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Acute Myeloid Leukemia (AML)NCT00363467

Busulfan Monotherapy as Conditioning for Autologous Hematopoietic Progenitor Cell Transplantation

A study testing G-CSF, Leukapheresis and Busulfan in people with Acute Myeloid Leukemia (AML), run by H. Lee Moffitt Cancer Center and Research Institute 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

During the pre-transplantation phase (following completion of consolidation chemotherapy), patients will begin to receive G-CSF at 10 mcg/kg twice daily; leukapheresis will also be given until a target goal for recipient body weight is obtained, or up to a maximum of 5 days. Read more about this study

Who is it for?
Ages 56 to 74 years
Conditions the study is for
Acute Myeloid Leukemia (AML)
What is being tested?
G-CSF, Leukapheresis and Busulfan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteUnited States
Study dates
May 2006to May 2009
Phase
Interventional
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 56 to 74 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have had histologically confirmed diagnosis of AML, in 1st complete remission, by a pathologic review at the H. Lee Moffitt Cancer Center and Research Institute. Any induction/consolidation regimen is permitted.
  • General Inclusion Criteria:
  • Age 56-74
  • Able to give informed consent
  • Hepatic and renal function: normal bilirubin, AST and ALT less than or equal to 2x normal limits, serum creatinine less than or equal to 1.5x normal
  • Left ventricular ejection fraction (LVEF) must be in normal range
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  • FEV1 AND DLCO greater than or equal to 50% predicted (at planned time of transplantation)
  • ECOG PS less than or equal to 2 (at planned time of transplantation)
  • Disease Specific Inclusion Criteria:
  • Adverse-risk karyotype (del 5/5q, 7/7q, 3q, greater than or equal to 3 abnormalities):
  • Intermediate-risk karyotype [46 XY, +8, -Y, +6, or any other isolated (<3 total) non-random abnormality not included in the adverse-risk category or favorable-risk category below]
  • AML arising from antecedent hematologic disorder (e.g. MDS)
  • Secondary AML (t-AML)

Who cannot join

  • Acute Promyelocytic Leukemia(FAB M3) subtype
  • Presence of (8;21) translocation or inversion 16/t(16;16) cytogenetic phenotype (i.e. favorable-risk AML)
  • Eligible for and willing to undergo matched-sibling allogeneic transplantation
  • Greater than 2 induction regimens required to achieve complete remission
  • Duration of > 8 weeks between completion of induction chemotherapy and initiation of consolidation chemotherapy
  • No prior malignancy is allowed, except for adequately treated basal cell (or squamous cell) skin cancer, in situ cervical cancer, or other cancer for which the patient has been disease-free for at least 5 years.
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  • Prior extensive radiation therapy (>25% of bone marrow reserve)
  • Concomitant radiation therapy, chemotherapy, or immunotherapy
  • Intrinsic impaired organ function (as stated above)
  • Active infection
  • Positive serum pregnancy test in women who have not yet reached menopause (no menstrual periods for >12 months or who have not undergone tubal ligation or complete hysterectomy.
  • Women who are breast-feeding
  • Positive HIV testing
  • Presence of CNS leukemia
  • Uncontrolled insulin-dependent diabetes mellitus or uncompensated major thyroid or adrenal gland dysfunction
  • Physical or psychiatric conditions that in the estimation of the PI or his designee place the patient at high-risk of toxicity or non-compliance

Trial locations and contacts

1 site in 1 country.

United States1 site

  • H. Lee Moffitt Cancer Center & Research Institute

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Pilot Study of Intravenous, Targeted-Dose Busulfan Monotherapy as Conditioning for Autologous Hematopoietic Progenitor Cell Transplantation in High-Risk AML

During the pre-transplantation phase (following completion of consolidation chemotherapy), patients will begin to receive G-CSF at 10 mcg/kg twice daily; leukapheresis will also be given until a target goal for recipient body weight is obtained, or up to a maximum of 5 days. Conditioning/Preparative therapy will follow PBSC collection for up to 30 days with Busulfan IV daily x 4 days; subsequent doses will be adjusted based on pharmacokinetic (plasma level)monitoring. Following 1 day of rest, stem cell reinfusion will begin with supportive care. During follow-up, patients will be monitored out to 730 days.

Read the detailed description
  1. Pre- Transplantation Phase -
  2. Twenty-four to 48 hours following completion of consolidation chemotherapy, patients will begin to receive G-CSF at 10 mcg/kg twice daily subcutaneously. Alternatively, patients may receive G-CSF alone (same dose) as mobilization therapy.
  3. Leukapheresis will begin day 4 of G-CSF administration and proceed according to institutional guidelines. Leukapheresis will continue until a target goal for recipient body weight is obtained, or up to a maximum of 5 days. A minimum recipient body weight is required to proceed to transplantation.
  4. Transplantation Phase
  5. Conditioning/Preparative therapy - up to 30 days following PBSC collection, patients will begin conditioning therapy with Busulfan IV daily x 4 days (transplantation days -5,-4,-3,-2). The day -5 and -4 dose will be 130mg/m2; subsequent doses will be adjusted based on pharmacokinetic monitoring.
  • Busulfan plasma level monitoring, collected around the first dose of busulfan b. Stem cell reinfusion - following 1 day of rest, previously collected autologous peripheral blood stem cells will be infused.
  • The administration of supportive measures (e.g. intravenous fluids, antihistamines) during stem cell reinfusion will be performed according to institutional guidelines.
  1. Supportive care
  2. Antibiotic prophylaxis- according to hospital/institutional guidelines, and at the discretion of the treating physician.
  3. Growth factor support
  4. Transfusion support
  5. Prophylaxis for busulfan-induced seizures
  6. During follow-up, patients will be seen at least weekly for the first month and there after periodically out to 730 days posttransplant. The following medical procedures will be done:
  • Medical history and physical exam (including concurrent meds, vital signs, performance status and weight)
  • Standard labs
  • Bone marrow aspirate and biopsy
Being tested
G-CSF, Leukapheresis, Busulfan, Stem cell reinfusion
Keywords
Busulfan, Acute myeloid leukemia (AML), Hematopoietic Stem Cell Transplantation (HSCT), Dendritic cells, Bone marrow transplantation, PBSC mobilization, Autologous stem cells
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

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

  • G-CSFDrug

    Also called filgrastim or NeupogenR

    Mobilization Option 1:Twenty-four to 48 hours following completion of consolidation chemotherapy, patients will begin to receive G-CSF at 10 mcg/kg twice daily subcutaneously.

    Mobilization Option 2: If patients have recovered hematologically from consolidation chemotherapy, they may receive G-CSF at 10 mcg/kg twice daily subcutaneously.

  • LeukapheresisDrug

    leukapheresis

  • BusulfanDrug

    Also called Busulfan (MyleranR, Busulfex™)

    Busulfan IV daily x 4 days (transplantation days -5,-4,-3,-2). The day -5 and -4 dose will be 130mg/m2

  • Stem cell reinfusionProcedure or surgery

    autologous stem cell transplant

Study groups in NCT00363467
Study groupRoleWhat they receive
Autologous Hematopoietic Progenitor Cell TransplantationOtherG-CSF Mobilization Leukepheresis Busulfan Stem Cell Reinfusion
Receives: Drug: G-CSF; Drug: Leukapheresis; Drug: Busulfan; Procedure: Stem cell reinfusion

What is being measured

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

  1. 100-day Non-relapse Mortality

    100-day non-relapse mortality is the number of participants who died before day 100 posttransplant from causes other than relapsed disease

    Measured: 100 days post transplant

Secondary outcomes (4)

  1. Successful Autologous Stem Cell Collection

    Number of subjects who were able to collect at least 2 million CD34+ cells/kg

    Measured: At time of stem cell collection

  2. Severe Regimen-related Toxicity

    Number of participants with severe regimen-related toxicity within 2 years posttransplant. Severe regimen-related toxicity was defined as CTC (version 3)grade 4.

    Measured: up to 100 days post translant

  3. 1 Year Event-free Survival

    Number of participants alive and without disease relapse at 1 year posttransplant

    Measured: 1 year post transplant

  4. 1 Year Overall Survival

    Number of participants alive at 1 year posttransplant

    Measured: 1 year 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 posted15 Aug 2006 (estimated)
  2. Study startMay 2006
  3. Primary completionMay 2009
  4. Study completionMay 2009

Why it stopped: Low accrual

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 N/A
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00363467
Sponsor's protocol ID
MCC-14604
Status last verified
Aug 2011
First submitted
10 Aug 2006
Last updated
23 Mar 2017
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A study testing G-CSF, Leukapheresis and Busulfan in people with Acute Myeloid Leukemia (AML), run by H. Lee Moffitt Cancer Center and Research Institute 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 13 inclusion and 16 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: stopped early, as of the sponsor's last update on 23 Mar 2017. 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Sep 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

100-day Non-relapse Mortality (time frame: 100 days post transplant). 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 NCT00363467.

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 23 Mar 2017.

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