Novacea Inc announced that patient enrollment has begun in a Phase 2 trial of its lead product, DN-101, for the potential treatment of patients with a blood disorder called myelodysplastic syndromes (MDS), a condition for which there is no approved therapy.

The study will evaluate the effect of high dose pulse administration (HDPA) DN-101 (calcitriol) on the anemia and red blood cell transfusion requirements for patients with MDS. Results of previous preclinical and clinical studies have suggested that calcitriol may be useful in treating MDS. Up to 46 patients with low-risk MDS who are red blood cell transfusion dependent because of severe anemia will be enrolled in this study at 12 research centers in the United States.

"We have made rapid progress in the clinical development of DN-101 and are excited to expand our program to explore this additional potential indication," said John Curd, President and Chief Medical Officer of Novacea. "We are evaluating high dose pulse administration of DN-101 as a therapy for patients with MDS who have few, if any, effective treatment options."

"MDS is a challenging area and we are hopeful that DN-101 may bring relief to some of these patients," said Dr. Robert L. Capizzi, a new member of the senior clinical team at Novacea who will be the physician in charge of the clinical trials of DN-101 in MDS. He brings years of experience in the development of novel therapies for hematologic malignancies in adults and children, some of which are curative. He also is the principle author of a patent for a novel therapy for MDS. "This study will be an important step in our research program for DN-101 and will provide important information to take the product's development to the next stage."

The objectives of this Phase 2, multicenter, single arm, open label clinical trial are safety and efficacy. Efficacy will be determined by measuring the change in the number of blood transfusions along with the change in the degree of anemia manifested as fatigue; changes in the number of white blood cells that influence the incidence of infections requiring antibiotics; and platelets that control the incidence of bleeding events.