Clinical trial NCT00997386: Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States
Official title: A Phase II Study of Reduced-Intensity Allogeneic Peripheral Blood Stem Cell Transplantation (PBSCT) for Treatment of Hematologic Malignancies and Hematopoietic Failure States
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hematologic Neoplasms
Multiple Myeloma
Anemia, Aplastic
Hemoglobinuria, Paroxysmal
Myelofibrosis
Keywords: Hematologic malignancies
lymphoma, leukemia, MDS (myelodysplastic syndrome)
reduced-intensity preparative regimen
allogeneic peripheral blood stem cell transplant
myelofibrosis or other myeloproliferative syndromes
Lead sponsor: University of Arizona
Sponsor class: OTHER
First posted: 2009-10-19
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
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 to look at whether the combination of lower-dose chemotherapy with two chemotherapy (anti-cancer) drugs, called busulfan and melphalan, and an antibody medication called alemtuzumab (Campath®), can prevent rejection of donor blood stem cells so that those cells take hold and build a healthy new blood cell factory after transplant. The study will also look at the safety of the combination of drugs and of the transplant of peripheral blood stem cells from a healthy relative or an unrelated donor.

Detailed description: Transplantation of related or unrelated allogeneic peripheral blood stem cells (PBSCs) after administration of a reduced-intensity regimen of busulfan, melphalan and alemtuzumab will be associated with satisfactory engraftment and acceptable post-transplant non-relapse mortality.

Eligibility criteria: Inclusion Criteria:

* Age 50 to 75 years or age 18 to 49 with one or more of these risk factors: prior autologous, allogeneic or syngeneic HCT (Hematopoietic cell transplantation); not in first complete remission or first chronic phase; and/or presence of one or more medical conditions that would place the subject at high risk such as heart and kidney disease.
* Subjects with hematologic cancers must have received at least one previous course of chemotherapy or biological therapy. In other words, the subject cannot enroll in this trial for initial treatment of the disease.
* Availability of a healthy related or unrelated volunteer allogeneic donor.

Exclusion Criteria:

* Eligible for another study or standard of care treatment that offers higher probability of cure or long-term control of subject's disease.
* Severe abnormal function of organs such as heart, kidneys, liver.
* Untreated or progressive central nervous system involvement by the disease.
* Subject is pregnant or breast-feeding.
* Performance score is below 50: at the least, requires considerable assistance and frequent medical care.
* Positive for the HIV \[AIDS\] virus
* Life expectancy less than 12 weeks with conventional treatments.
* For subjects capable of having children, refusal to practice birth control while on this study and for at least 12 months after PBSCT or after stopping post-transplant immunosuppressive treatments, whichever occurs later.

Primary outcomes:
measure: Number of Participants With Presence of Donor Lymphohematopoietic Chimerism (Defined as at Least 50% Donor Cells in the Peripheral Blood) in Peripheral Blood by Day +100 (i.e., 100 Days After Allogeneic PBSCT).
timeFrame: Day +100
Secondary outcomes:
measure: Number of Participants With Relapse-free Survival.
timeFrame: Day +100
measure: Number of Participants With Event-free Survival.
timeFrame: Day +100
measure: Number of Participants With Overall Survival.
timeFrame: Day +100
Other outcomes:
Locations:
city: Tucson
country: United States
facility: University Medical Center and UMC-North Clinic
state: Arizona
Study officials:
affiliation: University of Arizona
name: Andrew M Yeager, MD
role: PRINCIPAL_INVESTIGATOR
References: