Study of High-Dose Pulse Administration DN-101 (Calcitriol) in Patients With Myelodysplastic Syndrome (MDS)

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: NCT00057031
Recruitment Status : Unknown
Verified February 2005 by Novacea.
Recruitment status was:  Active, not recruiting
First Posted : April 1, 2003
Last Update Posted : November 1, 2006
Information provided by:

March 26, 2003
April 1, 2003
November 1, 2006
November 2002
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Complete list of historical versions of study NCT00057031 on Archive Site
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Study of High-Dose Pulse Administration DN-101 (Calcitriol) in Patients With Myelodysplastic Syndrome (MDS)
A Phase 2, Multicenter, Open Label Study of the Safety and Efficacy of High-Dose Pulse Administration DN-101 (Calcitriol) in Patients With Myelodysplastic Syndrome
The purpose of this study is to determine the safety and efficacy of DN-101 (calcitriol) in patients with myelodysplastic syndrome who are dependent on repeat blood transfusions.

DN-101 is an experimental drug that has not been approved by the Food and Drug Administration (FDA). It is a newly formulated pill that contains high amounts of calcitriol, a naturally occurring hormone and the biologically active form of vitamin D. The natural vitamin D found in dairy products or in typical vitamin pills, must be chemically changed by the liver and kidney into calcitriol before it is biologically active. The body normally uses small amounts of calcitriol to regulate its blood calcium levels. However, for any possible therapeutic effect, MDS patients require much higher levels of calcitriol than the body can produce from dietary vitamin D. DN-101 provides MDS patients with high doses of calcitriol in a pill form.

Laboratory studies have demonstrated evidence supporting the use of calcitriol in MDS. High dose calcitriol slows the growth of leukemic cells (cancerous cells) and increases the growth of normal bone marrow cells. Some patients with MDS may have low levels of calcitriol in their bone marrow.

Clinical study results in patients with MDS have been mixed– some positive and some negative results. Elevated calcium in the blood occurred frequently and prevented the use of higher, more potentially therapeutic doses.

Novacea tested a new formulation of calcitriol, DN-101, in a Phase 1 study. In that study the maximum tolerated dose of DN-101 that did not cause high blood calcium levels when given weekly for several months was determined. That dose is within the range that is potentially therapeutic for MDS patients and will be used in this MDS study.

The purposes of this study are to determine if HDPA DN-101 treatment:

  • increases the number of red blood cells, white blood cells, and platelets in the blood
  • reduces the number of blood transfusions
  • reduces the number of serious infections requiring antibiotics
  • reduces the number of serious bleeding events
  • improves fatigue
Phase 2
Allocation: Non-Randomized
Masking: None (Open Label)
Primary Purpose: Treatment
Myelodysplastic Syndrome
Drug: DN-101 (calcitriol)
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Unknown status
Same as current
March 2004
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  • Diagnosis of low or intermediate-1 risk MDS
  • Dependent on monthly blood transfusions
  • No cancer within the last 5 years (cured skin cancer is allowed)
  • No heart attack or stroke within the last 6 months
  • No kidney stones within the last 5 years
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
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February 2005

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP