Clinical trial NCT00943319: Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies
Official title: A Phase I-II Study of Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Leukemia
Lymphoma
Myeloma
Keywords: Cancers of the blood
Lead sponsor: University of Chicago
Sponsor class: OTHER
First posted: 2009-07-22
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is:

1. To establish the maximally tolerated dose (MTD) of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion.
2. To evaluate disease free and overall survival after this conditioning regimen in patients with advanced acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
3. To evaluate potential pharmacogenomic determinants of toxicity of this regimen.
4. To evaluate potential pharmacogenomic determinants of efficacy of this regimen.


Eligibility criteria: Inclusion Criteria:

Phase I portion:

* Relapsed or refractory acute myelogenous or lymphoid leukemia.
* Chronic myelogenous leukemia in accelerated phase or blast-crisis.
* Recurrent or refractory malignant lymphoma or Hodgkin's disease
* Recurrent or refractory multiple myeloma.
* Chronic lymphocytic leukemia, relapsed or with poor prognostic features.
* Myeloproliferative disorder (polycythemia vera, myelofibrosis) with transformation
* Myelodysplastic syndromes with more than 5% blasts.

Phase II portion:

* AML with active disease or beyond CR2.
* MDS with more than 5% blasts.

Exclusion Criteria:

* Clinical progression. Such patients may be treated on other treatment protocols or at the investigator's discretion. Such patients will continue to be monitored for survival and, may be asked to continue to provide specimens for studies of minimal residual disease and immune reconstitution as other treatments are recommended.

Primary outcomes:
measure: Maximum Tolerated Dose
timeFrame: 5 years
Secondary outcomes:
measure: Overall Survival
timeFrame: 5 years
measure: Disease Free Survival
timeFrame: 5 years
Other outcomes:
Locations:
city: Chicago
country: United States
facility: The University of Chicago
state: Illinois
Study officials:
affiliation: University of Chicago
name: Andrew Artz, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: O'Donnell PH, Artz AS, Undevia SD, Pai RK, Del Cerro P, Horowitz S, Godley LA, Hart J, Innocenti F, Larson RA, Odenike OM, Stock W, Van Besien K. Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease. Leuk Lymphoma. 2010 Dec;51(12):2240-9. doi: 10.3109/10428194.2010.520773. Epub 2010 Oct 4.