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

• Sponsor: Novacea (Industry)
• Overall Status: Unknown status
• CT.gov ID: NCT00057031
• Collaborator: (none)
• Enrollment: 46
• Locations: 10
• Duration (Months): 16
• Patients Per Site: 4.6
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndrome	Drug: DN-101 (calcitriol)	Phase 2

Detailed 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

Study Design

• Study Type: Interventional
• Allocation: Non-Randomized
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Study Start Date: Nov 1, 2002
• Study Completion Date: Mar 1, 2004

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

• 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Research Consultants, Inc.	Hoover	Alabama	United States	35216
2	University of California, San Francisco	San Francisco	California	United States	94143
3	James A. Haley Veterans Hospital	Tampa	Florida	United States	33612
4	Rush Cancer Institute MDS Center	Chicago	Illinois	United States	60612
5	Roswell Park Cancer Institute	Buffalo	New York	United States	14263
6	The Cleveland Clinic Foundation, Taussig Cancer Center	Cleveland	Ohio	United States	44195
7	Oregon Health Sciences University	Portland	Oregon	United States	97201
8	Boston Baskin Cancer Group	Memphis	Tennessee	United States	38104
9	University of Texas - MD Anderson Cancer Center	Houston	Texas	United States	77030
10	Fred Hutchinson Cancer Research Center	Seattle	Washington	United States	98109

Sponsors and Collaborators

• Novacea

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

• 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.
• 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.
• 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.

• ClinicalTrials.gov Identifier: NCT00057031
• Other Study ID Numbers: DN101-003
• First Posted: Apr 1, 2003
• Last Update Posted: Nov 1, 2006
• Last Verified: Feb 1, 2005

• Keywords provided by , ,: myelodysplastic syndrome; MDS
• Additional relevant MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome; Calcitriol

Study Results

No Results Posted as of Nov 1, 2006