Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|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 Erythropoietic Protoporphyria (EPP)|
- Severity of phototoxic reaction measured by visual analogue scale [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Number of phototoxic reactions [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Quality of life measured by patient completed questionnaire [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Free protoporphyrin IX level [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Treatment emergent adverse events [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||April 2010|
|Study Completion Date:||April 2011|
|Primary Completion Date:||April 2011 (Final data collection date for primary outcome measure)|
One 16mg subcutaneous implant every 2 months for 6 months.
|Placebo Comparator: Placebo||
One placebo subcutaneous implant every 2 months for 6 months
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.
|United States, Alabama|
|University of Alabama|
|Birmingham, Alabama, United States, 35294|
|United States, California|
|University of California, San Francisco|
|San Francisco, California, United States, 94143|
|United States, New York|
|New York, New York, United States, 10029|
|United States, North Carolina|
|Carolina's Medical Center Cannon Research|
|Charlotte, North Carolina, United States, 29203|
|United States, Texas|
|University of Texas|
|Galveston, Texas, United States, 77555|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84132|
|Principal Investigator:||Robert Desnick, MD||Mt. Sinai|