This is a study to determine the response rate in patients with myelodysplastic syndromes treated with calcitriol and dexamethasone.

Condition	Treatment or Intervention	Phase
Myelodysplastic Syndromes	Drug: Calcitriol  Drug: Dexamethasone	Phase II

Current therapeutic options for myelodysplastic syndromes (MDS) are limited and, aside from bone marrow transplantation, none have proven superior to supportive measures alone. Preclinical investigations have indicated the potential therapeutic role for vitamin D in the treatment of MDS. However, because of the dose-limiting toxicity of hypercalcemia, past clinical trials with vitamin D have been forced to utilize low doses, with promising but inconsistent results.

This study utilizes a dosing schema of dexamethasone (Dex) and calcitriol (the active form of vitamin D) that augments the therapeutic index of calcitriol, and allows for safe administration of 5-10 times higher dose of calcitriol than previously has been used in clinical trials for MDS. Patients will receive dexamethasone 4 times per week and calcitriol 3 times per week. This schedule will continue weekly until patients are off study. The dose of calcitriol will be increased until the maximum tolerated dose (MTD) is determined. History and physical examination, blood monitoring, urinary ultrasounds, and bone marrow aspirations and biopsies will be used to assess disease response.

Genders Eligible for Study:  Both

Criteria

Inclusion criteria:

• Histologically confirmed refractory anemia (RA), RA with excess blasts (RAEB), RAEB in transformation (RAEB-IT), or ringed sideroblasts (RARS)
• Evidence of cytopenia affecting at least 1 hematological cell lineage
• Adequate liver and renal function
• ECOG 0-2
• Expected survival of at least 12 weeks

Exclusion criteria:

• Symptomatic coronary artery disease
• Uncontrolled diabetes mellitus
• Uncontrolled and symptomatic glaucoma
• History of dangerous reactions to steroid therapy
• Chemotherapy or any hematopoietic growth factor therapy within the past 8 weeks
• History of nephrolithiasis
• Children
• Chronic myelomonocytic leukemia (CMML)

Pennsylvania
      University of Pittsburgh, Pittsburgh,  Pennsylvania,  15213,  United States; Recruiting

Study ID Numbers:  FD-R-2025-01; FD-R-002025-01
Record last reviewed:  January 2002
Record first received:  January 30, 2002
ClinicalTrials.gov Identifier:  NCT00030069
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2004-11-09