; small round blue cell tumor patients must have received either standard therapy or surgical intervention; the disease status and response to therapy must be known prior to transplant to establish the disease status at transplant; amyloidosis patients may proceed to BMT without receiving chemotherapy * No serious organ dysfunction unless it is caused by the underlying disease,

Exclusion criteria

include the following: * Uncontrolled or severe cardiovascular disease, including recent (\< 6 months) myocardial infarction, congestive heart failure, symptomatic angina, life-threatening arrhythmia or hypertension * Active bacterial, viral, or fungal infection * Active peptic ulcer disease * Uncontrolled diabetes mellitus * No serious medical or psychiatric illness * Not pregnant * No psychiatric conditions which would prevent delivery of care; psychology clearance is necessary * Allogeneic BMT not possible, or not desirable * Age \> 65 years * No compatible donor identified * Estimated risk of graft vs. host disease complications greater than risk of recurrence after autologous BMT * Adequate bone marrow or blood stem cell dose obtained: * For blood stem cells: total CD 34+ \>= 2 x 10\^6/kg or if unable to collect this dose, a total nucleated cell bone marrow dose of \>= l x 10\^8/kg

Design outcomes

Primary

MeasureTime frameDescription
Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 yearsAssessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed.

Secondary

MeasureTime frameDescription
Regimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))Up to 100 days after transplantationToxicities will be reported using descriptive statistics.
Response Rate (Complete Remission)At 100 daysResponse rates will be reported using descriptive statistics.
Overall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)Patients are followed up to maximum of 12 yearsAssessed using the product-limit based Kaplan Meier method.

Countries

United States

Participant flow

Participants by arm

ArmCount
Acute Leukemia
Patients diagnosed with and treated for acute myeloid leukemia or acute lymphoblastic leukemia
6
Hodgkin Lymphoma
Patients diagnosed with and treated for Hodgkin Lymphoma
21
Non-Hodgkin Lymphoma
Patients diagnosed with and treated for Non-Hodgkin Lymphoma
71
MM/Amyloid
Patients diagnosed with or treated for multiple myeloma or amyloidosis
65
Solid Tumors
Patients diagnosed with and treated for a solid tumor
11
Total174

Baseline characteristics

CharacteristicAcute LeukemiaHodgkin LymphomaNon-Hodgkin LymphomaMM/AmyloidSolid TumorsTotal
Age, Customized
0-39 years
1 Participants8 Participants6 Participants3 Participants9 Participants27 Participants
Age, Customized
40-59 years
1 Participants7 Participants43 Participants26 Participants2 Participants79 Participants
Age, Customized
60-75 years
4 Participants6 Participants22 Participants36 Participants0 Participants68 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants1 Participants1 Participants0 Participants0 Participants2 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
6 Participants20 Participants70 Participants65 Participants11 Participants172 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Black or African American
2 Participants1 Participants3 Participants8 Participants1 Participants15 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
White
4 Participants20 Participants68 Participants57 Participants10 Participants159 Participants
Region of Enrollment
United States
6 participants21 participants71 participants65 participants11 participants174 participants
Risk Group
High
6 Participants10 Participants43 Participants10 Participants11 Participants80 Participants
Risk Group
Standard
0 Participants11 Participants24 Participants44 Participants0 Participants79 Participants
Risk Group
Unknown
0 Participants0 Participants4 Participants11 Participants0 Participants15 Participants
Sex: Female, Male
Female
5 Participants11 Participants23 Participants29 Participants3 Participants71 Participants
Sex: Female, Male
Male
1 Participants10 Participants48 Participants36 Participants8 Participants103 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
EG002
affected / at risk
EG003
affected / at risk
EG004
affected / at risk
deaths
Total, all-cause mortality
4 / 68 / 2141 / 7145 / 656 / 11
other
Total, other adverse events
0 / 60 / 210 / 710 / 650 / 11
serious
Total, serious adverse events
1 / 69 / 2119 / 7131 / 653 / 11

Outcome results

Primary

Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed.

Time frame: From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 years

Population: Each disease strata presents PFS for each conditioning regimen defined in the protocol

ArmMeasureGroupValue (NUMBER)
Acute LeukemiaProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)BuCy33 Proportion of participants
Hodgkin LymphomaProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)CBV43 Proportion of participants
Hodgkin LymphomaProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)BuCy14 Proportion of participants
Non-Hodgkin LymphomaProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)VCp0 Proportion of participants
Non-Hodgkin LymphomaProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)BuCy22 Proportion of participants
Non-Hodgkin LymphomaProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)CBV37 Proportion of participants
MM/AmyloidProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)Mel12022 Proportion of participants
MM/AmyloidProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)Mel20013 Proportion of participants
Solid TumorsProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)VCp43 Proportion of participants
Solid TumorsProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)CyTtCp0 Proportion of participants
Solid TumorsProgression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)TtC150050 Proportion of participants
Secondary

Overall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method.

Time frame: Patients are followed up to maximum of 12 years

ArmMeasureValue (NUMBER)
Acute LeukemiaOverall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)33 Proportion of participants
Hodgkin LymphomaOverall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)61 Proportion of participants
Non-Hodgkin LymphomaOverall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)45 Proportion of participants
MM/AmyloidOverall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)39 Proportion of participants
Solid TumorsOverall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)46 Proportion of participants
Secondary

Regimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))

Toxicities will be reported using descriptive statistics.

Time frame: Up to 100 days after transplantation

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Acute LeukemiaRegimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))1 Participants
Hodgkin LymphomaRegimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))9 Participants
Non-Hodgkin LymphomaRegimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))19 Participants
MM/AmyloidRegimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))31 Participants
Solid TumorsRegimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))3 Participants
Secondary

Response Rate (Complete Remission)

Response rates will be reported using descriptive statistics.

Time frame: At 100 days

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Acute LeukemiaResponse Rate (Complete Remission)5 Participants
Hodgkin LymphomaResponse Rate (Complete Remission)17 Participants
Non-Hodgkin LymphomaResponse Rate (Complete Remission)54 Participants
MM/AmyloidResponse Rate (Complete Remission)17 Participants
Solid TumorsResponse Rate (Complete Remission)7 Participants

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026