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A Phase I, Multicenter, Dose-Escalation Trial Evaluating Maximum-Tolerated Dose of Single and Repeated Administration of Allogeneic MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia

Phase 1
18 Years
65 Years
Not Enrolling
Hematologic Malignancies

Thank you

Trial Information

A Phase I, Multicenter, Dose-Escalation Trial Evaluating Maximum-Tolerated Dose of Single and Repeated Administration of Allogeneic MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia

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.

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

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

maximum tolerated dose

Outcome Time Frame:

30 days

Safety Issue:


Principal Investigator

Richard Maziarz, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Oregon Health and Science University


United States: Food and Drug Administration

Study ID:




Start Date:

July 2008

Completion Date:

November 2011

Related Keywords:

  • Hematologic Malignancies
  • Leukemia
  • Graft vs. Host Disease
  • Hematopoietic Stem Cell Transplant
  • Bone Marrow Transplant
  • Myelodysplasia
  • Neoplasms
  • Leukemia
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms



Mayo Clinic Hospital Phoenix, Arizona  85054-4502
University of Pennsylvania Philadelphia, Pennsylvania  19104
Texas Transplant Institute San Antonio, Texas  78229
University Hospitals Case Medical Center Cleveland, Ohio  44106
Oregon State University Medical Center Portland, Oregon  97239