NCT00053989 · REGISTRY CONDITION SEARCH

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2020-02-10

RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving immunosuppressive therapy before or after the transplant may stop this from happening. PURPOSE: This phase II trial is studying how well chemotherapy followed by donor peripheral stem cell transplant works in treating patients with hematologic cancer or aplastic anemia.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Day 100 TRM

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outcome · POSTED

Day 100 Best Response

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outcome · POSTED

PFS

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outcome · POSTED

OS

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outcome · POSTED

Acute GvHD

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

eligibility Criteria: DISEASE CHARACTERISTICS: * Diagnosis of aplastic anemia * Severe disease * Failed at least 1 course of standard immunosuppressive regimen with cyclosporine and anti-thymocyte globulin OR * Histologically confirmed hematologic malignancy including the following: * Acute leukemia * Any of the following types: * Acute myeloid leukemia (AML) with antecedent myelodysplastic syndromes * Secondary AML * AML with high-risk cytogenetic abnormalities * Acute lymphoblastic leukemia with high-risk cytogenetic abnormalities * Resistant or recurrent disease after combination chemotherapy with at least 1 standard regimen OR * In first remission at high risk of relapse * Chronic myelogenous leukemia * Chronic phase meeting at least 1 of the following criteria: * Failed imatinib mesylate * Failed interferon after at least 6 months of treatment with minimum of 21 million units of interferon per week * Unable to tolerate interferon * Accelerated phase (blasts less than 20%) * Myeloproliferative and myelodysplastic syndromes * Myelofibrosis (after splenectomy) * Refractory anemia * Refractory anemia with excess blasts * Chronic myelomonocytic leukemia * Lymphoproliferative disease * Chronic lymphocytic leukemia * Symptomatic disease after first-line chemotherapy * Low-grade non-Hodgkin's lymphoma (recurrent or persistent) * Symptomatic disease after first-line chemotherapy * Multiple myeloma * Progressive disease after autologous stem cell transplantation * Waldenstrom's macroglobulinemia * Failed 1 standard regimen * Non-Hodgkin's lymphoma meeting the following criteria: * Intermediate or high grade * Controlled and chemosensitive disease * First remission lymphoblastic or small non-cleaved cell lymphoma at high risk of relapse * Hodgkin's lymphoma * Relapsed and chemosensitive disease * Not eligible for standard myeloablative allogeneic stem cell transplantation * Availability of any of the following donor types: * Related donor matched at 5 or 6 HLA antigens (A, B, DR) * Unrelated donor fully matched by molecular analysis at A, B, DRB1, and DQB1 loci * Single antigen mismatch at C allowed * Cord blood that is 4, 5, or 6 match with recipient HLA antigens (A, B, DR) NOTE: No syngeneic donors permitted * No uncontrolled CNS disease (for hematologic malignancies) NOTE: A new classification scheme for adult non-Hodgkin's lymphoma has been adopted by PDQ. The terminology of "indolent" or "aggressive" lymphoma will replace the former terminology of "low", "intermediate", or "high" grade lymphoma. However, this protocol uses the former terminology. PATIENT CHARACTERISTICS: Age * 4 to 75 (if related or unrelated donor peripheral blood or marrow transplantation) * 4 to 60 (if unrelated cord blood transplantation) Performance status * Karnofsky \> 50% Life expectancy * Not specified Hematopoietic * Not specified Hepatic * Bilirubin less than 3 times normal * Alkaline phosphatase less than 3 times normal * AST/ALT less than 3 times normal * No Child's class B or C liver failure Renal * Creatinine clearance greater than 40 mL/min Cardiovascular * Cardiac ventricular ejection fraction at least 35% by MUGA * No cardiovascular disease Pulmonary * DLCO at least 40% of predicted, corrected for hemoglobin and/or alveolar ventilation Other * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * HIV antibody negative * No uncontrolled diabetes mellitus * No active serious infection * No other disease that would preclude study therapy * No other concurrent malignancy except non-melanoma skin cancer * No concurrent serious psychiatric illness PRIOR CONCURRENT THERAPY: Biologic therapy * See Disease Characteristics * patients may have received a prior autologous blood or marrow transplantation (BMT) * At least 6 months since prior allogeneic BMT Chemotherapy * See Disease Characteristics * At least 2 weeks since prior chemotherapy, radiation or surgery Endocrine therapy * Not specified Radiotherapy * At least 2 weeks since prior radiotherapy Surgery * At least 2 weeks since prior surgery
healthy Volunteers: false
maximum Age: 75 Years
minimum Age: 4 Years
sex: ALL
std Ages: CHILD
ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
All patients enrolled on study
intervention Names
Biological: anti-thymocyte globulin
Biological: graft-versus-tumor induction therapy
Biological: sargramostim
Biological: therapeutic allogeneic lymphocytes
Drug: cyclophosphamide
Drug: fludarabine phosphate
Drug: methylprednisolone
Drug: mycophenolate mofetil
Drug: tacrolimus
Procedure: allogeneic bone marrow transplantation
Procedure: peripheral blood stem cell transplantation
Procedure: umbilical cord blood transplantation
label
All patients
type
EXPERIMENTAL

interventions

arm Group Labels
All patients
description
iv
name
anti-thymocyte globulin
type
BIOLOGICAL
arm Group Labels
All patients
description
iv
name
graft-versus-tumor induction therapy
type
BIOLOGICAL
arm Group Labels
All patients
description
iv
name
sargramostim
type
BIOLOGICAL
arm Group Labels
All patients
description
iv
name
therapeutic allogeneic lymphocytes
type
BIOLOGICAL
arm Group Labels
All patients
description
injection
name
cyclophosphamide
type
DRUG
arm Group Labels
All patients
description
iv
name
fludarabine phosphate
other Names
FLUDARA
type
DRUG
arm Group Labels
All patients
description
oral
name
methylprednisolone
type
DRUG
arm Group Labels
All patients
description
oral
name
mycophenolate mofetil
type
DRUG
arm Group Labels
All patients
description
oral
name
tacrolimus
type
DRUG
arm Group Labels
All patients
description
iv
name
allogeneic bone marrow transplantation
type
PROCEDURE
arm Group Labels
All patients
description
iv
name
peripheral blood stem cell transplantation
type
PROCEDURE
arm Group Labels
All patients
description
iv
name
umbilical cord blood transplantation
type
PROCEDURE

Study design

allocation

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 41
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
treatment related mortality within 100 days from hematopoietic stem cell (HSC) infusion on day 0
measure
Day 100 TRM
time Frame
from start or conditioning (day -6 or -5) through day +100 after HSC infusion
description
Best disease response measured within 100 days from hematopoietic stem cell (HSC) infusion on day 0 using disease specific response criteria defined in the protocol
measure
Day 100 Best Response
time Frame
from start of conditioning on day -6 or -5 through day +100 after HSC infusion

secondary Outcomes

description
Progression free survival defined as time from HSC infusion (day 0) until progression of disease or death due to any cause. Patients are censored if alive without disease progression through 1 year after HSC infusion
measure
PFS
time Frame
1 year
description
Overall survival with events defined as death due to any cause and censored patients are alive as of 1 year post HSC infusion
measure
OS
time Frame
1 year
description
overall grade II-IV acute GvHD
measure
Acute GvHD
time Frame
Day +100

Full study description

brief Summary: RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving immunosuppressive therapy before or after the transplant may stop this from happening. PURPOSE: This phase II trial is studying how well chemotherapy followed by donor peripheral stem cell transplant works in treating patients with hematologic cancer or aplastic anemia.
detailed Description: OBJECTIVES: * Determine the safety and toxic effects of nonmyeloablative allogeneic peripheral blood stem cell transplantation in patients with a hematologic malignancy or aplastic anemia. * Determine clinical response and overall outcome of patients treated with this regimen. * Determine the incidence of graft-vs-tumor effect, graft-vs-host disease, and chimerism in patients treated with this regimen. OUTLINE: * Preparative regimen: * Matched related and unrelated donor transplantation: * Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and fludarabine IV over 30 minutes on days -5 to -1. * Cord blood transplantation: * Patients receive the same regimen as above plus anti-thymocyte globulin IV over 4 hours on days -3 to -1. * Graft-vs-host disease (GVHD) prophylaxis: * Matched related and unrelated donor transplantation: * Patients receive oral tacrolimus (or IV) once daily and oral mycophenolate mofetil (MMF) (or IV) twice daily on days -1 to 60 followed by tapering\* of this regimen. Patients then receive methotrexate IV on days 1, 3, and 6. * Cord blood transplantation: * Patients receive tacrolimus and MMF in the same regimen as above plus methylprednisolone twice daily on days 1-19 or until blood counts recover. * Allogeneic stem cell reinfusion: Patients undergo allogeneic bone marrow or peripheral blood stem cell transplantation on day 0. Patients then receive sargramostim (GM-CSF) subcutaneously daily beginning on day 7 and continuing until blood counts recover. * Donor lymphocyte infusion (DLI): Patients not converting to 100% donor T-cell chimerism by day 120 and showing signs of progresson of disease after tacrolimus and MMF withdrawal may receive DLI every 8 weeks for up to 3 infusions. Cord blood recipients do not receive DLI. Patients are followed at day 100-120, every 3 months for 2 years, and then every 6 months for 5 years. PROJECTED ACCRUAL: A total of 30-60 patients will be accrued for this study within 6-7 years.

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice