Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Status:
Completed
Trial end date:
2011-04-01
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). 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 using the EPP specific questionnaire (EPP-QoL) every 60 days and the DLQI
questionnaire 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.