Clinical Trial: Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With 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.


Detailed Summary:

PURPOSE:

To determine whether afamelanotide can reduce the severity of phototoxic reactions in patients with EPP.

THEORETICAL FRAMEWORK:

EPP is a genetic photosensitivity disorder where the mainstays of management are covering up from sunlight, systemic beta carotene and the use of controlled courses of UVR treatment. One of the mechanisms for the protective effects of UVR treatment is the increase in melanin content of the skin. UVR treatment causes DNA damage to skin cells and increases the risk for skin cancers, hence it is unwise for this to be used on a recurring basis. Afamelanotide, through its ability to stimulate melanin production without causing the DNA damage associated with UVR treatment, appears to be a promising agent to combat this distressing disorder.

STUDY DESIGN:

This is a phase III, randomised, placebo controlled study to evaluate the safety and efficacy of subcutaneous implants of afamelanotide in patients suffering from EPP. The study will be performed in compliance with Good Clinical Practice (GCP) including the archiving of essential documents.

METHODOLOGY:

The target population consists of male and female participants. Up to 70 patients with diagnosed EPP (from past case history) and fulfilling the necessary inclusion/exclusion criteria will be enrolled. Potential study patients will be identified from each centre's records of patients with well characterised history (or documented diagnosis) of EPP.

Patients will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the fo
Sponsor: Clinuvel Pharmaceuticals Limited

Current Primary Outcome: Severity of phototoxic reaction measured by visual analogue scale [ Time Frame: 9 months ]

Original Primary Outcome: Severity of phototoxic reaction measured by visual analogue scale [ Time Frame: 6 months ]

Current Secondary Outcome:

  • Number of phototoxic reactions [ Time Frame: 9 months ]
  • Quality of life measured by patient completed questionnaire [ Time Frame: 9 months ]
  • Free protoporphyrin IX level [ Time Frame: 9 months ]
  • Treatment emergent adverse events [ Time Frame: 9 months ]


Original Secondary Outcome:

  • Number of phototoxic reactions [ Time Frame: 6 months ]
  • Quality of life measured by patient completed questionnaire [ Time Frame: 6 months ]
  • Free protoporphyrin IX level [ Time Frame: 6 months ]
  • Treatment emergent adverse events [ Time Frame: 6 months ]


Information By: Clinuvel Pharmaceuticals Limited

Dates:
Date Received: September 17, 2009
Date Started: September 2009
Date Completion:
Last Updated: August 3, 2011
Last Verified: August 2011