Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States (ALBUM)

• ClinicalTrials.gov Identifier: NCT00997386
• Recruitment Status: Completed
• First Posted: October 19, 2009
• Results First Posted: December 24, 2018
• Last Update Posted: September 9, 2019
• Sponsor: University of Arizona
• Information provided by (Responsible Party): University of Arizona

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Hematologic Neoplasms; Multiple Myeloma; Anemia, Aplastic; Hemoglobinuria, Paroxysmal; Myelofibrosis	Drug: busulfan, and melphalan, and alemtuzumab	Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 16 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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 Start Date: September 2009
• Actual Primary Completion Date: April 2014
• Actual Study Completion Date: January 2016

Arms and Interventions

Arm	Intervention/treatment
Experimental: busulfan, and melphalan, and alemtuzumab; Three drug regimen using busulfan, and melphalan, and alemtuzumab.	Drug: busulfan, and melphalan, and alemtuzumab; intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT). intravenous melphalan 100 mg/m2 on day -3. intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.; Other Names: Busulfan (Busulfex®),; Melphalan (Alkeran®); Alemtuzumab (Campath®)

Outcome Measures

Primary Outcome Measures :

1. 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). [ Time Frame: Day +100 ]
   To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the 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).

Secondary Outcome Measures :

1. Number of Participants With Relapse-free Survival. [ Time Frame: Day +100 ]
   To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.
2. Number of Participants With Event-free Survival. [ Time Frame: Day +100 ]
   To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.
3. Number of Participants With Overall Survival. [ Time Frame: Day +100 ]
   To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 75 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

United States, Arizona

University Medical Center and UMC-North Clinic

Tucson, Arizona, United States, 85719

Sponsors and Collaborators

University of Arizona

Investigators

• Principal Investigator: Andrew M Yeager, MD; University of Arizona

More Information

• Responsible Party: University of Arizona
• ClinicalTrials.gov Identifier: NCT00997386
• Other Study ID Numbers: 09-0679-04
• First Posted: October 19, 2009
• Results First Posted: December 24, 2018
• Last Update Posted: September 9, 2019
• Last Verified: September 2019

Keywords provided by University of Arizona:

Hematologic malignancies; lymphoma, leukemia, MDS (myelodysplastic syndrome); reduced-intensity preparative regimen; allogeneic peripheral blood stem cell transplant; myelofibrosis or other myeloproliferative syndromes

Additional relevant MeSH terms:

Multiple Myeloma; Neoplasms; Hematologic Neoplasms; Hemoglobinuria; Primary Myelofibrosis; Anemia, Aplastic; Hemoglobinuria, Paroxysmal; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Myeloproliferative Disorders; Bone Marrow Diseases; Neoplasms by Site; Proteinuria; Urination Disorders; Urologic Diseases; Urological Manifestations; Anemia; Bone Marrow Failure Disorders; Anemia, Hemolytic; Myelodysplastic Syndromes