Phase III Confirmatory Study in Erythropoietic Protoporphyria
Status:
Completed
Trial end date:
2013-07-01
Target enrollment:
Participant gender:
Summary
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.