Arsenic Trioxide and Cholecalciferol (Vitamin D) in Treating Patients With Myelodysplastic Syndromes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00104806
Recruitment Status : Terminated (sponsor discontinues support)
First Posted : March 4, 2005
Last Update Posted : May 30, 2017
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Wake Forest University Health Sciences

Brief Summary:

RATIONALE: Drugs used in chemotherapy, such as arsenic trioxide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Cholecalciferol (vitamin D) may help cancer cells become normal cells. Giving arsenic trioxide together with cholecalciferol (vitamin D) may kill more cancer cells.

PURPOSE: This phase II trial is studying how well giving arsenic trioxide together with cholecalciferol (vitamin D) works in treating patients with myelodysplastic syndromes.

Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms Dietary Supplement: cholecalciferol Drug: arsenic trioxide Phase 2

Detailed Description:



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


  • 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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of Arsenic Trioxide and Dose-Escalated Cholecalciferol in Myelodysplastic Syndrome
Study Start Date : November 2004
Actual Primary Completion Date : January 2006
Actual Study Completion Date : May 2010

Intervention Details:
  • Dietary Supplement: cholecalciferol
    100 milligrams orally once a day for 28 days
  • Drug: arsenic trioxide
    0.3 milligram/kilogram weight intravenously for 5 days (loading) then 0.25/kg weight intravenously biweekly

Primary Outcome Measures :
  1. Complete response rate [ Time Frame: 6 months ]
  2. toxicity assessment after therapy [ Time Frame: 28 days ]

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Ages Eligible for Study:   up to 120 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of myelodysplastic syndromes (MDS)

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



  • Any age

Performance status

  • ECOG 0-2

Life expectancy

  • More than 6 months


  • Ferritin ≥ 50 ng/mL
  • Folate (serum and/or red blood cell) normal


  • Not specified


  • Creatinine < 2.0 mg/dL
  • No history of hypercalcemia


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


  • 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


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


  • Prior chemotherapy allowed

Endocrine therapy

  • Not specified


  • Prior radiotherapy allowed


  • Not specified


  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00104806

United States, North Carolina
Wake Forest University Comprehensive Cancer Center
Winston-Salem, North Carolina, United States, 27157-1096
Sponsors and Collaborators
Wake Forest University Health Sciences
National Cancer Institute (NCI)
Study Chair: Istvan Molnar, MD Wake Forest University Health Sciences

Responsible Party: Wake Forest University Health Sciences Identifier: NCT00104806     History of Changes
Other Study ID Numbers: CDR0000415574
First Posted: March 4, 2005    Key Record Dates
Last Update Posted: May 30, 2017
Last Verified: July 2012

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Wake Forest University Health Sciences:
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

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Arsenic trioxide
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents
Antineoplastic Agents