Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

• Status: Completed
• Trial end date: 2011-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Clinuvel Pharmaceuticals Limited
• Treatments: Afamelanotide; alpha-MSH

Criteria

Inclusion Criteria:

• Male or female subjects with characteristic photosensitivity of EPP symptoms and positive diagnosis of EPP confirmed by laboratory result of elevated total protoporphyrin IX.

• Aged 18 years old and above (inclusive).

• Able to understand and sign the written Informed Consent Form.

• Willing to take precautions to prevent pregnancy until completion of the study (Day 180).

Exclusion Criteria:

• Any allergy to afamelanotide or the polymer contained in the implant or to lidocaine or other local anesthetic 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 other photodermatosis such as PLE, DLE or solar urticaria.

• 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 6 months).

• Patient assessed as not suitable for the study in the opinion of the Investigator (e.g. noncompliance history, allergic to local anesthetics, faints when given injections or giving blood).

• 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, including drugs that cause photosensitivity or skin pigmentation within 60 days prior to the screening visit.

• Female who is pregnant (confirmed by positive serum β-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).