A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) (NCT04578496) | Clinical Trial Compass
CompletedPhase 3
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
16 participantsStarted 2011-07-11
Plain-language summary
The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP patients.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* male or female subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies;
* aged 18-75 years (inclusive);
* provide written informed patient consent prior to the performance of any study-specific procedure.
Exclusion Criteria:
* any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant;
* any allergy to lignocaine or other local anaesthetic to be used during the administration of the study medication;
* EPP patients with significant hepatic involvement;
* personal history of melanoma or dysplastic nevus syndrome;
* current Bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions;
* any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations;
* acute history of drug or alcohol abuse (in the last 12 months);
* female who is pregnant (confirmed by positive serum β-Human Chorionic Gonadotropin (HCG) pregnancy test prior to baseline) or lactating;
* females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, intrauterine device);
* sexually active men with partners of child-bearing potential not using barrier contraception during the trial and for a period of three months thereafter;
* participation in a clinica…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Timeframe: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
2
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Timeframe: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)