A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

Study Overview
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Brief Summary
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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.

Official Title
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A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

Conditions
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Erythropoietic Protoporphyria

Intervention / Treatment
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* Drug: Afamelanotide


Participation Criteria
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Eligibility Criteria
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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 clinical trial for an investigational agent within 30 days prior to the screening visit; prior and concomitant therapy with medications which may interfere with the objectives of the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Afamelanotide<br> | Drug: Afamelanotide<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life. | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | 
| Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) |