Table 2.
Primary and Secondary End Points.*
| End Point | Afamelanotide | Placebo | P Value |
|---|---|---|---|
| European Union trial | |||
| No. of patients | 38 | 36 | |
| Primary end point: no. of hours in direct sunlight between 10 a.m. and 3 p.m. without pain | 0.005 | ||
| Median per patient (range) | 6.0 (0–193) | 0.8 (0–35) | |
| Mean per patient | 20.4±40.5 | 5.6±9.3 | |
| Secondary end points | |||
| Phototoxic reactions — no.† | 0.04 | ||
| Median per patient (range) | 1.0 (0–11) | 2.0 (0–20) | |
| Mean per patient | 2.0±2.8 | 4.1±5.1 | |
| Phototoxic reactions during study — no. | 77 | 146 | 0.04 |
| Duration of longest phototoxic reaction — days | 0.08 | ||
| Median per patient (range) | 1.0 (0–7) | 2.0 (0–37) | |
| Mean per patient | 1.5±1.8 | 3.8±7.4 | |
| Duration of phototoxicity — days | 0.04 | ||
| Median per patient (range) | 1.0 (0–23) | 3.0 (0–90) | |
| Mean per patient | 3.7±5.6 | 10.0±18.3 | |
| Sum of Likert score for severity of phototoxic reactions during study | 0.02 | ||
| Median per patient (range) | 5.0 (0–113) | 17.5 (0–490) | |
| Mean per patient | 18.0±27.9 | 52.9±98.2 | |
| Patients with severe phototoxic reactions — no. (%) | 25 (66) | 28 (78) | 0.25 |
| U.S. trial | |||
| No. of patients | 46 | 43 | |
| Primary end point: no. of hours in direct sunlight between 10 a.m. and 6 p.m. without pain | 0.04 | ||
| Median per patient (range) | 69.4 (0–651) | 40.8 (0–224) | |
| Mean per patient | 115.6±140.6 | 60.6±60.6 | |
| Secondary end points | |||
| No. of days in some direct sunlight without pain | 0.005 | ||
| Median per patient (range) | 85.5 (0–167) | 54.0 (0–124) | |
| Mean per patient | 80.5±48.9 | 510.7±37.3 | |
| No. of hours in direct sunlight between 10 a.m. and 6 p.m. on days with no pain or mild pain | 0.05 | ||
| Median per patient (range) | 80.0 (0.5–825) | 51.0 (1.25–251) | |
| Mean per patient | 141.1±165.1 | 74.6±67.5 | |
| No. of days with some sunlight between 10 a.m. and 6 p.m. on days with no pain or mild pain | 0.004 | ||
| Median per patient (range) | 97.0 (2–185) | 61.0 (3–145) | |
| Mean per patient | 93.9±51.0 | 64.0±40.6 | |
| No. of hours in direct sunlight between 10 a.m. and 3 p.m. without pain | 0.09 | ||
| Median per patient (range) | 39.6 (0–419) | 31.8 (0–199) | |
| Mean | 71.2±89.2 | 41.6±45.3 | |
| Phototoxic reactions — no.† | 0.60 | ||
| Median per patient (range) | 1.0 (0–15) | 1.0 (0–35) | |
| Mean per patient | 2.0±3.3 | 3.3±6.8 | |
| Duration of phototoxic reactions — days | 0.50 | ||
| Median (range) | 1.0 (0–34) | 1.0 (0–98) | |
| Mean | 3.2±6.0 | 6.6±16.8 | |
| Sum of Likert score for severity of phototoxic reactions during study | 0.44 | ||
| Median per patient (range) | 4.0 (0–196) | 6.0 (0–507) | |
| Mean per patient | 16.3±33.2 | 34.1±86.7 |
*
Plus-minus values are means ±SD. P values for the differences between the afamelanotide and placebo groups were calculated with the use of the Kruskal-Wallis test.
†
A phototoxic reaction was defined as a Likert score of 4 or higher. Scores on the Likert scale range from 0 to 10, with 0 indicating no pain and higher scores indicating greater severity of symptoms. Scores between 1 and 3 indicate mild pain.