Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
|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 Erythropoietic Protoporphyria (EPP)|
- Severity of phototoxic reaction measured by visual analogue scale [ Time Frame: 9 months ] [ Designated as safety issue: No ]
- Number of phototoxic reactions [ Time Frame: 9 months ] [ Designated as safety issue: No ]
- Quality of life measured by patient completed questionnaire [ Time Frame: 9 months ] [ Designated as safety issue: No ]
- Free protoporphyrin IX level [ Time Frame: 9 months ] [ Designated as safety issue: No ]
- Treatment emergent adverse events [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||September 2009|
|Study Completion Date:||May 2011|
|Primary Completion Date:||May 2011 (Final data collection date for primary outcome measure)|
One 16mg subcutaneous implant every 2 months for 9 months.
Other Name: CUV1647
|Placebo Comparator: Placebo||
One 16mg subcutaneous implant every 2 months for 9 months.
To determine whether afamelanotide can reduce the severity of phototoxic reactions in patients with EPP.
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.
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.
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 following dosing regime:
- Group A will be administered active implants on Days 0, 60, 120, 180 and 240.
- Group B will be administered placebo implants on Days 0, 60, 120, 180 and 240.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00979745
|HUS:n Iho-ja allergiasairaala (Skin and Allergy Hospital)|
|Centre Français des Porphyries, Hôpital Louis Mourier|
|Colombes, Cedex, France, 92701|
|Department of Dermatology , Heinrich-Heine-University Duesseldorf|
|Duesseldorf, Germany, 40225|
|Beaumont Hospital, Department of Dermatology|
|Dublin, Ireland, 9|
|Academisch Ziekenhuis Maastricht|
|Erasmus Medical Center|
|St Woolos Hospital|
|Newport, Wales, United Kingdom|
|Photobiology Unit - Hope Hospital, University of Manchester|
|Manchester, United Kingdom, M6 8HD|
|Principal Investigator:||Alex Anstey, MBBS, FRCP||St Woolos Hospital, Newport|
|Principal Investigator:||Jorge Frank, MD, PhD||Academisch Ziekenhuis Maastricht|
|Principal Investigator:||Raili Kauppinen, MD, PhD||University Central Hospital of Helsinki|
|Principal Investigator:||Eric JG Sijbrands, MD, PhD||Erasmus Medical Center, Rotterdam|
|Principal Investigator:||Jean-Charles Deybach, MD. PhD||Centre Francais des Porphyries, Hopital Louis Mourier, Colombes, France|
|Principal Investigator:||Sandra Hanneken, MD||Heinrich-Heine Universität, Düsseldorf, Germany|
|Principal Investigator:||Gillian M Murphy, MD PhD||Beaumont Hospital, Dublin, Ireland|
|Principal Investigator:||Lesley E Rhodes, MD PhD||Hope Hospital, University of Manchester, UK|