AzTrials
AzTrials

Chronic Myeloproliferative DisordersPhase 2NCT00006379

Non-Ablative Allo HSCT For Hematologic Malignancies or SAA

A Phase 2 study testing anti-thymocyte globulin, graft-versus-tumor induction therapy and cyclophosphamide in people with Chronic Myeloproliferative Disorders and Leukemia, run by Case Comprehensive Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by chemotherapy used to kill cancer cells. Read more about this study

Who is it for?
Ages Up to 70 years
What is being tested?
anti-thymocyte globulin, graft-versus-tumor induction therapy and cyclophosphamide
How many people
58 participants
Where
1 siteUnited States
Study dates
Jun 2000to Oct 2011
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

DISEASE CHARACTERISTICS:

  • Histologically proven high-risk hematologic malignancy
  • Acute non-lymphocytic leukemia (ANLL) after induction failure, or in first complete remission (CR) with high-risk features, including any of the following:
  • Stem cell or biphenotypic classification (AML-M0)
  • Erythroleukemia (AML-M6)
  • Acute megakaryocytic leukemia (AML-M7)
  • Cytogenetic markers indicative of poor prognosis
  • Failure to achieve CR after standard induction therapy
  • Acute lymphocytic leukemia (ALL) or ANLL in relapse or second or subsequent remission
  • Chronic myelogenous leukemia (CML) in chronic or accelerated phase
  • Patients with CML in blast crisis are eligible after reinduction chemotherapy places them in chronic phase
  • High-risk ALL in first CR with high risk defined by presence of t(4;11), t(9;22) translocation, hyperleukocytosis (initial WBC greater than 30,000/mm^3), or failure to achieve CR by day 28 after standard induction
  • No T-cell ALL or t(8;14) positive B-cell ALL in first remission with hyperleukocytosis
  • Myelodysplastic syndrome by peripheral blood smear and bone marrow examination
  • Refractory to medical management OR
  • Cytogenetic abnormalities predictive of transformation into acute leukemia including 5q-, 7q-, monosomy 7, and trisomy 8 OR
  • Evidence of evolution to AML (e.g., refractory anemia with excess blasts (RAEB) or RAEB in transformation)
  • Multiple myeloma, non-Hodgkin's lymphoma (NHL), ANLL, or ALL with recurrent disease after autologous stem cell transplantation (SCT)
  • At least 3 months since prior autologous SCT
  • Hodgkin's lymphoma, NHL, or multiple myeloma beyond first CR or primary induction failures whose disease has demonstrated sensitivity to pre-transplantation cytoreduction (defined as greater than 50% reduction in tumor burden)
  • Mantle zone NHL allowed after induction therapy
  • Myeloproliferative disorder that is non-responsive to medical management and requires allografting, unless evidence of grade 3 or worse myelofibrosis on marrow biopsy OR
  • Histologically proven acquired severe aplastic anemia (SAA) that is recurrent or unresponsive after anti-thymocyte globulin and/or cyclosporine
  • SAA defined by at least 2 of the following conditions:
  • Granulocyte count less than 500/mm^3
  • Platelet count less than 20,000/mm^3
  • Absolute reticulocyte count less than 20,000/mm^3 after correction for hematocrit
  • Ineligible for full ablative conditioning due to any of the following conditions:
  • Prior extensive therapy (more than 2 salvage chemotherapy regimens and/or autologous transplantation)
  • Over age 55 OR
  • Under age 55 with comorbid disease (e.g., suboptimal cardiac, pulmonary, or renal function and/or prior life-threatening infection)
  • HLA-A, B, and DR phenotypically identical sibling donor OR
  • HLA-A, B, and DR identical genetically matched unrelated donor
  • No ANLL in first CR (less than 5% blasts in marrow) with translocations t(8;21) and inv(16) unless failed first-line induction therapy OR
  • No ANLL in first CR (less than 5% blasts in marrow) with translocations t(15;17) abnormality unless failed first-line induction therapy OR molecular evidence of persistent disease
  • No active CNS disease

PATIENT CHARACTERISTICS:

Age:

  • 0 to 70

Performance status:

  • Zubrod 0-1
  • Karnofsky 80-100%

Life expectancy:

  • At least 3 months

Hematopoietic:

  • See Disease Characteristics

Hepatic:

  • ALT/AST no greater than 4 times normal
  • Bilirubin no greater than 2.0 mg/dL

Renal:

  • See Disease Characteristics
  • Creatinine clearance at least 50 mL/min

Cardiovascular:

  • See Disease Characteristics
  • Shortening fraction or ejection fraction at least 40% of normal for age by echocardiogram or radionuclide scan
  • No clinically significant comorbid illnesses (e.g., myocardial infarction or cerebrovascular accident)

Pulmonary:

  • See Disease Characteristics
  • FVC and FEV_1 at least 60% of predicted for age
  • DLCO at least 60% of predicted for adults

Other:

  • No severe neurosensory symptoms (i.e., peripheral neuropathy)
  • HIV negative
  • Active infection allowed if controlled by appropriate drug therapy
  • Not pregnant or nursing
  • Negative pregnancy test

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • See Disease Characteristics

Chemotherapy:

  • See Disease Characteristics

Endocrine therapy:

  • Not specified

Radiotherapy:

  • Not specified

Surgery:

  • Not specified

Other:

  • Recovered from prior therapy
  • No concurrent investigational agents unless approved by protocol investigators

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center

    Cleveland, Ohio, 44106-5065

Other trials in United States

About this study

Official title: Purine-Analog-Containing Non-Myeloablative Allogeneic Stem Cell Transplantation for Treatment of Hematologic Malignancies and Severe Aplastic Anemia

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by chemotherapy used to kill cancer cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy followed by peripheral stem cell transplantation in treating patients who have hematologic cancer or aplastic anemia.

Read the detailed description

OBJECTIVES:

  • Determine the rates of durable full donor hematologic engraftment in patients with high-risk hematologic malignancies or severe aplastic anemia treated with non-myeloablative conditioning using fludarabine, cyclophosphamide, and anti-thymocyte globulin followed by allogeneic peripheral blood stem cell transplantation.
  • Determine the acute and delayed toxic effects of this non-myeloablative conditioning regimen in this patient population.
  • Determine the event-free and overall survival of patients treated with this regimen.
  • Determine the incidence and severity of acute and chronic graft-versus-host disease in patients treated with this regimen.
  • Determine the rate and quality of immune reconstitution in patients treated with this regimen.
  • Determine the rate of disease relapse and incidence of post-transplantation lymphoproliferative disease in these patients.

OUTLINE: Patients are stratified according to disease category (malignant vs non-malignant) and graft source (unrelated vs HLA-matched sibling).

Beginning at least 4 weeks after conventional-dose chemotherapy, patients receive non-myeloablative conditioning comprising fludarabine IV over 30 minutes on days -8 to -4, cyclophosphamide IV over 2 hours on days -3 to -2, and anti-thymocyte globulin IV over at least 4 hours on days -2 and -1. Patients undergo filgrastim (G-CSF)-mobilized allogeneic peripheral blood stem cell transplantation on day 0.

Patients are followed weekly for 3 months, every 2 weeks for 3 months, monthly for 6 months, and then every 2 months thereafter.

PROJECTED ACCRUAL: A minimum of 30 patients will be accrued for this study within 4 years.

Being tested
anti-thymocyte globulin, graft-versus-tumor induction therapy, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation
Keywords
monoclonal gammopathy of undetermined significance, recurrent childhood acute lymphoblastic leukemia, recurrent adult Hodgkin lymphoma, recurrent cutaneous T-cell non-Hodgkin lymphoma, isolated plasmacytoma of bone, extramedullary plasmacytoma, refractory multiple myeloma, Waldenstrom macroglobulinemia, recurrent childhood lymphoblastic lymphoma, recurrent childhood acute myeloid leukemia, recurrent adult acute myeloid leukemia, recurrent adult acute lymphoblastic leukemia, relapsing chronic myelogenous leukemia, small intestine lymphoma, chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, adult acute myeloid leukemia in remission, adult acute lymphoblastic leukemia in remission, childhood acute myeloid leukemia in remission
Medical subject headings
Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Precancerous Conditions

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • anti-thymocyte globulinBiological

    anti-thymocyte globulin IV over at least 4 hours on days -2 and -1

  • graft-versus-tumor induction therapyBiological

    Patients undergo filgrastim (G-CSF)-mobilized allogeneic peripheral blood stem cell transplantation on day 0.

  • cyclophosphamideDrug

    cyclophosphamide IV over 2 hours on days -3 to -2

  • fludarabine phosphateDrug

    fludarabine IV over 30 minutes on days -8 to -4

  • peripheral blood stem cell transplantationProcedure or surgery

    Patients undergo filgrastim (G-CSF)-mobilized allogeneic peripheral blood stem cell transplantation on day 0.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Evaluation of Donor Engraftment

    Peripheral blood from the donor and patient is obtained for chimerism studies. The primary analysis will consist of estimating the graft failure proportions for each of the separate patient groups and calculating confidence intervals for these proportions. This analysis will be done conditional on patients surviving at least 28 days.

    Measured: at 28 days

Secondary outcomes (2)
  1. Stable donor hematopoietic chimerism

    Number of Patients Transplanted More Than 100 Days Ago

    Measured: at day 100

  2. Event free and overall survival

    Measured: to progression/death

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jan 2003 (estimated)
  2. Study startJun 2000
  3. Primary completionJan 2004
  4. Study completionOct 2011

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
58 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00006379
Sponsor's protocol ID
CWRU3Y00
Other IDs
P30CA043703, CWRU-3Y00, 05-00-07, NCI-G00-1868
Status last verified
Dec 2011
First submitted
4 Oct 2000
Last updated
8 Dec 2011
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing anti-thymocyte globulin, graft-versus-tumor induction therapy and cyclophosphamide in people with Chronic Myeloproliferative Disorders and Leukemia, run by Case Comprehensive Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Dec 2011. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Evaluation of Donor Engraftment (time frame: at 28 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2011.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in