Clinical Trials Resource Center

Congenital Skin Diseases

October 24, 2016

Trevi Therapeutics reported results of a phase II study of Nalbuphine ER for the treatment of moderate to severe prurigo nodularis. The multicenter, randomized, double-blind, placebo-controlled, parallel, three-arm study evaluated the safety and anti-pruritic efficacy of Nalbuphine ER tablets dosed twice-daily at 90mg and 180mg in 62 patients in the U.S. and Europe. Patients with moderate to severe itch intensity, defined as ≥5 on the zero to 10 Numerical Rating Score (NRS) scale, were enrolled to evaluate drug efficacy across a representative patient population for treatment of this chronic indication. The actual average baseline worst itch for enrolled patients was ≥8, indicating the severe nature of the disease. The main outcome variables for this study were responder analyses of the proportion of patients with at least a 30% or 50% reduction in their seven-day worst itch intensity NRS from baseline to completion of treatment at week 10 or last observation visit. The proportion of patients in the Nalbuphine ER 180mg BID arm meeting 50% responder criteria at week 10 or last observed visit (MITT population with n=18) approached statistical significance (p=0.083), and this arm met statistical significance for patients (n=12) completing treatment (p=0.028). The mean change in worst itch NRS was additionally evaluated, and the MITT population of the Nalbuphine ER 180mg BID arm as compared to placebo approached statistical significance (p=0.083) as well. This arm also met statistical significance for patients (n=12) completing treatment (p=0.025). The company expects the open label extension study to be completed in the third quarter of 2017.

November 7, 2011

Clinuvel reported results from a phase II trial of afamelanotide for the treatment of Erythropoietic Protoporphyria (EPP). The randomized, double-blind, placebo-controlled study (CUV030) enrolled 77 subjects, 68 of whom completed the trial. The subjects were administered either the afamelanotide implant or placebo at the start of study, after 60 days and 120 days, and were followed up to 180 days. The primary endpoint was to determine whether afamelanotide implants can reduce the severity of phototoxic reactions associated with EPP. Based on analysis of time spent outside, afamelanotide was shown to increase subjects ability to expose their skin to direct sunlight compared to placebo. The subjects who received afamelanotide spent significantly more time in direct sunlight between the most intense hours of 10 AM and 3 PM (p≡0.036) and between 10 AM and 8 PM (p≡0.025). Data from an EPP-specific quality of life assessment also demonstrated an improvement from baseline for subjects receiving afamelanotide compared to placebo at 60 days (p≡0.001), 120 days (p≡0.003) and 180 days (p<0.001). The afamelanotide implant was safe and well-tolerated.

July 19, 2010

Clinuvel reported positive results from a phase III trial of afamelanotide for the treatment of erythropoietic protoporphyria (EPP). This multicenter, randomized, double-blind, placebo-controlled trial, CUV017, treated 91 subjects over the course of 12 months. The subjects received a 16 mg subcutaneous resorbable implant formulation of afamelanotide or placebo once every two months for a total of six implants. The primary endpoints were the mean number of phototoxic reactions and the mean severity score for phototoxic reactions for active compared with placebo implants. The frequency of specific pain severity (severe, moderate, mild and none) was significantly reduced in the afamelanotide arm versus placebo (p≡0.0023). The average pain severity was also significantly reduced in the afamelanotide arm compared to placebo (p≡0.0017). An additional evaluation of the pain scores in patients willing to modify behavior by continuous exposure to daily sunlight showed a positive trend toward a reduction in average pain score following active drug treatment (p≡0.1654). The afamelanotide implant was well tolerated.

January 11, 2010

Clinuvel reported positive preliminary results from a phase III trial of afamelanotid for the treatment of erythropoietic protoporphyria (EPP). To date, this randomized, placebo-controlled trial (CUV017) has enrolled 100 subjects across Europe and Australia. The subjects received alternating 16 mg afamelanotid or placebo implants once every two months, for a total of six implants administered subcutaneously over a 12 months period. The primary endpoints were number and severity of phototoxic skin reactions. This analysis is from all 100 subjects who had received the first 4 months of treatment, each of whom had received a single dose of either treatment. Both treatment arms showed an overall reduction in the average number of phototoxic reactions. The greatest reduction in mean number of reactions (p≡0.03) was reported in the subgroup of subjects with moderate to severe pain (n≡35). Analysis of pain severity was positively correlated with treatment, indicating that pain scores differed significantly between treatment groups (p≡0.006).

January 26, 2009

Clinuvel issued positive interim results from a phase III trial of afamelanotide for the treatment of erythropoietic protoporphyria (EPP). To date, this randomized, placebo-controlled trial had enrolled 14 subjects who received 16 mg implants of the drug. The primary endpoints are reduction in the number and severity of phototoxic skin reactions. The results showed that the maximum severity of phototoxic reactions for subjects on the drug was significantly reduced compared to those on the placebo (p<0.001). In addition, the total severity of phototoxic reactions was reduced during spring and summer by afamelanotide, compared with placebo (p≡0.028). No drug related serious adverse events were observed. Based on the data, Clinuvel plans to continue with the study as planned; full results are expected by the end of 2009.

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