NCT04053270

Multicentre Phase III Erythropoietic Protoporphyria Study

A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutanenous Bioresorbable CUV1647 Implants in Patients With Erythropoietic Protoporphyria (EPP)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: Clinuvel Pharmaceuticals Limited · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Erythropoietic Protoporphyria

Interventions

Type	Name	Description
DRUG	Afamelanotide	16mg subcutaneous implant
DRUG	Placebo	Placebo subcutaneous implant

Timeline

• Start date: 2007-05-01
• Primary completion: 2009-12-09
• Completion: 2009-12-09
• First posted: 2019-08-12
• Last updated: 2019-10-10
• Results posted: 2019-10-10