Bone Marrow Transplant

February 20, 2012

AiCuris reported results from a phase IIb trial of letermovir for the prevention of human cytomegalovirus (HCMV) following bone marrow transplantation. This randomized, double-blind, placebo controlled trial enrolled 133 subjects undergoing allogeneic stem cell transplant. The subjects received one of three doses of letermovir or placebo for 12 weeks. The primary endpoints were the incidence and time to onset of HCMV prophylaxis failure. Two doses of letermovir, 120 mg and 240 mg once daily for 84 days, meet both primary efficacy endpoints with high statistical significance versus placebo. The incidence of failure due to efficacy failure of prophylaxis or due to discontinuation of treatment was significantly lower in the Letermovir 240 mg/day (29.4%; p≡0.007) and 120 mg/day (32.3%; p≡0.014) groups compared to placebo (63.6%). The incidence of HCMV prophylaxis failure among subjects receiving treatment for at least seven days prior to HCMV replication was none for Letermovir 240 mg (p≡0.004 vs. placebo) and two subjects for the 120 mg group (p≡0.109 vs. placebo). Similarly, the time to onset of prophylaxis failure among subjects receiving 240 mg/day of Letermovir was significantly different (p≡0.02) compared to those receiving placebo. All doses were safe and well tolerated.

October 15, 2007

CuraGen released negative results from a phase II trial of velafermin for the treatment of oral mucositis. This randomized, double-blind, placebo-controlled study enrolled 390 subjects on high dose chemotherapy, in the United States. The subjects received a single infusion of either placebo or one of three dose levels of velafermin (10 mcg/kg, 30 mcg/kg or 60 mcg/kg) administered 24 hours after an autologous bone marrow transplantation. The primary endpoint was the reduction in incidence of severe oral mucositis in the subjects receiving 30 mcg/kg compared to placebo. While treatment was deemed to be safe and well tolerated, the primary endpoint was not met. Based on the results, CuraGen has decided to discontinue the development of velafermin.

September 10, 2007

Kiadis released positive results from a phase II trial of Reviroc for the treatment of hematological cancers. This non-randomized open label study enrolled 25 subjects with end-stage Non-Hodgkin's lymphoma in Canada. All subjects underwent an autologous graft in bone marrow transplantation using Reviric or a control. The objective of the study was to determine the safety of the Reviroc treatment and its ability to eliminate cancer cells from a contaminated graft. Reviroc was able to eliminate cancer cells from a contaminated graft with out negatively impacting the graft itself. In addition, the Reviroc group had an 80% chance of survival at three years post-transplantation while the control group had a 55% chance of survival. Based on the results, Kiadis is preparing to commence phase III trials.

January 10, 2005

DOR BioPharma reported top-line results from a phase III trial of orBec (oral beclomethasone dipropionate), for the treatment of intestinal Graft-versus-Host Disease (iGVHD). The results indicate that orBec failed to meet its primary endpoint, significant improvement in time to treatment failure through day 50 vs. placebo (p=0.1177), nor was the treatment failure rate at 50 days statistically significant (p=0.0515). The drug did achieve significance in several secondary endpoints, including time to treatment failure at day 80 (p=0.0226), treatment failure rate at day 80 (0.0048), and mortality rate 200 days post transplant (p=0.006). The randomized, double-blind, placebo-controlled, multi-center clinical trial enrolled 129 post-bone marrow transplant patients, who received high-dose prednisone plus orBec or placebo for 10 days. If initial response was noted after 10 days, prednisone was rapidly tapered and subjects continued to receive orBec or placebo through day 50.

January 27, 2003

Amgen reported positive results from a phase III trial investigating rHu-KGF, a natural keratinocyte growth factor for the treatment of oral mucositis as a complication of cancer treatments. Preliminary results from the randomized, double blind trial were positive on all endpoints showing a highly significant decrease in both the duration and incidence of severe mucositis. In addition, results showed that the drug was well tolerated. The study enrolled subjects who underwent bone marrow transplantation treatment for hematologic malignancies such as lymphoma, multiple myeloma, and leukemia.

August 12, 2002

The results of a phase I trial of OC-1012 for the treatment of mucositis, a serious side effect of chemotherapy and head and neck radiation therapy, showed no safety issues with the compound. In addition, OC-1012 positively changed the mucositis profile over time. Subjects in the trial were bone marrow transplant patients undergoing chemotherapy treatment. OC-1012 is being developed by OraPharma.

December 17, 2001

Phase II trial results show that SuperGen's Nipent (pentostatin) produced a 60% overall response rate in subjects with refractory chronic graft versus host disease (GvHD). The trial consisted of 10 evaluable subjects who had failed at least one prior immunosuppressive regimen. Subjects received Nipent every two weeks for three months, and at the end of this period those who had achieved either stable disease or who had shown improvement were weaned off other medications. Five subjects experienced a complete response and one had a partial response, producing an overall response rate of 60%.

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