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Phase II Trial of Arsenic Trioxide and Dose-Escalated Cholecalciferol in Myelodysplastic Syndrome


Phase 2
N/A
N/A
Not Enrolling
Both
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms

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Trial Information

Phase II Trial of Arsenic Trioxide and Dose-Escalated Cholecalciferol in Myelodysplastic Syndrome


OBJECTIVES:

Primary

- Determine the complete response rate and the rate of hematological improvement in
patients with myelodysplastic syndromes treated with arsenic trioxide and
cholecalciferol (vitamin D).

Secondary

- Determine the safety of this regimen in these patients.

- Determine the time to progression to acute myeloid leukemia, defined as blast ≥ 20%, in
patients treated with this regimen.

- Determine overall survival and progression-free survival of patients treated with this
regimen.

- Determine the effect of this regimen on bone marrow and peripheral blood mononuclear
cell apoptosis and p21 protein expression in these patients.

OUTLINE: This is an open-label, nonrandomized study.

Patients receive oral cholecalciferol (vitamin D)* once daily on days 1-28. Patients also
receive arsenic trioxide IV over 1-4 hours on days 1-5 (week 1) and then twice weekly for 3
weeks (weeks 2-4) for course 1 and twice weekly for 4 weeks for all subsequent courses.
Courses repeat every 28 days for up to 12 months in the absence of disease progression or
unacceptable toxicity.

NOTE: * Patients who do not achieve a complete hematologic response receive escalating doses
of cholecalciferol (vitamin D) at 3, 6, and 9 months during therapy in the absence of
disease progression and unacceptable toxicity.

At the completion of study treatment, patients are followed for survival.

PROJECTED ACCRUAL: A total of 25-60 patients will be accrued for this study.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of myelodysplastic syndromes (MDS)

- Bone marrow aspirate and biopsy with karyotyping performed within the past 12
weeks

PATIENT CHARACTERISTICS:

Age

- Any age

Performance status

- ECOG 0-2

Life expectancy

- More than 6 months

Hematopoietic

- Ferritin ≥ 50 ng/mL

- Folate (serum and/or red blood cell) normal

Hepatic

- Not specified

Renal

- Creatinine < 2.0 mg/dL

- No history of hypercalcemia

Cardiovascular

- Absolute QT interval ≤ 460 msec by EKG with normal potassium and magnesium levels

Other

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception during and for 2 weeks after study
participation

- Serum vitamin B_12 normal

PRIOR CONCURRENT THERAPY:

Biologic therapy

- Prior biologic therapy allowed

- More than 28 days since prior hematopoietic growth factors (e.g., filgrastim [G-CSF],
sargramostim [GM-CSF], or epoetin alfa) for MDS

- No concurrent hematopoietic growth factors (e.g., G-CSF, GM-CSF, or epoetin alfa)

- No concurrent interleukin-11

Chemotherapy

- Prior chemotherapy allowed

Endocrine therapy

- Not specified

Radiotherapy

- Prior radiotherapy allowed

Surgery

- Not specified

Other

- More than 28 days since prior therapy for MDS except supportive therapy

- No concurrent cholecalciferol (vitamin D) analog, including topical therapy

- No concurrent vitamins or supplements containing cholecalciferol (vitamin D)

- No other concurrent therapy for MDS

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Complete response rate

Outcome Time Frame:

6 months

Safety Issue:

No

Principal Investigator

Istvan Molnar, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Comprehensive Cancer Center of Wake Forest University

Authority:

United States: Institutional Review Board

Study ID:

CDR0000415574

NCT ID:

NCT00104806

Start Date:

November 2004

Completion Date:

May 2010

Related Keywords:

  • Leukemia
  • Myelodysplastic Syndromes
  • Myelodysplastic/Myeloproliferative Neoplasms
  • de novo myelodysplastic syndromes
  • myelodysplastic/myeloproliferative neoplasm, unclassifiable
  • previously treated myelodysplastic syndromes
  • secondary myelodysplastic syndromes
  • refractory anemia with excess blasts in transformation
  • refractory anemia with excess blasts
  • refractory anemia
  • refractory anemia with ringed sideroblasts
  • refractory cytopenia with multilineage dysplasia
  • chronic myelomonocytic leukemia
  • childhood myelodysplastic syndromes
  • Neoplasms
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Myeloproliferative Disorders
  • Myelodysplastic-Myeloproliferative Diseases

Name

Location

Wake Forest University Comprehensive Cancer CenterWinston-Salem, North Carolina  27157-1096