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Accelerated Phase of DiseasePhase 2NCT00003145

Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Peripheral Blood Stem Cell Transplant Followed by Donor Lymphocyte Infusion in Treating Older Patients With Chronic Myeloid Leukemia

A Phase 2 study testing Fludarabine Phosphate, Total-Body Irradiation and Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation in people with Accelerated Phase of Disease and Childhood Chronic Myelogenous Leukemia, BCR-ABL1 Positive, run by Fred Hutchinson Cancer Center at 8 sites in 3 countries.

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

This clinical trial studies fludarabine phosphate, low-dose total-body irradiation, and peripheral blood stem cell transplant followed by donor lymphocyte infusion in treating older patients with chronic myeloid leukemia. Read more about this study

Who is it for?
Ages Up to 74 years
What is being tested?
Fludarabine Phosphate, Total-Body Irradiation and Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
8 sitesin 3 countries
Study dates
Aug 1997to Mar 2005
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 74 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients with Philadelphia chromosome positive (Ph+) CML in first and second chronic and first accelerated phases
  • Patients =< 65 years old who are at high risk of regimen related toxicity through pre-existing chronic disease affecting liver, lungs, and/or heart, or others who wish to be treated on this protocol, will be considered on a case-by-case basis; transplants should be approved for these inclusion criteria by both the participating institutions' patient review committees such as the Patient Care Conference (PCC) at the Fred Hutchinson Cancer Research Center (FHCRC) and by the principal investigators at the collaborating centers; patients =< 65 years of age who have received previous high-dose transplantation do not require patient review committee approvals; all children < 12 years must be discussed with the FHCRC principal investigator (PI) (Brenda Sandmaier, MD 206-667-4961) prior to registration
  • HLA genotypically identical related donor willing to undergo leukapheresis initially for collection of peripheral blood stem cell (PBSC) and subsequently for collection of peripheral blood mononuclear cell (PBMC)
  • Patients treated with alpha interferon must have discontinued drug at least 1 month prior to transplant
  • DONOR: HLA genotypically identical family member (excluding identical twins)
  • DONOR: Donor must consent to filgrastim (G-CSF) administration and leukapheresis
  • DONOR: Donor must have adequate veins for leukapheresis or agree to placement of central venous catheter (femoral, subclavian)

Who cannot join

  • Patients who are human immunodeficiency virus positive (HIV+)
  • GROUP 1: (PATIENTS AGED > 65 AND < 75 YEARS)
  • Patients unwilling to use contraceptive techniques during and for 12 months following treatment
  • Presence of circulating leukemic blasts (in the peripheral blood) detected by standard pathology for patients with CML
  • Patients in an interferon induced complete or partial cytogenetic remission
  • Organ dysfunction:
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  • Patients with renal failure are eligible, however patients with renal compromise (Serum creatinine greater than 2.0) will likely have further compromise in renal function and may require hemodialysis (which may be permanent) due to the need to maintain adequate serum cyclosporine levels
  • Cardiac ejection fraction < 40%; ejection fraction is required if the patient has a history of anthracyclines or history of cardiac disease
  • Diffusing capacity of the lung for carbon monoxide (DLCO) < 50% of predicted
  • Liver function tests including total bilirubin, serum glutamic pyruvate transaminase (SGPT) and serum glutamic oxaloacetic transaminase (SGOT) > 2x the upper limit of normal unless proven to be due to the malignancy
  • Karnofsky score < 70
  • Patients with poorly controlled hypertension
  • GROUP 2 (PATIENTS AGED =< 65)
  • Patients who are HIV+
  • Presence of circulating leukemic blasts (in the peripheral blood) detected by standard pathology for patients with CML
  • Females who are pregnant
  • Patients unwilling to use contraceptive techniques during and for 12 months following treatment
  • Patients in an interferon induced complete or partial cytogenetic remission
  • Organ dysfunction:
  • Patients with renal failure are eligible, however patients with renal compromise (Serum creatinine greater than 2.0) will likely have further compromise in renal function and may require hemodialysis (which may be permanent) due to the need to maintain adequate serum cyclosporine levels
  • Cardiac ejection fraction < 40% or a history of congestive heart failure; ejection fraction is required if the patient has a history of anthracyclines or history of cardiac disease
  • Severe defects in pulmonary function testing as defined by the pulmonary consultant (defects are currently categorized as mild, moderate and severe) or receiving supplementary continuous oxygen; DLCO < 50% of predicted
  • Liver function tests: total bilirubin > 2x the upper limit of normal, SGPT and SGOT 4x the upper limit of normal
  • Karnofsky score < 50
  • Patients with poorly controlled hypertension
  • DONOR: Age less than 12 years
  • DONOR: Pregnancy
  • DONOR: Infection with HIV
  • DONOR: Inability to achieve adequate venous access
  • DONOR: Known allergy to G-CSF
  • DONOR: Current serious systemic illness

Trial locations and contacts

8 sites in 3 countries.

8 sites in 3 countries

United States6 sites
  • Baylor University Medical Center

    Dallas, Texas, 75246

  • University of Colorado

    Denver, Colorado, 80217-3364

  • City of Hope Medical Center

    Duarte, California, 91010

  • VA Puget Sound Health Care System

    Seattle, Washington, 98101

  • Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

    Seattle, Washington, 98109

  • Stanford University Hospitals and Clinics

    Stanford, California, 94305

Other trials in United States

Germany1 site
  • Universitaet Leipzig

    Leipzig, D-04103

Other trials in Germany

Italy1 site
  • University of Torino

    Torino, 10126

Other trials in Italy

About this study

Official title: Induction of Mixed Hematopoietic Chimerism Using Fludarabine, Low Dose TBI , PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to be Followed by Donor Lymphocyte Infusion In Patients With Chronic Myeloid Leukemia in Chronic and Accelerated Phases: A Multi-center Study

This clinical trial studies fludarabine phosphate, low-dose total-body irradiation, and peripheral blood stem cell transplant followed by donor lymphocyte infusion in treating older patients with chronic myeloid leukemia. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect). Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine if mixed hematopoietic chimerism can be safely established using a non-myeloablative conditioning regimen in patients > 65 years of age with chronic myeloid leukemia (CML) in chronic or accelerated phase who have human leukocyte antigen (HLA) identical related donors.

II. To determine if mixed chimerism, established with non-myeloablative conditioning regimens, can be converted to full donor hematopoietic chimerism by infusions of donor lymphocytes (DLI), and thereby produce an immunologic cure of the malignancy.

OUTLINE:

CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously (IV) on days -4 to -2 and undergo low-dose total-body irradiation (TBI) on day 0.

TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplantation (PBSCT) on day 0.

IMMUNOSUPPRESSION: Patients receive cyclosporine orally (PO) twice daily (BID) or IV BID or thrice daily (TID) on days -3 to 56 with taper to day 77 or 180, and mycophenolate mofetil PO or IV BID on days 0-27.

DLI: At least 2 weeks after completion of immunosuppression, patients with > 5% donor cluster of differentiation (CD)3+ T cells and no evidence of graft-versus-host disease (GVHD) receive donor lymphocytes IV over 30 minutes. Patients may receive up to 3 DLIs at increasing cell doses in the absence of GVHD.

After completion of study treatment, patients are followed up periodically for 5 years.

Being tested
Fludarabine Phosphate, Total-Body Irradiation, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Peripheral Blood Stem Cell Transplantation, Cyclosporine
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Recurrence

What participants receive

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

  • Fludarabine PhosphateDrug

    Also called 2-F-ara-AMP, Beneflur, SH T 586

    Given IV

  • Total-Body IrradiationRadiation

    Also called TBI, Total Body Irradiation, Whole-Body Irradiation

    Undergo TBI

  • Nonmyeloablative Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Non-myeloablative allogeneic transplant, Nonmyeloablative Stem Cell Transplantation, NST

    Undergo allogeneic PBSCT

  • Peripheral Blood Stem Cell TransplantationProcedure or surgery

    Also called PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation

    Undergo allogeneic PBSCT

  • CyclosporineDrug

    Also called 27-400, CsA, Neoral, OL 27-400

    Given PO or IV

  • Mycophenolate MofetilDrug

    Also called Cellcept, MMF

    Given PO or IV

  • Therapeutic Allogeneic LymphocytesBiological

    Also called Allogeneic Lymphocytes, ALLOLYMPH, Tumor-Derived Lymphocyte

    Given IV

  • Laboratory Biomarker AnalysisOther

    Correlative studies

Study groups in NCT00003145
Study groupRoleWhat they receive
Treatment (chemotherapy, TBI, PBSCT, DLI)ExperimentalCONDITIONING REGIMEN: Patients receive fludarabine phosphate IV on days -4 to -2 and undergo low-dose TBI on day 0. TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0. IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID or IV BID or TID on days -3 to 56 with taper to day 77 or 180, and mycophenolate mofetil PO or IV BID on days 0-27. DLI: At least 2 weeks after completion of immunosuppression, patients with > 5% donor CD3+ T cells and no evidence of GVHD receive donor lymphocytes IV over 30 minutes. Patients may receive up to 3 DLIs at increasing cell doses in the absence of GVHD.
Receives: Drug: Fludarabine Phosphate; Radiation: Total-Body Irradiation; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation; Drug: Cyclosporine; Drug: Mycophenolate Mofetil; Biological: Therapeutic Allogeneic Lymphocytes; Other: Laboratory Biomarker Analysis

What is being measured

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

  1. Safety of establishing mixed chimerism using this non-lethal conditioning regimen, in terms of development of GVHD, myelosuppression, infections, and treatment-related mortality

    All unexpected toxicities will be summarized and reported.

    Measured: Within the first 65 days

  2. Proportion of patients who successfully establish mixed chimerism (evidence of donor engraftment) and the proportion who convert to full donor chimeras among those who were mixed

    Estimated and corresponding confidence intervals presented. Mixed chimerism is detection of donor T cells (CD3+) and granulocytes (CD33+), as proportion of total T cell and granulocyte population of > 5% and < 95% in peripheral blood. Full donor chimerism is > 95% donor CD3+ T cells. Increasing donor chimerism is absolute increase of 20% of CD3+ T cells over chimerism evaluation of previous month. Decreasing donor chimerism is absolute decrease of 20% of CD3+ T cell chimerism over previous month. Low donor chimerism is < 40% CD3+ T cells after HSCT.

    Measured: Day 28

  3. Proportion of patients who successfully establish mixed chimerism (evidence of donor engraftment) and the proportion who convert to full donor chimeras among those who were mixed

    Estimated and corresponding confidence intervals presented. Mixed chimerism is detection of donor T cells (CD3+) and granulocytes (CD33+), as proportion of total T cell and granulocyte population of > 5% and < 95% in peripheral blood. Full donor chimerism is > 95% donor CD3+ T cells. Increasing donor chimerism is absolute increase of 20% of CD3+ T cells over chimerism evaluation of previous month. Decreasing donor chimerism is absolute decrease of 20% of CD3+ T cell chimerism over previous month. Low donor chimerism is < 40% CD3+ T cells after HSCT.

    Measured: Day 56

Secondary outcomes (9)
  1. Proportion of patients experiencing a complete antileukemic response

    Reported in a descriptive manner and confidence intervals will be presented for all estimates.

    Measured: At 12 weeks after the final DLI

  2. Proportion of patients experiencing GVHD

    Reported in a descriptive manner and confidence intervals will be presented for all estimates.

    Measured: Until day 90 after the last DLI

  3. Proportion of patients experiencing non-relapse mortality

    Reported in a descriptive manner and confidence intervals will be presented for all estimates.

    Measured: Within 65 days of transplant

  4. Incidence of myelosuppression after initial PBSC infusion

    Defined as absolute neutrophil count [ANC] < 500 for > 2 days, platelets < 20,000 for > 2 days.

    Measured: Until 2 months post-transplant

  5. Incidence of aplasia after DLI

    Measured: Until 2 months post-transplant

  6. Incidence of grades 2-4 acute GVHD after DLI

    Measured: Until day 90 after the last DLI

  7. Incidence of grades 2-4 acute GVHD after PBSC infusion

    Measured: Until day 90 after the last DLI

  8. Incidence of grade chronic extensive GVHD after DLI

    Measured: At 1 year

  9. Dose of CD3+ cells required to convert mixed to full lymphoid chimeras

    Measured: Day 56

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 startAug 1997
  3. Primary completionMar 2005

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
18 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00003145
Sponsor's protocol ID
1209.00
Other IDs
NCI-2012-00579, 1209.00, P30CA015704
Status last verified
Jan 2020
First submitted
1 Nov 1999
Last updated
10 Jan 2020
Results posted
No
Data monitoring committee
Yes

Publications

  • Cooper JP, Storer BE, Granot N, Gyurkocza B, Sorror ML, Chauncey TR, Shizuru J, Franke GN, Maris MB, Boyer M, Bruno B, Sahebi F, Langston AA, Hari P, Agura ED, Lykke Petersen S, Maziarz RT, Bethge W, Asch J, Gutman JA, Olesen G, Yeager AM, Hubel K, Hogan WJ, Maloney DG, Mielcarek M, Martin PJ, Flowers MED, Georges GE, Woolfrey AE, Deeg JH, Scott BL, McDonald GB, Storb R, Sandmaier BM. Allogeneic hematopoietic cell transplantation with non-myeloablative conditioning for patients with hematologic malignancies: Improved outcomes over two decades. Haematologica. 2021 Jun 1;106(6):1599-1607. doi: 10.3324/haematol.2020.248187.

    PubMed 32499241

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Fludarabine Phosphate, Total-Body Irradiation and Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation in people with Accelerated Phase of Disease and Childhood Chronic Myelogenous Leukemia, BCR-ABL1 Positive, run by Fred Hutchinson Cancer Center at 8 sites in 3 countries.

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

Who may be eligible to take part?

The sponsor lists 7 inclusion and 31 exclusion criteria. 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 10 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States, Germany, Italy. 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?

Safety of establishing mixed chimerism using this non-lethal conditioning regimen, in terms of development of GVHD, myelosuppression, infections, and treatment-related mortality (time frame: Within the first 65 days). The study lists 3 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 NCT00003145, which also lists 1 linked publication.

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 10 Jan 2020.

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