Safety Study of MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia

NCT ID: NCT00677859

Last Updated: 2012-01-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-07-31
• Study Completion Date: 2011-11-30

Brief Summary

The purpose of this study is to determine if MultiStem® can safely be given to patients with acute leukemia, chronic myeloid leukemia, or myelodysplasia after they have received hematopoietic stem cell transplantation.

Detailed Description

Graft-vs.-Host Disease (GVHD) is one of the major limitations of allogeneic hematopoietic stem cell transplants (HSCT). This complication is major cause of morbidity and mortality and is thought to be initiated by activation of donor T-cells through recognition of "foreign" cells resident in the transplant recipient. Acute GVHD is associated with damage to the liver, skin, gastrointestinal tract and mucosa. Moderate to severe GVHD Grades II-IV occurs in 30-50% of matched related HSCTs and 50-70% of unrelated donor recipients. Severe GHVD requires intense immunosuppression involving steroids and additional agents to get it under control, and patients may develop severe infections as a result of such immunosuppression. An agent or cell therapy that could reduce the incidence and/or severity of GVHD without increasing relapse or infectious risk in HSCT patients would provide substantial benefits.

Conditions

Hematologic Malignancies

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

Single dose Arm

There will be six cohorts of three patients each. Three escalating doses of MultiStem will be evaluated.

Group Type: EXPERIMENTAL

MultiStem®

Intervention Type: BIOLOGICAL

Patients will receive a single IV infusion of MultiStem® 2 days after HSCT.

Repeat Dose Arm

There will be six cohorts of three patients each. Four dosing regimens will be evaluated,varying doses at three times weekly or five times weekly.

Group Type: EXPERIMENTAL

MultiStem®

Intervention Type: BIOLOGICAL

Patients will receive either 3 weekly IV infusions or 5 weekly infusions of MultiStem®

Interventions

MultiStem®

Patients will receive a single IV infusion of MultiStem® 2 days after HSCT.

Intervention Type: BIOLOGICAL

MultiStem®

Patients will receive either 3 weekly IV infusions or 5 weekly infusions of MultiStem®

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Patients of either sex aged 18-65 years of age
• Diagnosis of acute myeloid or lymphoblastic leukemia (second or subsequent remission, if not in remission, then <20% bone marrow blasts), chronic myelogenous leukemia resistant to or intolerant of tyrosine kinase inhibitor therapy (accelerated phase, first chronic phase with TKI resistance, or second chronic phase), or myelodysplastic syndrome (intermediate/high or high risk by International Prognostic Scoring System (IPSS), lower risk by IPSS with patient having progressed after prior therapy. Complete remission is defined as the absence of blasts in the peripheral circulation at the time of enrollment and <5% blasts in the marrow within 28 days of enrollment.
• Life expectancy of at least 100 days
• Patients scheduled for allogeneic bone marrow transplant or peripheral blood stem cell transplant (PBST) procedure
• Family-related or unrelated donors
• HLA matching should either be matched related or matched unrelated donors, 6/6 match or 5/6 single allelic mismatch, with provision that the DRB1 is molecularly matched
• Performance status (ECOG ≤2)
• Signed informed consent

Exclusion Criteria

• Active infection
• Known allergies to bovine or porcine products
• Renal function: Serum creatinine >2 mg/dL or creatinine clearance ≤50 mL/min
• Hepatic function: Screening ALT or AST ≥3x than the upper limit of normal for the laboratory OR total bilirubin ≥2.0 mg/dL (Exception: acceptable if patient is identified with pre-existing condition e.g., Gilbert's disease that will contribute to baseline elevations of bilirubin)
• Pulmonary function: FEV1, FVC, DLCO ≤50% predicted
• Cardiac function: left ventricular ejection fraction ≤50%
• Patient received an investigational agent within 30 days prior to transplant
• The patient is pregnant, has a positive serum BhCG, or is lactating
• Patient on corticosteroids at a dose >0.25 mg/kg/day
• Planned non-myeloablative transplant
• Planned cord blood transplant
• Prior allogeneic myeloablative HSCT
• HIV seropositive, HTLV seropositive, hepatitis B or C seropositive, varicella virus active infection, or syphilis active infection
• Other serious medical or psychiatric illness that, in the investigator's opinion, would not permit the patient to be managed according to the protocol

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cato Research

INDUSTRY

Sponsor Role: collaborator

Healios K.K.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Richard Maziarz, MD

Role: PRINCIPAL_INVESTIGATOR

Oregon Health and Science University

Steven Devine, MD

Role: PRINCIPAL_INVESTIGATOR

Ohio State University

Hillard Lazarus, MD

Role: PRINCIPAL_INVESTIGATOR

Case Western Reserve University

Locations

Mayo Clinic Hospital

Phoenix, Arizona, United States

University Hospitals Case Medical Center

Cleveland, Ohio, United States

Oregon State University Medical Center

Portland, Oregon, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Texas Transplant Institute

San Antonio, Texas, United States

UZ Leuven

Leuven, , Belgium

Countries

United States; Belgium

Other Identifiers

GVHD-2007-001

Identifier Type: -

Identifier Source: org_study_id