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Pilot Study of Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies


Phase 1
18 Years
55 Years
Not Enrolling
Both
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes

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Trial Information

Pilot Study of Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies


OBJECTIVES:

Primary

- Determine the safety and feasibility of performing donor umbilical cord blood
transplantation (UCBT) in patients with advanced hematologic malignancies, in terms of
> 80% engraftment rate at day 100 post-transplant and ≤ 50% transplant-related
mortality.

Secondary

- Determine the toxicity of a myeloablative preparative regimen comprising busulfan,
fludarabine, and etoposide prior to UCBT in these patients.

- Determine the neutrophil and platelet recovery in patients treated with this regimen.

- Determine the event-free and overall survival of patients treated with this regimen.

- Evaluate lineage-specific chimerism after UCBT and assess the contribution of each
individual cord blood unit to post-transplantation hematopoiesis in these patients.

- Determine the incidence, severity, and timing of acute and chronic graft-vs-host
disease in patients treated with this regimen.

OUTLINE: This is a pilot study.

- Preparative regimen: Patients receive fludarabine IV over 30 minutes on days -7 to -3,
busulfan IV over 2 hours 4 times daily on days -7 and -4, etoposide IV over 4 hours on
day -3, and anti-thymocyte globulin IV over 6 hours on days -2 and -1.

- Donor umbilical cord blood transplantation (UCBT): Patients undergo donor UCBT on day
0. Beginning on day 7, patients receive sargramostim (GM-CSF) IV or subcutaneously once
daily until blood counts recover.

- Graft-vs-host disease prophylaxis: Patients receive tacrolimus IV continuously over 24
hours or orally twice daily beginning on day -2 and continuing until day 180 followed
by a taper. Patients also receive oral prednisone twice daily on days 13-50 and then
once daily on days 50-60, followed by a rapid taper.

After completion of study treatment, patients are followed periodically for approximately 2
years.

PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of 1 of the following advanced hematologic malignancies:

- Acute myeloid leukemia (AML) meeting the following criteria:

- Not expected to be curable with chemotherapy and meets ≥ 1 of the following
criteria:

- High-risk cytogenetics (-7, -7q, -5, -5q, t[6,9], t[9,11], complex,
Philadelphia chromosome positive [Ph+])

- AML evolved from prior myelodysplasia

- AML secondary to prior chemotherapy

- Failed to achieve remission

- In second or subsequent remission

- Marrow blasts ≤ 10% (may be achieved using chemotherapy)

- Myelodysplastic syndromes (MDS) with high-risk features

- International Prognostic Scoring System (IPSS) score intermediate -2 or
high-risk

- Marrow blasts ≤ 20% (may be achieved using chemotherapy)

- Acute lymphoblastic leukemia meeting the following criteria:

- Not expected to be curable with chemotherapy and meets ≥ 1 of the following
criteria:

- High-risk cytogenetics (Ph+, t[4,11], 11q23 abnormalities, and
monosomy 7)

- Required > 1 induction course to achieve remission

- Failed to achieve remission

- In second or subsequent remission

- Marrow blasts ≤ 10% (may be achieved using chemotherapy)

- Chronic myelogenous leukemia meeting ≥ 1 of the following criteria:

- Accelerated phase

- Chronic phase refractory to imatinib mesylate

- Blastic phase

- Marrow blasts ≤ 10% (may be achieved using chemotherapy)

- Multiple myeloma meeting 1 of the following criteria:

- Stage II or III disease with > first relapse or refractory disease

- Newly diagnosed disease with chromosome 13 abnormalities

- Lymphoma meeting the following criteria:

- One of the following subtypes:

- Diffuse large cell lymphoma

- Mantle cell lymphoma

- Peripheral T-cell lymphoma

- T-natural killer (NK) cell lymphoma

- Hodgkin's lymphoma

- Disease failed to respond to primary therapy, progressed, or recurred after
prior therapy

- Patients who have failed autologous stem cell transplantation are
eligible provided it has been > 1 year since transplant

- No rapid progression of malignant disease

- Not eligible for autologous stem cell transplantation

- Available umbilical cord blood (1-3 units) donor matching at ≥ 4 of 6 HLA antigens
(A, B, and DR)

- Patients with an HLA-identical or 1 antigen-mismatched related donor OR a
potential HLA-matched unrelated donor matching at > 6/8 (A, B, C, DR) alleles
are not eligible

PATIENT CHARACTERISTICS:

- ECOG performance status 0-2

- Creatinine < 2.0 mg/dL

- Creatinine clearance > 40 mL/min

- Bilirubin < 2.0 mg/dL

- AST and alkaline phosphatase < 3 times upper limit of normal

- Hepatitis C and active hepatitis B allowed if patient has ≤ grade 2 inflammation or
fibrosis by liver biopsy

- Ejection fraction > 40% by echocardiogram or MUGA

- DLCO > 40% of predicted

- Not pregnant or nursing

- Negative pregnancy test

- No known HIV infection

- No active infection requiring ongoing antibiotic treatment

PRIOR CONCURRENT THERAPY:

- See Disease Characteristics

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies

Outcome Description:

Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies, in terms of > 80% engraftment rate at day 100 post-transplant and ≤ 50% transplant-related mortality.

Outcome Time Frame:

up to 24 months post-transplant

Safety Issue:

Yes

Principal Investigator

Thomas G. Martin, MD

Investigator Role:

Study Chair

Investigator Affiliation:

University of California, San Francisco

Authority:

United States: Federal Government

Study ID:

CDR0000463370

NCT ID:

NCT00304018

Start Date:

October 2002

Completion Date:

March 2009

Related Keywords:

  • Leukemia
  • Lymphoma
  • Multiple Myeloma and Plasma Cell Neoplasm
  • Myelodysplastic Syndromes
  • adult acute myeloid leukemia with 11q23 (MLL) abnormalities
  • recurrent adult acute myeloid leukemia
  • secondary acute myeloid leukemia
  • adult acute myeloid leukemia in remission
  • adult acute erythroid leukemia (M6)
  • adult acute megakaryoblastic leukemia (M7)
  • adult acute monoblastic leukemia (M5a)
  • adult acute monocytic leukemia (M5b)
  • refractory multiple myeloma
  • adult acute lymphoblastic leukemia in remission
  • accelerated phase chronic myelogenous leukemia
  • blastic phase chronic myelogenous leukemia
  • relapsing chronic myelogenous leukemia
  • chronic phase chronic myelogenous leukemia
  • stage II multiple myeloma
  • stage III multiple myeloma
  • recurrent adult acute lymphoblastic leukemia
  • recurrent adult diffuse large cell lymphoma
  • stage III adult diffuse large cell lymphoma
  • recurrent mantle cell lymphoma
  • stage III mantle cell lymphoma
  • recurrent adult Hodgkin lymphoma
  • stage III adult Hodgkin lymphoma
  • adult acute myeloid leukemia with inv(16)(p13;q22)
  • adult acute myeloid leukemia with t(15;17)(q22;q12)
  • adult acute myeloid leukemia with t(16;16)(p13;q22)
  • adult acute myeloid leukemia with t(8;21)(q22;q22)
  • de novo myelodysplastic syndromes
  • previously treated myelodysplastic syndromes
  • secondary myelodysplastic syndromes
  • recurrent adult T-cell leukemia/lymphoma
  • stage IV adult diffuse large cell lymphoma
  • stage IV adult Hodgkin lymphoma
  • stage IV adult T-cell leukemia/lymphoma
  • stage IV mantle cell lymphoma
  • stage III adult T-cell leukemia/lymphoma
  • stage I multiple myeloma
  • Neoplasms
  • Leukemia
  • Lymphoma
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Plasmacytoma
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms

Name

Location

UCSF Helen Diller Family Comprehensive Cancer Center San Francisco, California  94115