Conditions: Erythropoietic Protoporphyria
Keywords: Erythropoietic Protoporphyria EPP Afamelanotide
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 100
Healthy volunteers: no

Brief summary:
This was a phase III, multicentre, randomised, double-blind, placebo-controlled study, to evaluate the safety and efficacy of subcutaneous bioresorbable afamelanotide implants in patients with Erythropoietic Protoporphyria (EPP).

The study was conducted with two parallel study arms with crossover between treatments every 60 days.

Eligible patients were randomised to a treatment group, and received implants of active treatment (afamelanotide 16mg) or placebo, in an alternating crossover fashion according to the following dosing regime:

* Group A was administered active implants on Days 0, 120, 240 and placebo implants on Days 60, 180, 300
* Group B was administered placebo implants on Days 0, 120, 240 and active implants on Days 60, 180, 300

Detailed description:
Afamelanotide is a man-made drug being studied for use as a preventative medication for Erythropoietic Protoporphyria (EPP) sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH).

The study will involve the use of an implant, which comes in the form of a small rod to be administered under the skin. The implant may contain the study drug afamelanotide or a placebo (inactive medication).

This study aims to provide insight into the effectiveness of afamelanotide under normal conditions of use in EPP patients.

Arms:
arm 1: Group A was administered active implants on Days 0, 120, 240 and placebo implants on Days 60, 180, 300 arm 2: Group B was administered placebo implants on Days 0, 120, 240 and active implants on Days 60, 180, 300

Interventions: intervention 1: Afamelanotide intervention 2: Placebo
Intervention descriptions: intervention 1: 16mg subcutaneous implant intervention 2: Placebo subcutaneous implant

Protocol document: