Clinical Trials Resource Center
New Medical Therapies™
Patient Medical Areas
Clinuvel Pharmaceuticals reported results
of a phase III study of SCENESSE (afamelanotide
16mg) to patients with erythropoietic
protoporphyria (EPP). The six-month, randomized,
multicenter, double-blind, placebocontrolled
study enrolled 93 adult EPP
patients in the U.S. Patients were randomized
into two treatment groups and given
either SCENESSE or placebo implants every
two months. Median total direct sunlight
exposure was 64.13 hours (range 0 to 650.5
hours) in the active group compared with
47.5 hours (range 0 to 224 hours) for placebo
recipients (p=0.107, Kruskal-Wallis test). The
distribution of the number of days with sun
exposure of various time intervals (30-minute
intervals) was significantly different between
the treatment groups (p<0.001, Cochran-
Mantel-Haenszel test). SCENESSE recipients
reported more days when they had pain-free
exposure of 60 minutes or more (the time of
greatest risk of burns). The results showed
that after receiving their second SCENESSE
administration, patients had a significantly
higher tolerance to light irradiation on the
lower back and back of the hand (median
change from baseline in minimum symptom
dose on lower back at day 90, 227.5 v.
-2.4 J/cm2; p<0.001, Wilcoxon test). The
difference in the distribution of days during
which pain was experienced was significant
between the two treatment groups
(p<0.0001, Cochran-Mantel-Haenszel test).
The final stage of EMA review of SCENESSE is
expected to continue in January 2014.
Kuros reported positive interim results from a phase IIa trial of KUR-212 for the treatment of severe burns. This randomized, open-label, active-control, single-group assignment study enrolled 10 subjects with severe burns requiring autologous meshed skin grafting. The subjects served as their own control, with an area of graft that was stapled and an area of graft that was treated with KUR-212. The trial was designed to assess the safety and efficacy of KUR-212 on wound closure (full re-epithelialization) over two separately reported periods of one and 12 months respectively. The primary endpoint was incidence of treatment-related adverse events up to 28 days post surgery. In the one-month follow-up there were no treatment-related adverse events. A difference was observed in the time to wound closure between treatments, with a shorter time for the KUR-212-treated group. However, the small sample size did not allow for statistical evaluation of efficacy. The 12 month data is expected in Q2 2009.
ZymoGenetics reported positive data from a clinical trial of Recothrom in subjects undergoing skin grafting for burns. This multiple site, single-arm, open-label study enrolled 72 subjects receiving partial- or full-thickness autologous sheet or mesh graft following burn injury. Recothrom was applied to newly excised wounds (graft recipient sites) using a ZymoGenetics pump spray device. Adverse events, skin graft survival and formation of anti-Recothrom antibodies were measured at baseline and Day 29 after application of Recothrom. Treatment was generally well tolerated; adverse events occurred in 63 (88%) of the subjects and were typical of adverse events seen following acute burn wound treatment. One subject had antibodies to Recothrom at baseline (1.4%); another subject developed specific antibodies to Recothrom at Day 29 resulting in a low rate of antibody formation (1.6%). The antibodies detected did not neutralize human thrombin.
BioMarin reported positive results of their phase I b study of Vibrilase (vibriolysin), their topical enzymatic treatment for serious burns. Data indicated that the drug was safe and well tolerated at all trial doses, and demonstrated preliminary efficacy in debriding burn tissues and improving the time to 90% epithelialization (the point at which wounds appear to be healing normally); cohort size was deemed insufficient to establish a dose response relationship. The trial found the drug to be safe and well tolerated, with non-dose related transient application pain observed most frequently. The trial collected safety data from a total of 19 subjects, and efficacy data from 12 subjects, all of whom were conscious and had partial-thickness burns over 1-2% of their body. Subjects received one of three topical doses of Vibrilase daily for three days, followed by a 28 day observation period.