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

NCT00997386 | Completed Phase 2 Results DMC

• 1 other identifier: 09-0679-04
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (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).

  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).

  Day +100

Secondary Outcomes (3)

• Number of Participants With Relapse-free Survival.

  Day +100

• Number of Participants With Event-free Survival.

  Day +100

• Number of Participants With Overall Survival.

  Day +100

Study Arms (1)

busulfan, and melphalan, and alemtuzumab

EXPERIMENTAL

Three drug regimen using busulfan, and melphalan, and alemtuzumab.

Drug: busulfan, and melphalan, and alemtuzumab

Interventions

busulfan, and melphalan, and alemtuzumab DRUG

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.

Also known as: Busulfan (Busulfex®),, Melphalan (Alkeran®), Alemtuzumab (Campath®)

busulfan, and melphalan, and alemtuzumab

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Arizona: lead

Study Sites (1)

University Medical Center and UMC-North Clinic

Tucson, Arizona, 85719, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms; Multiple Myeloma; Anemia, Aplastic; Hemoglobinuria, Paroxysmal; Primary Myelofibrosis; Lymphoma; Leukemia; Myelodysplastic Syndromes

Interventions

Busulfan; Melphalan; Alemtuzumab

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Anemia; Bone Marrow Failure Disorders; Bone Marrow Diseases; Anemia, Hemolytic; Myeloproliferative Disorders; Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Program Coordinator
• Organization: University of Arizona Cancer Center

aselegue@email.arizona.edu

52-626-0301

Study Officials

• Andrew M Yeager, MD

  University of Arizona

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2009
• First Posted: October 19, 2009
• Study Start: September 1, 2009
• Primary Completion: April 1, 2014
• Study Completion: January 1, 2016
• Last Updated: September 9, 2019
• Results First Posted: December 24, 2018

Record last verified: 2019-09

Locations

US (1)