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

Myelodysplastic Syndrome Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00057031
• Other study ID #: DN101-003
• Status: Active, not recruiting
• Phase: Phase 2
• First received: March 26, 2003
• Last updated: October 31, 2006
• Start date: November 2002
• Est. completion date: March 2004

Study information

• Verified date: February 2005
• Source: Novacea
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 46
• Est. completion date: March 2004
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

• 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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Drug:
• DN-101 (calcitriol)

Locations

Country	Name	City	State
United States	Roswell Park Cancer Institute	Buffalo	New York
United States	Rush Cancer Institute MDS Center	Chicago	Illinois
United States	The Cleveland Clinic Foundation, Taussig Cancer Center	Cleveland	Ohio
United States	Clinical Research Consultants, Inc.	Hoover	Alabama
United States	University of Texas - MD Anderson Cancer Center	Houston	Texas
United States	Boston Baskin Cancer Group	Memphis	Tennessee
United States	Oregon Health Sciences University	Portland	Oregon
United States	University of California, San Francisco	San Francisco	California
United States	Fred Hutchinson Cancer Research Center	Seattle	Washington
United States	James A. Haley Veterans Hospital	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Novacea

Country where clinical trial is conducted

United States, 

References & Publications (3)

Beer TM, Eilers KM, Garzotto M, Egorin MJ, Lowe BA, Henner WD. Weekly high-dose calcitriol and docetaxel in metastatic androgen-independent prostate cancer. J Clin Oncol. 2003 Jan 1;21(1):123-8. — View Citation

Beer TM, Munar M, Henner WD. A Phase I trial of pulse calcitriol in patients with refractory malignancies: pulse dosing permits substantial dose escalation. Cancer. 2001 Jun 15;91(12):2431-9. — View Citation

Mellibovsky L, Díez A, Pérez-Vila E, Serrano S, Nacher M, Aubía J, Supervía A, Recker RR. Vitamin D treatment in myelodysplastic syndromes. Br J Haematol. 1998 Mar;100(3):516-20. — View Citation