Erythropoietic Protoporphyria Clinical Trial
Official title:
A Phase III, Multicentre, Double-Blind, Randomized, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With 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). Between 75 and 100 eligible patients will be enrolled.
Patients 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
The number and severity of phototoxic reactions, the type and duration of sun exposure,
treatment-emergent adverse events and the use of concomitant medication will be recorded by
patients in study diaries between Days 0 and 180. Quality of life will be measured using the
DLQI and EPP-QoL at Days 0, 60, 120 and 180. Participants will visit the clinic on Days 60,
120 and 180 for assessments of adverse events.
A subset of patients will be photoprovoked on the lower back and dorsal surface of the hand
and the minimal symptom dose (MSD) will be determined on Days 0, 30, 60, 90 and 120.
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) and is available in Europe.
The purpose of this study is to look at the type and duration of sun exposure 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 620 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.
This study aims to confirm the photoprotective properties if afamelanotide demonstrated in
the earlier Phase II and phase III studies.
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