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

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

Official Title: A Phase II, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the following dosing regimen:

  • Group A will be administered afamelanotide implants on Days 0, 60 and 120
  • Group B will be administered placebo implants on Days 0, 60 and 120

To determine eligibility for study inclusion, patients will undergo a screening evaluation 7 to 14 days prior to the administration of the first dose. The number and severity of phototoxic reactions will be determined Days 60, 120, and 180. Quality of life will be measured every 7 days, beginning at Day 0 until Day 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.


Detailed 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 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.


Sponsor: Clinuvel Pharmaceuticals Limited

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

Original Primary Outcome: Same as current

Current 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 ]


Original Secondary Outcome: Same as current

Information By: Clinuvel Pharmaceuticals Limited

Dates:
Date Received: March 30, 2010
Date Started: April 2010
Date Completion:
Last Updated: March 21, 2013
Last Verified: March 2013