Phase 2
N=60
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
Leukemia · Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT00253513 ↗Enrolled (actual)
60
Serious AEs
8.3%
Results posted
Feb 2011
Primary outcome: Primary: Number of Patients Experiencing Regimen-related Toxicity Events in Study Population — 2; 5 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- fludarabine (Drug); treosulfan (Drug); allogeneic blood or bone marrow transplantation (Procedure)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- OHSU Knight Cancer Institute
- Primary completion
- Oct 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Experiencing Regimen-related Toxicity Events in Study Population |
2; 5 | — |
| PRIMARY Number of Patients Experiencing Graft Failure |
36; 31 | — |
| PRIMARY Incidence (Percent of Participants) With Nonrelapse Mortality (NRM) by Day 200 (Secondary Phase Only) |
5 | — |
| SECONDARY Number of Subjects Who Are Without Disease at One Year as Indicator of Disease Free Survival. |
58 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT00253513). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.