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Accelerated Phase Chronic Myelogenous LeukemiaPhase 2NCT01652014

Single or Double Donor Umbilical Cord Blood Transplant in Treating Patients With High-Risk Hematologic Malignancies

A Phase 2 study testing cyclophosphamide, fludarabine phosphate and mycophenolate mofetil in people with Accelerated Phase Chronic Myelogenous Leukemia and Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, run by University of Medicine and Dentistry of New Jersey at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This study will determine the safety and applicability of experimental forms of umbilical cord blood (UCB) transplantation for patients with high risk hematologic malignancies who might benefit from a hematopoietic stem cell transplant (HSCT) but who do not have a standard donor option (no available HLA-matched… Read more about this study

Who is it for?
Ages 18 to 65 years
What is being tested?
cyclophosphamide, fludarabine phosphate and mycophenolate mofetil
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
Jan 2014to Jan 2017
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 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients with histologically proven hematologic malignancy with anticipated 2 year survival < 20% with standard therapy; patients age <18 are excluded by virtue of the policies and procedures of the allogeneic hematopoietic stem cell transplant (HSCT) program (Cancer Institute of New Jersey [CINJ]/Robert Wood Johnson University Hospital [RWJUH] is not an approved Pediatric Transplant Center); patients > age 65 are generally not considered candidates for experimental unrelated allogeneic HSCT, as utilized in this study by virtue of the anticipated delayed immune reconstitution, high risk of GVHD, and known negative impact of age on outcomes
  • Patients eligible for this trial will have high risk diseases that include, but are not limited to:
  • Acute myeloid leukemia (AML) in second complete remission (CR2) or greater or early relapse with < 5% marrow blasts and no circulating blasts
  • AML in first complete remission (CR1) with high risk cytogenetics (complex, monosomy 5, monosomy 7, 11q23 (not t(9;11)), t(6;9), chromosome 3, monosomy phenotype and other karyotypes estimated to have =< 20% disease free survival at 3 years) or secondary/transformed AML without favorable cytogenetics;
  • Acute lymphoblastic leukemia (ALL) with t(9;22), 11q23 abnormality or early relapse (< 5% marrow blasts) or CR2 or greater;
  • Chronic myeloid leukemia (CML) resistant/refractory to all commercially available Abelson (abl) kinase inhibitors (e.g. imatinib mesylate, dasatinib, nilotinib) or predicted to be so based upon clinical course or abl kinase domain mutation analysis; or in accelerated phase or blast crisis;
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  • High intermediate to high international prognostic score myelodysplasia;
  • Non-Hodgkin lymphoma (NHL)/Hodgkin lymphoma (HL)/other lymphoproliferative diseases resistant/refractory to standard therapies and for whom an autologous transplant is considered to be inappropriate (e.g. bone marrow involvement, chemotherapy refractory disease, prior transplant);
  • Chronic lymphocytic leukemia (CLL) resistant/refractory to standard therapies (e.g. fludarabine) or high risk cytogenetics/fluorescence in situ hybridization (FISH) (e.g. 17p-);
  • Myeloproliferative disorders with progressive disease or cytopenias or clinical symptoms refractory to standard therapy (e.g. hypomethylating agents)
  • Relapsed or refractory multiple myeloma after (or not eligible for) high dose chemotherapy/autologous hematopoietic stem cell rescue and following salvage therapy with thalidomide, lenalidomide or bortezomib/other Food and Drug Administration (FDA)-approved multiple myeloma salvage therapies;
  • Other hematologic malignancies/disorders with anticipated 2 year survival < 20%, as established by available data bases, medical literature and the documented consensus of the Hematologic Malignancies Tumor Study Group
  • Patients must be an allogeneic HSCT candidate but have no standard donor (matched related donor [MRD], human leukocyte antigen [HLA]-matched unrelated donor [MUD] or single UCB unit of appropriate size and HLA type) available
  • Patients must have available UCB unit(s)
  • Patients considered for Arm 2 must not be eligible for Arm 1 and must have an HLA-haploidentical sibling, parent, child, or other relative (uncle, aunt, first cousin, niece or nephew) who meets donor requirements as outlined in Donor Eligibility criteria
  • Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status =< 2
  • Left ventricular (LV) ejection fraction >= 50%
  • Diffusion capacity of carbon monoxide (DLCO) corrected for hemoglobin > 60%
  • Total bilirubin within normal institutional limits unless the patient has Gilbert's disease
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase [SGPT]) =< 2.5 X institutional upper limit of normal (ULN)
  • Measured or estimated creatinine clearance > 50 ml/min
  • Hematopoietic stem cell co-morbidity index =< 2
  • There must be a negative pregnancy test for women of childbearing potential within 1 week of therapy; there must be willingness to avoid pregnancy and undergo counseling about contraceptive techniques throughout the course of treatment
  • There must be no uncontrolled infections or active acute or chronic illnesses such as diabetes, angina/myocardial ischemia, cardiac arrhythmia, venous thrombosis/embolism, cerebrovascular disease, seizure disorder, psychiatric illness or other intercurrent illness that is not well controlled or is anticipated to be difficult to control during the proposed therapy
  • The patient must be aware of the high risk and experimental nature of the treatment and provide informed consent
  • The patient must have clearance for HSCT after psychosocial evaluation
  • The patient must have adequate insurance or other support to meet the anticipated financial burden imposed by the costs of therapy
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Relative (parent, child, sibling, first cousin, uncle aunt, nephew, niece) with appropriate HLA match (>= 3/6 HLA A, B, DR match)
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Age >= 18 years old
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Normal hemogram; potential donors not having a normal hemogram may be utilized at the discretion of the Principal Investigator
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Not pregnant or lactating
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Not human immunodeficiency virus (HIV)-1, HIV-2, hepatitis C (HCV), Hepatitis B core or human T-lymphotropic virus (HTLV)-I/II seropositive; hepatitis B surface antigen (HB Sag)(-); must meet other infectious disease screening criteria utilized by New Brunswick Affiliated Hospital (NBAH) Blood Center
  • DONOR (for allogeneic lymphocytes, Arm 2 only): No uncontrolled infections, other medical or psychological/social conditions, or required medications that might increase the likelihood of patient or donor adverse effects or poor outcomes
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Meet other blood bank criteria for blood product donation (as determined by NBAH Blood Center screening history)
  • DONOR (for allogeneic lymphocytes, Arm 2 only): Donors must be informed of the investigational nature of this study, understand the requirements, potential benefits and potential risks of the experimental treatment, and give written informed consent in accordance with institutional and federal guidelines

Who cannot join

  • Prior extensive radiation therapy that the radiation oncologist feels precludes additional TBI
  • Patients with known human immunodeficiency virus (HIV) are excluded due to side effects of the therapy on the immune system
  • Patients with known active central nervous system (CNS) disease will be excluded from this clinical trial because they often develop progressive neurologic dysfunction unresponsive to HSCT therapy

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Cancer Institute of New Jersey

    New Brunswick, New Jersey, 08903

Other trials in United States

About this study

Official title: Hematopoietic Stem Cell Transplantation Using Alternate Donor Umbilical Cord Blood Options

This study will determine the safety and applicability of experimental forms of umbilical cord blood (UCB) transplantation for patients with high risk hematologic malignancies who might benefit from a hematopoietic stem cell transplant (HSCT) but who do not have a standard donor option (no available HLA-matched related donor (MRD), HLA-matched unrelated donor (MUD)), or single UCB unit with adequate cell number and HLA-match).

Keywords
Transplantation, Hematopoietic Stem Cell Transplantation
Medical subject headings
Leukemia, Myeloid, Accelerated Phase, Congenital Abnormalities, Lymphoma, Extranodal NK-T-Cell, Lymphoma, Large-Cell, Anaplastic, Immunoblastic Lymphadenopathy, Blast Crisis, Intraocular Lymphoma, Myeloproliferative Disorders

What participants receive

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

  • cyclophosphamideDrug

    Also called CPM, CTX, Cytoxan, Endoxan

    Given IV over 1 hour on Day -6; after pre-hydration

  • fludarabine phosphateDrug

    Also called 2-F-ara-AMP, Beneflur, Fludara

    Given IV daily over 30 minutes for 5 days (Days -6 to -2)

  • mycophenolate mofetilDrug

    Also called Cellcept, MMF

    Given PO 1.0 g BID Day 1-30

  • allogeneic hematopoietic stem cell transplantationProcedure or surgery

    Undergo double-unit allogeneic UCB transplant

  • umbilical cord blood transplantationProcedure or surgery

    Also called cord blood transplantation, transplantation, umbilical cord blood, UCB transplantation

    Undergo single allogeneic UCB transplant

  • double-unit umbilical cord blood transplantationProcedure or surgery

    Undergo double-unit allogeneic UCB transplant

  • total-body irradiationRadiation

    Also called TBI

    Undergo TBI

  • tacrolimusDrug

    Also called FK 506, Prograf

    Given IV 0.03 mg/kg/d as continuous infusion over 24 hours starting Day -3 with dose adjustments to maintain level of 8-20 mg/ml

  • allogeneic hematopoietic stem cell transplantationProcedure or surgery

    Undergo single allogeneic UCB transplant

  • peripheral blood stem cell transplantationProcedure or surgery

    Also called PBPC transplantation, PBSC transplantation, peripheral blood progenitor cell transplantation, transplantation, peripheral blood stem cell

    Undergo irradiated allogeneic peripheral blood stem cell transplant

Study groups in NCT01652014
Study groupRoleWhat they receive
Arm IExperimentalDouble UCB transplantation Patients receive conditioning comprising fludarabine phosphate IV over 30 minutes on days -6 to -2, cyclophosphamide IV over 1 hour on day -6, and undergo TBI on day -1. Patients also receive GVHD prophylaxis comprising tacrolimus IV continuously or PO beginning on day -3 with taper and mycophenolate mofetil PO BID days 1-30. Patients undergo double allogeneic UCB transplant on day 0.
Receives: Drug: cyclophosphamide; Drug: fludarabine phosphate; Drug: mycophenolate mofetil; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: double-unit umbilical cord blood transplantation; Radiation: total-body irradiation; Drug: tacrolimus
Arm IIExperimentalSub-threshold single UCB + irradiated PBMCs transplantation Patients receive conditioning comprising fludarabine phosphate IV over 30 minutes on days -6 to -2, cyclophosphamide IV over 1 hour on day -6, and undergo TBI on day -1. Patients also receive GVHD prophylaxis comprising tacrolimus IV continuously or PO beginning on day -3 with taper and mycophenolate mofetil PO BID days 1-30. Patients undergo single allogeneic UCB transplant on day 0. Patients also undergo irradiated allogeneic PBMC transplant within 8 hours following the UCB infusion.
Receives: Drug: cyclophosphamide; Drug: fludarabine phosphate; Drug: mycophenolate mofetil; Procedure: umbilical cord blood transplantation; Radiation: total-body irradiation; Drug: tacrolimus; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation

What is being measured

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

  1. Engraftment of white blood cells (WBC) (absolute neutrophil count > 500/mm^3)

    Measured: 3 years

  2. Non-relapse mortality

    Measured: 40 months

Secondary outcomes (7)

  1. Platelet engraftment rate (non-transfusion dependent)

    Measured: At 100 days

  2. Transplant related mortality

    Measured: At 1 year

  3. Rates of infection requiring hospitalization or prolongation of hospitalization

    Measured: Up to 2 years

  4. Incidence of steroid-refractory acute GVHD

    GVHD will be staged per standard guidelines of the American Society for Blood and Bone Marrow Transplantation.

    Measured: at 100 days

  5. Incidence of extensive chronic GVHD

    GVHD will be staged per standard guidelines of the American Society for Blood and Bone Marrow Transplantation.

    Measured: up to 2 years

  6. Total time on immunosuppressive therapy

    Measured: Up to 2 years

  7. Time to CD4 count > 200/mm^3

    Measured: Up to 2 years

Timeline

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

  1. First posted27 Jul 2012 (estimated)
  2. Study startJan 2014
  3. Primary completionJan 2017 (estimated)
  4. Study completionJan 2017 (estimated)

Why it stopped: Funding unavailable

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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01652014
Sponsor's protocol ID
021002
Other IDs
NCI-2011-03187, 0220100266, P30CA072720
Status last verified
Jul 2016
First submitted
20 Jul 2012
Last updated
14 Jul 2016
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing cyclophosphamide, fludarabine phosphate and mycophenolate mofetil in people with Accelerated Phase Chronic Myelogenous Leukemia and Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, run by University of Medicine and Dentistry of New Jersey 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?

The sponsor lists 35 inclusion and 3 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: withdrawn, as of the sponsor's last update on 14 Jul 2016. 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 withdrawn before enrolling participants, so there are no results.

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?

Engraftment of white blood cells (WBC) (absolute neutrophil count > 500/mm^3) (time frame: 3 years). The study lists 2 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 NCT01652014.

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 14 Jul 2016.

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