This trial is active, not recruiting.

Condition myelodysplastic syndrome
Treatment dn-101 (calcitriol)
Phase phase 2
Sponsor Novacea
Start date November 2002
End date March 2004
Trial size 46 participants
Trial identifier NCT00057031, DN101-003


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Masking open label
Primary purpose treatment

Eligibility Criteria

Male or female participants at least 18 years old.

- 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

Additional Information

Official title 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
Description 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
Trial information was received from ClinicalTrials.gov and was last updated in October 2006.
Information provided to ClinicalTrials.gov by Novacea.