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Chronic Myeloproliferative DisordersPhase 2NCT00066417

Peripheral Stem Cell Transplant in Treating Patients With High-Risk Leukemia

A Phase 2 study testing cyclophosphamide, cyclosporine and fludarabine phosphate in people with Chronic Myeloproliferative Disorders and Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. Read more about this study

Who is it for?
Ages 10 to 50 years
What is being tested?
cyclophosphamide, cyclosporine and fludarabine phosphate
Type of treatment
Surgery or procedure
How many people
51 participants
Where
1 siteUnited States
Study dates
Start not givento Jan 2007
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 10 to 50 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Diagnosis of 1 of the following:
  • High-risk myelodysplastic syndromes (MDS), meeting 1 of the following criteria:
  • Transformation to acute leukemia defined by at least 15% blasts
  • Secondary to prior treatment with chemotherapy and/or radiotherapy
  • Presence of complex cytogenetics (at least 3 karyotypic abnormalities)
  • Monosomy or deletion of chromosome 7
  • Acute myeloid leukemia (AML), meeting 1 of the following criteria :
  • High-risk AML in first remission and meeting 1 of the following criteria:
  • At least 3 karyotypic abnormalities
  • Monosomy or deletion of chromosome 5 or 7 = 11q23 chromosomal abnormality
  • Prior diagnosis of MDS
  • Received prior radiotherapy or chemotherapy
  • In second or subsequent remission
  • Primary induction failure or partial remission
  • Untested or sensitive relapse
  • Chronic myelogenous leukemia, meeting 1 of the following criteria:
  • Blast crisis
  • Accelerated phase disease that has failed prior treatment with imatinib mesylate, defined as a failure to achieve hematologic response after 3 months of standard dose (600 mg/day) therapy or disease progression on therapy
  • Myeloproliferative disease
  • The following diagnoses are eligible:
  • Agnogenic myeloid metaplasia
  • Essential thrombocythemia
  • Polycythemia vera
  • Must have evidence of transformation to acute leukemia
  • Acute lymphocytic leukemia (ALL), meeting 1 of the following criteria:
  • High-risk ALL in first remission defined by 1 of the following:
  • t(9;22) or 11q23 chromosomal abnormality
  • Complete response at least 4 weeks after induction therapy OR requiring at least 2 induction regimens
  • Second or subsequent remission
  • No relapsed leukemia refractory to appropriate salvage therapy
  • Availability of an HLA-mismatched family donor
  • Donor age 75 or under
  • No better donor alternative (i.e., HLA-matched related or unrelated stem cell donor) is available

PATIENT CHARACTERISTICS:

Age

  • 10 to 50

Performance status

  • ECOG 0-1

Life expectancy

  • More than 3 months

Hematopoietic

  • See Disease Characteristics

Hepatic

  • Bilirubin no greater than 4 mg/dL
  • Transaminases no greater than 3 times upper limit of normal

Renal

  • Creatinine no greater than 2.0 mg/dL OR
  • Creatinine clearance at least 60 mL/min

Cardiovascular

  • LVEF at least 40%

Pulmonary

  • DLCO at least 65% of predicted

Other

  • Not pregnant
  • Negative pregnancy test
  • HIV negative
  • No other prior malignancy except basal cell or squamous cell skin cancer or a remote history of cancer now considered cured
  • No major organ dysfunction that would preclude transplantation
  • No major anticipated illness or organ failure that would preclude transplantation
  • No severe psychiatric illness or mental deficiency that would preclude giving informed consent or complying with study
  • No uncontrolled infection

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • See Disease Characteristics

Endocrine therapy

  • Not specified

Radiotherapy

  • See Disease Characteristics

Surgery

  • Not specified

Trial locations and contacts

1 site in 1 country.

United States1 site

  • NIH - Warren Grant Magnuson Clinical Center

    Bethesda, Maryland, 20892-1182

Other trials in United States

About this study

Official title: Pilot Study Of T-Cell-Depleted Peripheral Blood Stem Cell Transplantation From Partially Matched Related Donors For Patients With High-Risk Leukemia

RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the stem cells from a related donor, that do not exactly match the patient's blood, are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.

PURPOSE: This phase II trial is studying how well peripheral stem cell transplant works in treating patients with high-risk leukemia.

Read the detailed description

OBJECTIVES:

  • Determine the safety of a preparative regimen comprising total body irradiation, cyclophosphamide, thiotepa, and fludarabine, but without anti-thymocyte globulin, in patients with high-risk leukemia treated with peripheral blood stem cell transplantation from partially matched related donors.
  • Determine the incidence of graft failure, acute graft-versus-host disease (GVHD), and treatment-related mortality in patients treated with this regimen.
  • Determine rates of chronic GVHD and relapse in patients treated with this regimen.
  • Determine disease-free and overall survival in patients treated with this regimen.

OUTLINE: This is a pilot study.

Patients receive a preparative regimen comprising total lymphoid irradiation once daily on days -13 to -11; cyclophosphamide IV over 1 hour on days -8 and -7; thiotepa IV over 4 hours every 12 hours on day -6; fludarabine IV over 30 minutes on days -5 to -1; and total body irradiation once on day -1. Patients also receive cyclosporine IV over 12 hours on days -8 to -1 and methylprednisolone IV twice daily on days -3 and -2. Patients receive CD34-enriched T-cell-depleted allogeneic stem cell infusion on day 0.

Patients with disease progression or uncontrolled infection but without grade II or greater graft-versus-host disease may receive up to 3 donor lymphocyte infusions at least 4 weeks apart until disease regression.

Patients are followed at least weekly until day 100 and then at 6, 12, 18, 24, 36, and 48 months.

PROJECTED ACCRUAL: A total of 20-51 patients will be accrued for this study.

Being tested
cyclophosphamide, cyclosporine, fludarabine phosphate, methylprednisolone, therapeutic allogeneic lymphocytes
Keywords
essential thrombocythemia, polycythemia vera, blastic phase chronic myelogenous leukemia, secondary myelodysplastic syndromes, previously treated myelodysplastic syndromes, adult acute myeloid leukemia in remission, childhood acute myeloid leukemia in remission, de novo myelodysplastic syndromes, secondary acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, adult acute lymphoblastic leukemia in remission, childhood acute lymphoblastic leukemia in remission, recurrent adult acute myeloid leukemia, relapsing chronic myelogenous leukemia, recurrent childhood acute myeloid leukemia, chronic idiopathic myelofibrosis, atypical chronic myeloid leukemia, myelodysplastic/myeloproliferative disease, unclassifiable, adult acute myeloid leukemia with t(8;21)(q22;q22)
Medical subject headings
Myeloproliferative Disorders, Leukemia, Myelodysplastic-Myeloproliferative Diseases, Thrombocythemia, Essential, Polycythemia Vera, Blast Crisis, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Acute

What participants receive

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

  • cyclophosphamideDrug

  • cyclosporineDrug

  • fludarabine phosphateDrug

  • methylprednisoloneDrug

  • therapeutic allogeneic lymphocytesDrug

  • thiotepaDrug

  • allogeneic bone marrow transplantationProcedure or surgery

  • biological therapyProcedure or surgery

  • bone marrow ablation with stem cell supportProcedure or surgery

  • bone marrow transplantationProcedure or surgery

  • chemotherapyProcedure or surgery

  • leukocyte therapyProcedure or surgery

  • non-specific immune-modulator therapyProcedure or surgery

  • peripheral blood lymphocyte therapyProcedure or surgery

  • peripheral blood stem cell transplantationProcedure or surgery

  • radiation therapyProcedure or surgery

What is being measured

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

  1. Incidence of graft failure 100 days post-transplant

  2. Incidence of acute and chronic graft-vs-host disease100 days post-transplant

  3. Transplant-related mortality 100 days post-transplant

  4. Disease-free survival 100 days post-transplant

  5. Overall survival 100 days post-transplant

Timeline

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

  1. First posted7 Aug 2003 (estimated)
  2. Study completionJan 2007

Why it stopped: Trial was withdrawn for drug availability issues.

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
Masking
None (open label)
Enrolment
51 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00066417
Sponsor's protocol ID
CDR0000315900
Other IDs
NHLBI-03-H-0209
Status last verified
Nov 2006
First submitted
6 Aug 2003
Last updated
1 May 2013
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing cyclophosphamide, cyclosporine and fludarabine phosphate in people with Chronic Myeloproliferative Disorders and Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) 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: stopped early, as of the sponsor's last update on 1 May 2013. 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 was stopped early (reason given: Trial was withdrawn for drug availability issues.). Results are not available in the source record.

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?

Incidence of graft failure 100 days post-transplant. The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00066417.

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 1 May 2013.

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