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Allogeneic Stem Cell Transplant With a Novel Conditioning Therapy Using Helical Tomotherapy, Melphalan, and Fludarabine in Hematological Malignancies

7 Years
Open (Enrolling)
Leukemia, Myelodysplastic Syndromes

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Trial Information

Allogeneic Stem Cell Transplant With a Novel Conditioning Therapy Using Helical Tomotherapy, Melphalan, and Fludarabine in Hematological Malignancies

PRIMARY OBJECTIVES: I. To assess the feasibility in terms of toxicity and safety of HT in
combination with Fludarabine (fludarabine phosphate) and Melphalan as a preparative regimen
for allogeneic stem cell transplantation in patients with Hematological Malignancies: acute
lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), and myelodysplastic syndromes

SECONDARY OBJECTIVES: I. To evaluate within the confines of this pilot study, incidence of
neutrophil and platelet engraftment, survival on day +180, the overall survival, and disease
free survival in patients with Hematological Malignancies: ALL, AML, and MDS.

II. To evaluate incidence of primary and secondary engraftment failure, incidence of
relapse, incidence of acute and chronic transplant related complications, including
veno-occlusive disease of the liver (VOD), organ toxicity, secondary malignancies, including
treatment-related myelodysplastic syndrome, and acute and chronic graft-versus-host disease
(GVHD), as well as post-transplant chimerism.

OUTLINE: PREPARATIVE REGIMEN*: Patients receive fludarabine phosphate intravenously (IV) on
days -7 to -3 and melphalan IV on day -2. Patients also undergo HT twice daily on days -7 to

TRANSPLANTATION: Patients undergo allogeneic hematopoietic stem cell transplantation on day
0. NOTE: *Treatment begins 2 days earlier in patients receive tacrolimus and/or sirolimus
for GVHD prophylaxis.

After completion of study treatment, patients are followed up periodically for 2 years.

Inclusion Criteria:

- Recipient, or recipient's parents, or recipient's legal guardians must have signed a
voluntary, informed consent in accordance with institutional and federal guidelines

- Must have histopathologically confirmed diagnosis in one of the followed categories:


- MDS with intermediate or high-risk disease


- Children and adults at any age with significant morbidity, as determined by the
primary bone marrow transplant (BMT) doctor (MD), and approved by the principal
investigator (PI)

- Able to lie supine in a full body cast for approximately 30 minutes, the anticipated
duration of each treatment session; for younger patients deep conscious sedation may
be required

- Performance status evaluated by Zubrod or Karnofsky (KPS) Performance Scales in
patients > 16 years or Lanksy Performance Scale in children =< 16 years must have a
score >= 70%

- Adequate cardiac function: cardiac ejection fraction > 50% by multi gated acquisition
scan (MUGA) scan and/or by echocardiogram

- Adequate pulmonary function: adults (older than 16 years): diffusing capacity of
carbon monoxide (DLCO) > 50%; for young children in whom pulmonary function tests
(PFT) are not applicable: assessment by a pediatrician or pulmonary consult

- Adequate renal function as demonstrated by: creatinine clearance or glomerular
filtration rate (GFR) > 60 cc/min (24 hour urine collection)

- Serum glutamic oxaloacetic transaminase (SGOT) and serum glutamic pyruvic
transaminase (SGPT) =< 5.0 times the institutional upper limits of normal

- Patients must have less than 15% peripheral blasts

- Pre-treatment tests must have been preformed within 30 days prior to initiation of
high-dose chemotherapy

- No other medical and/or psychosocial problems, which in the opinion of the primary
physician or principle investigator would place the patient at unacceptable risk from
this regimen

Exclusion Criteria:

- Patients with Acute Undifferentiated Leukemia (AUL), i.e. no lymphoid or myeloid

- Previous radiation therapy to more than 20% of bone marrow containing areas, or to
any area exceeding 2000 cGy

- Patients with Fanconi Anemia

- Major medical or psychiatric disorders that would seriously compromise patient
tolerance of this regimen

- Human immunodeficiency virus (HIV) infection

- Evidence of Hepatitis B or C infection or evidence of cirrhosis

- Uncontrolled viral, bacterial or fungal infection

- Patients with recent (within 4 weeks) serious viral, fungal, or bacterial infection
are excluded

- Patients with radiographic changes indicating pulmonary disease, including but not
limited to: pulmonary nodules, infiltrates, pleural effusion are excluded unless
cleared by pulmonary biopsy showing no evidence for active pulmonary disease

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Toxicity of helical tomotherapy (HT) in combination with fludarabine and melphalan followed by allogeneic stem cell transplantation

Outcome Time Frame:

100 days post treatment

Safety Issue:


Principal Investigator

Joseph Rosenthal, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

City of Hope Medical Center


United States: Federal Government

Study ID:




Start Date:

November 2005

Completion Date:

Related Keywords:

  • Leukemia
  • Myelodysplastic Syndromes
  • acute myeloid leukemia with multilineage dysplasia following myelodysplastic syndrome
  • adult acute lymphoblastic leukemia in remission
  • adult acute myeloid leukemia in remission
  • adult acute myeloid leukemia with 11q23 (MLL) abnormalities
  • adult acute myeloid leukemia with del(5q)
  • adult acute myeloid leukemia with inv(16)(p13;q22)
  • adult acute myeloid leukemia with t(15;17)(q22;q12)
  • adult acute myeloid leukemia with t(16;16)(p13;q22)
  • adult acute myeloid leukemia with t(8;21)(q22;q22)
  • untreated adult acute myeloid leukemia
  • recurrent adult acute myeloid leukemia
  • childhood acute myeloid leukemia in remission
  • recurrent childhood acute myeloid leukemia
  • untreated childhood acute myeloid leukemia and other myeloid malignancies
  • de novo myelodysplastic syndromes
  • previously treated myelodysplastic syndromes
  • secondary myelodysplastic syndromes
  • secondary acute myeloid leukemia
  • childhood acute lymphoblastic leukemia in remission
  • recurrent adult acute lymphoblastic leukemia
  • recurrent childhood acute lymphoblastic leukemia
  • untreated adult acute lymphoblastic leukemia
  • untreated childhood acute lymphoblastic leukemia
  • childhood myelodysplastic syndromes
  • Neoplasms
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms



City of Hope Medical Center Duarte, California  91010