NCT00253513 · REGISTRY CONDITION SEARCH

Treosulfan and Fludarabine in Treating Younger Patients Who Are Undergoing a Donor Stem Cell Transplant for Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndrome

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

Phase: PHASE1, PHASE2
Status at capture: COMPLETED
Registry last update: 2012-06-01

RATIONALE: Drugs used in chemotherapy, such as treosulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving treosulfan and fludarabine together with a donor bone marrow transplant or a peripheral stem cell transplant may be an effective treatment for acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome. PURPOSE: This phase II trial is studying giving treosulfan together with fludarabine to see how well it works in treating patients who are undergoing a donor stem cell transplant for acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome.

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

Number of Patients Experiencing Regimen-related Toxicity Events in Study Population

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outcome · POSTED

Number of Patients Experiencing Graft Failure

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outcome · POSTED

Incidence (Percent of Participants) With Nonrelapse Mortality (NRM) by Day 200 (Secondary Phase Only)

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outcome · POSTED

Number of Subjects Who Are Without Disease at One Year as Indicator of Disease Free Survival.

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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 acute myeloid leukemia, lymphoblastic leukemia, or myelodysplastic syndrome * Any phase allowed, including any of the following: * Disease in remission * Relapsed or primary refractory disease * No CNS leukemic involvement not clearing with prior intrathecal chemotherapy and/or cranial radiotherapy * Planning to undergo unmanipulated allogeneic bone marrow or peripheral blood stem cell transplantation * Filgrastim (G-CSF) mobilization of bone marrow or stem cells allowed * Donor available, meeting 1 of the following criteria: * HLA-identical related donor * HLA-A, -B, -C, -DRB1, and -DQB1 matched unrelated donor by high-resolution DNA typing * A single allele mismatch allowed PATIENT CHARACTERISTICS: Performance status * Karnofsky 70-100% OR * Lansky 70-100% Life expectancy * Not specified Hematopoietic * Not specified Hepatic * Bilirubin ≤ 2 times upper limit of normal (ULN) * AST ≤ 2 times ULN * No evidence of synthetic dysfunction * No severe cirrhosis * No active infectious hepatitis Renal * Creatinine clearance ≥ 50% * Creatinine ≤ 2 times ULN * Dialysis independent Cardiovascular * No cardiac insufficiency requiring treatment * No symptomatic coronary artery disease * Ejection fraction ≥ 35% (for patients with history of cardiac disease or anthracycline exposure) Pulmonary * PO\_2 ≥ 70 mm Hg AND DLCO ≥ 70% of predicted OR * PO\_2 ≥ 80 mm Hg AND DLCO ≥ 60% of predicted * Not requiring supplementary continuous oxygen Other * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * No other disease that would severely limit life expectancy * No HIV positivity * No active infection requiring deferral of conditioning * No known hypersensitivity to the study drugs PRIOR CONCURRENT THERAPY: Biologic therapy * See Disease Characteristics * No prior allogeneic bone marrow or stem cell transplantation * No concurrent umbilical cord blood or autologous transplantation Chemotherapy * See Disease Characteristics Radiotherapy * See Disease Characteristics Other * More than 4 weeks since prior experimental drugs * Concurrent enrollment on another protocol for graft-versus-host disease prophylaxis allowed
healthy Volunteers: false
maximum Age: 60 Years
sex: ALL
std Ages: CHILD
ADULT

Treatment arms and interventions

interventions

description
30 mg/m2, IV for 5 days
name
fludarabine
type
DRUG
description
12 or 14 g/m2, IV for 5 days
name
treosulfan
type
DRUG
description
bone marrow or peripheral blood stem cells
name
allogeneic blood or bone marrow transplantation
type
PROCEDURE

Study design

allocation

NON_RANDOMIZED

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 60
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Proportion of patients experiencing regimen-related toxicity to major organ systems from day minus 6 to day 28. Major organ systems: cardiac, bladder/renal, pulmonary, hepatic, neurologic and gastrointestinal
measure
Number of Patients Experiencing Regimen-related Toxicity Events in Study Population
time Frame
34 days and 2 years
description
Graft versus Host Disease (GVHD) is a frequent complication of allogeneic bone marrow transplant in which the engrafted donor cells attacks the patient's organs and tissue. Acute GVHD (aGVHD) usually occurs during the first three months following an allogeneic BMT. Chronic GVHD (cGVHD) usually develops after the third month post-transplant. Patients may experience one, both or neither.
measure
Number of Patients Experiencing Graft Failure
time Frame
42 days
description
NRM (Non relapse mortality) - death not attributed to the primary cancer.
measure
Incidence (Percent of Participants) With Nonrelapse Mortality (NRM) by Day 200 (Secondary Phase Only)
time Frame
200 days

secondary Outcomes

measure
Number of Subjects Who Are Without Disease at One Year as Indicator of Disease Free Survival.
time Frame
One year

Full study description

brief Summary: RATIONALE: Drugs used in chemotherapy, such as treosulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving treosulfan and fludarabine together with a donor bone marrow transplant or a peripheral stem cell transplant may be an effective treatment for acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome. PURPOSE: This phase II trial is studying giving treosulfan together with fludarabine to see how well it works in treating patients who are undergoing a donor stem cell transplant for acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome.
detailed Description: OBJECTIVES: Primary Phase * Determine the best dose of treosulfan when administered with fludarabine as a reduced-intensity conditioning regimen followed by allogeneic hematopoietic stem cell transplantation, in terms of incidence of severe to fatal toxicity to major organ systems and incidence of graft failure, in patients with acute myeloid leukemia, lymphoblastic leukemia, or myelodysplastic syndrome. Secondary Phase * Determine the safety of this regimen, in terms of incidence of grade II-IV acute and chronic graft-versus-host disease, in these patients. * Determine, preliminarily, the efficacy of this regimen, in terms of incidence of relapse, overall and disease-free survival, donor chimerism on days 28 and 100, and incidence of 200-day and 1-year nonrelapse mortality, in these patients. * Determine the pharmacokinetic and pharmacodynamic profile of treosulfan in patients treated with this regimen. OUTLINE: This is a multicenter, dose-finding study of treosulfan. * Reduced-intensity conditioning: Patients receive treosulfan IV over 2 hours on days -6 to -4 and fludarabine IV over 30 minutes on days -6 to -2. Cohorts of 5-10 patients receive escalating/de-escalating doses of treosulfan until the best dose is determined among the 3 pre-selected doses. The best dose is defined as the dose preceding that at which 4 of 10 patients experience dose-limiting toxicity. * Allogeneic hematopoietic cell transplantation (AHCT): Patients undergo allogeneic bone marrow or peripheral blood stem cell transplantation on day 0. All male patients with acute lymphoblastic leukemia OR male patients with acute myeloid leukemia who have prior or current testicular involvement receive external-beam radiotherapy to the testicles before AHCT. After completion of study treatment, patents are followed periodically. PROJECTED ACCRUAL: A total of 60 patients will be accrued for this study.

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice