ClinicalTrials.gg
CompletedNCT00534469Updated Dec 2, 2025Results posted

Auto BMT for Non-M3 AML in 1st Remission in Pts </=60y of Age Using Busulfan/FTBI & VP16 as a Prep R

A Phase 2 interventional study of aldesleukin and filgrastim in Leukemia, sponsored by City of Hope Medical Center. Completed. Open to participants aged 16 Years to 60 Years. Per ClinicalTrials.gov, last updated 2025-12-02.

Primary conditionLeukemia, Myeloid, AcuteRegistry: LeukemiaReclassifiedWhy we changed it
Registry
Leukemia — the first condition term the registry derived for this study.
ClinicalTrials.gg
Leukemia, Myeloid, Acute
Why
  1. The study's title name Leukemia, Myeloid, Acute, a narrower term within Leukemia.
Confidence
High: the new condition is named in the study's own title. Of 46 hand-checked studies reclassified this way, 72% were exactly right and 89% in the right disease family; the registry's first term was right for 4% (measured 2026-10-21). How we classify →
Wrong?
Tell us the study's main condition →

Sponsored by City of Hope Medical Center · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Registered 7 years 7 months after the study started (first participant enrolled Feb 2000, registered Sep 2007).
Phase
Phase 2
Study type
Interventional
Enrollment
60
Allocation
Not applicable
Ages
16 Years to 60 Years
Sex
All
01

Study summary

RATIONALE: Giving chemotherapy before an autologous stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and/or bone marrow and stored. More chemotherapy and radiation therapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and radiation therapy. Giving aldesleukin after transplant may help keep cancer cells from coming back after transplant.

PURPOSE: This phase II trial is studying the side effects and how well giving busulfan and etoposide together with total-body irradiation followed by autologous stem cell transplant and aldesleukin works in treating patients with acute myeloid leukemia in first remission.

Read the detailed description

OBJECTIVES:

  • To evaluate the efficacy and toxicity of a preparative regimen comprising busulfan, etoposide, and fractionated total-body irradiation followed by autologous stem cell transplantation and aldesleukin after treatment with consolidation therapy comprising high-dose cytarabine with or without idarubicin in patients with acute myeloid leukemia in first remission.
  • To estimate the long-term disease-free survival of patients treated with this regimen.
  • To further evaluate the effect of prognostic factors (e.g., cytogenetics, WBC at presentation, and number of courses of induction therapy required to achieve remission) on the outcome of autologous stem cell transplantation and targeted busulfan dose.

OUTLINE:

  • Consolidation therapy: Patients who received prior consolidation therapy are evaluated to determine the need for additional consolidation therapy. Patients who have not received prior consolidation therapy receive high-dose cytarabine IV over 3 hours every 12 hours on days 1-4 and idarubicin* IV over 5-10 minutes on days 1-3.

NOTE: *Patients with good risk cytogenetics t(8;21), inv(16), or t(16;16) or patients who received > 200 mg/m² of anthracycline do not receive idarubicin.

  • Stem cell collection: All patients receive filgrastim (G-CSF) IV or subcutaneously (SC) twice daily beginning 7 days after completion of high-dose cytarabine and continuing until peripheral blood stem cell (PBSC) collection is completed. Patients who do not have an adequate number of PBSCs collected also undergo bone marrow collection.
  • Preparative regimen: Patients receive busulfan IV over 2 hours on days -13 and -11 to -7 and etoposide IV on day -2. Patients also undergo fractionated total-body irradiation on days -6 to -3 for a total of 8-10 fractions.
  • Autologous stem cell transplantation: Patients undergo autologous stem cell transplantation using PBSCs (with or without bone marrow) on day 0. Patients receive G-CSF IV or SC daily beginning on day 5 and continuing until blood counts recover.
  • Interleukin therapy: Within 100 days post-transplantation, patients receive aldesleukin IV continuously on days 1-4 and 9-18.

After completion of study treatment, patients are followed periodically.

02

Conditions studied

  • Leukemia

Keywords

  • adult acute myeloid leukemia in remission
  • adult acute myeloid leukemia with 11q23 (MLL) abnormalities
  • adult acute myeloid leukemia with inv(16)(p13;q22)
  • adult acute myeloid leukemia with t(16;16)(p13;q22)
  • adult acute myeloid leukemia with t(8;21)(q22;q22)
  • adult acute basophilic leukemia
  • adult acute eosinophilic leukemia
  • adult erythroleukemia (M6a)
  • adult pure erythroid leukemia (M6b)
  • adult acute megakaryoblastic leukemia (M7)
  • adult acute minimally differentiated myeloid leukemia (M0)
  • adult acute monoblastic leukemia (M5a)
  • adult acute monocytic leukemia (M5b)
  • adult acute myeloblastic leukemia with maturation (M2)
  • adult acute myeloblastic leukemia without maturation (M1)
  • adult acute myelomonocytic leukemia (M4)
03

In context

Leukemia, Myeloid, Acute Reclassified

3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.

This study's enrollment of 60 is above the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.

Browse Leukemia, Myeloid, Acute studies →

Lead sponsor

City of Hope Medical Center is the lead sponsor of 677 studies on the registry; 181 are open to participants now.

Of its 31 completed or terminated interventional studies of FDA-regulated products, 12 (39%) have results posted.

Compared within Leukemia, Myeloid, Acute, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Leukemia. The counts are of registry records indexed under each term, refreshed daily. How we classify →

04

Who can participate

Ages eligible
16 Years to 60 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

DISEASE CHARACTERISTICS:

  • Diagnosis of acute myeloid leukemia (AML)

    • FAB types M0-2 and M4-M7

      • No M3 disease
  • In first complete hematological remission as confirmed by marrow aspiration and

    • No cytogenetic abnormality in the remission marrow
    • In complete remission for less than 6 months

      • Patients who have been in complete remission for more than 6 months may be eligible upon approval of the principal investigator
  • No prior myeloproliferative disorder (e.g., chronic myeloid leukemia, myelofibrosis, essential thrombocytosis, or polycythemia vera)
  • No prior myelodysplasia or secondary leukemia

PATIENT CHARACTERISTICS:

  • FEV_1 > 60%
  • DLCO > 50%
  • Cardiac ≥ 50%
  • > 60 mL/min
  • No severe chronic medical or psychological illness that, in the judgement of the principal investigator, would jeopardize the ability of the patient to tolerate aggressive chemotherapy
  • No positivity
  • Not pregnant
  • Negative pregnancy test

PRIOR CONCURRENT THERAPY:

  • Prior consolidation therapy allowed
  • No concurrent use the following medications during aldesleukin therapy :

    • (including blood product "pre-meds")
    • Pentoxifylline
    • IV or intrathecal methotrexate
    • IV immunoglobulin
    • Other cytokines or growth factors
Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Biopsy and archival tissue

A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.

Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
60 participants (actual)

Study arms

  • Experimental
    HD ARA-C with or without Idarubicin

    This study was designed as a single-arm study. Strata were: HD ARA-C w/ Idarubicin, CR\<6 months N = 24 patients. HD ARA-C w/ Idarubicin, CR\>6 months N=1. HD ARA-C consolidation, CR\<6 months N=5. HD ARA-C consolidation, CR\>6 months N= 0.

    Biological: aldesleukin · Biological: filgrastim · Drug: busulfan · Drug: cytarabine · Drug: etoposide · Drug: idarubicin · Procedure: autologous hematopoietic stem cell transplantation · Procedure: bone marrow transplantation · Procedure: peripheral blood stem cell transplantation · Radiation: total-body irradiation

Interventions

  • Biologicalaldesleukin

    Also known as: Proleukin

  • Biologicalfilgrastim

    Also known as: Neupogen

  • Also known as: Myleran, Busulfex IV

  • Also known as: cytosine arabinoside, ara-C, arabinofuranosyl cytidine, Cytosar-U

  • Also known as: VP-16, Vepesid, Toposar, Etophosphos

  • Also known as: Idamycin, Zavedos, 4-demethoxydaunorubicin

  • Procedureautologous hematopoietic stem cell transplantation
  • Procedurebone marrow transplantation
  • Procedureperipheral blood stem cell transplantation
  • Radiationtotal-body irradiation
06

What researchers measure

Primary outcomes

  1. Disease-Free Survival at 2-Year Post-Transplant

    Kaplan-Meier estimates at 2-years post-transplant, with 95% confidence intervals based on logit limit transformation of Greenwood's variance. Outcome is death or relapse, censor is alive in continual complete remission at the date of last clinical disease assessment.

    Time frame: Estimate at 2 years post treatment

Secondary outcomes

  1. Disease-Free Survival at 2-Year Post-Transplant by Cytogenetic Risk

    Kaplan-Meier estimates at 2-years post-transplant, with 95% confidence intervals based on logit limit transformation of Greenwood's variance. Outcome is death or relapse, censor is alive in continual complete remission at the date of last clinical disease assessment.

    Time frame: Kaplan-Meier estimate 2 years post treatment

07

Results

Posted Mar 21, 2023

Participant flow

60 patients went on-study. 52 went on to autologous transplant. Seven completed consolidation therapy without moving on to transplant. One was given an allogeneic transplant.

Participant flow — Overall Study
MilestoneHD ARA-C With Idarubicin, or Without Idarubicin if Previously Treated With an Anthracycline.
Started52
Completed52
Not completed0

Outcome measures

PrimaryDisease-Free Survival at 2-Year Post-Transplant

Kaplan-Meier estimates at 2-years post-transplant, with 95% confidence intervals based on logit limit transformation of Greenwood's variance. Outcome is death or relapse, censor is alive in continual complete remission at the date of last clinical disease assessment.

Time frame:
Estimate at 2 years post treatment
Reported as:
Number · Proportion of pts alive in remission
Disease-Free Survival at 2-Year Post-Transplant
Proportion of pts alive in remissionHD ARA-C Intermediate Cytogenetics
Disease-Free Survival at 2-Year Post-Transplant0.65 (0.52 to 0.77)
SecondaryDisease-Free Survival at 2-Year Post-Transplant by Cytogenetic Risk

Kaplan-Meier estimates at 2-years post-transplant, with 95% confidence intervals based on logit limit transformation of Greenwood's variance. Outcome is death or relapse, censor is alive in continual complete remission at the date of last clinical disease assessment.

Time frame:
Kaplan-Meier estimate 2 years post treatment
Reported as:
Number · Proportion of pts alive in remission
Disease-Free Survival at 2-Year Post-Transplant by Cytogenetic Risk
Proportion of pts alive in remissionUnfavorable Risk CytogeneticsIntermediate Risk CytogeneticsFavorable Risk CytogeneticsUnknown Risk: Rejected Cytogenetics Study
Disease-Free Survival at 2-Year Post-Transplant by Cytogenetic Risk0.33 (0.04 to 0.85)0.75 (0.52 to 0.89)0.75 (0.44 to 0.92)0.73 (0.41 to 0.91)
Statistical analysis
  • Unfavorable Risk Cytogenetics vs Intermediate Risk Cytogenetics vs Favorable Risk Cytogenetics vs Unknown Risk: Rejected Cytogenetics Study · Log Rank · p = 0.43 (Log-rank test (Mantel-Haenszel test). This p-value is not adjusted for multiple comparisons. The a priori threshold for statistical significance is 0.05.)

Adverse events

Collected over Adverse Events monitored/assessed up to 1 year post-transplant. All-Cause Mortality monitored/assessed up to 19 years post-transplant.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
HD ARA-C With or Without Idarubicin37/60 (61.7%)6/60 (10%)47/60 (78.3%)
Most frequent serious events
Showing 10 of 12
Most frequent serious events
EventHD ARA-C With or Without Idarubicin
Adult Respiratory Distress Syndrome (ARDS)Respiratory, thoracic and mediastinal disorders2/60
Cardiac General - Other: CARDIOGENIC SHOCKCardiac disorders1/60
Cardiac General - Other: CONGESTIVE HEART FAILURECardiac disorders1/60
Cardiac ischemia/infarctionCardiac disorders1/60
MyocarditisCardiac disorders1/60
Febrile neutropenia (fever of unknown origin)Infections and infestations1/60
Infection, Bacterial (COH)Infections and infestations1/60
Dyspnea (shortness of breath)Respiratory, thoracic and mediastinal disorders1/60
HypoxiaRespiratory, thoracic and mediastinal disorders1/60
Pulmonary/Upper Respiratory - Other: PULMONARY HYPERTENSIONRespiratory, thoracic and mediastinal disorders1/60
Most frequent other events
Showing 10 of 475
Most frequent other events
EventHD ARA-C With or Without Idarubicin
Leukocytes (total WBC) for BMT studies, if specified in the protocol.Blood and lymphatic system disorders46/60
Neutrophils/granulocytes (ANC/AGC) for BMT studies, if specified in the protocol.Blood and lymphatic system disorders45/60
Albumin, serum-low (hypoalbuminemia)Metabolism and nutrition disorders45/60
Calcium, serum-low (hypocalcemia)Metabolism and nutrition disorders44/60
Platelets for BMT studies, if specified in the protocol.Blood and lymphatic system disorders43/60
Transfusion: Platelets for BMT studies, if specified in the protocol.Blood and lymphatic system disorders43/60
Transfusion: pRBCs for BMT studies, if specified in the protocol.Blood and lymphatic system disorders43/60
NauseaGastrointestinal disorders43/60
VomitingGastrointestinal disorders42/60
Glucose, serum-high (hyperglycemia)Metabolism and nutrition disorders42/60

Baseline characteristics

Patients who went on to autologous transplant.

Age, Categorical
Age, Categorical(Participants)HD ARA-C With Idarubicin, or Without Idarubicin if Previously Treated With an Anthracycline.
<=18 years2
Between 18 and 65 years50
>=65 years0
Age, Continuous
Age, Continuous(years)HD ARA-C With Idarubicin, or Without Idarubicin if Previously Treated With an Anthracycline.
Median42.3 (16.5 to 59.7)
Sex: Female, Male
Sex: Female, Male(Participants)HD ARA-C With Idarubicin, or Without Idarubicin if Previously Treated With an Anthracycline.
Female31
Male21
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)HD ARA-C With Idarubicin, or Without Idarubicin if Previously Treated With an Anthracycline.
Race/Ethnicity — American Indian or Alaska Native, not of Hispanic Origin1
Race/Ethnicity — Asian or Pacific Islander, not of Hispanic Origin3
Race/Ethnicity — Black, not of Hispanic Origin2
Race/Ethnicity — Hispanic6
Race/Ethnicity — White, not of Hispanic Origin40
Region of Enrollment
Region of Enrollment(participants)HD ARA-C With Idarubicin, or Without Idarubicin if Previously Treated With an Anthracycline.
United States52
08

Study locations

No study locations are listed for this record.

09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 2, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00534469
Responsible party
Sponsor
First posted
Sep 24, 2007
Start date
Feb 8, 2000
Primary completion
Dec 31, 2022
Completion
Jul 16, 2025
Results posted
Mar 21, 2023
Last update
Dec 2, 2025

Study contacts

Anthony S. Stein, MD
study chair · City of Hope Medical Center
View the source record on ClinicalTrials.gov ↗

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Studies of the drugs tested here: Busulfan · Etoposide

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