Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Status:
Completed
Trial end date:
2011-05-01
Target enrollment:
Participant gender:
Summary
Afamelanotide is a man-made drug being studied for use as a preventative medication for EPP
sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating
hormone (alpha-MSH) and is not yet available on the market.
The purpose of this study is to look at whether afamelanotide can reduce the number and
severity of EPP symptoms when patients are exposed to light. This study will also look at how
the drug is tolerated when taken by people with EPP.
The study will involve the use of an implant, which comes in the form of a small rod
(approximately 2 cm x 0.15 cm) to be administered under the skin. The implant may contain the
study drug afamelanotide or a placebo (inactive medication).
Over 450 subjects have been treated with afamelanotide to date with no serious safety
concerns identified. For this study, afamelanotide has been formulated as a controlled
release depot injection (implant). This means that the afamelanotide will be released slowly
into the body over a few days. Once inserted, the implant will remain in the body after
afamelanotide has been released and will slowly dissolve.
This study will help to provide more information about afamelanotide. This information will
be used to determine the safety and efficacy (the ability of the drug to produce an effect)
of this drug in EPP sufferers.
Up to 70 people will participate in this study from study sites across Europe.