A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP).
- Conditions
- Erythropoietic Protoporphyria (EPP)10027424
- Registration Number
- NL-OMON35418
- Lead Sponsor
- Clinuvel Pharmaceuticals Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 20
The participants have to fulfill all of the following criteria for study participation:
- Male or female subjects with a diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes) of sufficient severity that they have requested treatment to alleviate their symptoms
- Aged 18 - 70 years (inclusive)
- Written informed consent prior to the performance of any study-specific procedures.
Any of the following criteria will exclude the patient from the study:
- Any allergy to afamelanotide or the polymer contained in the implant or to lignocaine or other local anaesthetic to be used during the administration of 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 12 months).
- Patient assessed as not suitable for the study in the opinion of the Investigator (e.g. noncompliance history allergic to local anaesthetics, faints when given injections or giving blood).
- 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).
- 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 of 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p> - To determine whether afamelanotide can reduce the severity of phototoxic<br /><br>reactions in patients with EPP</p><br>
- Secondary Outcome Measures
Name Time Method <p>- To determine whether afamelanotide can reduce the number of phototoxic<br /><br>reactions in patients with EPP<br /><br>- To evaluate the safety and tolerability of afamelanotide by measuring<br /><br>treatment-emergent adverse events (AEs)<br /><br>- To determine whether afamelanotide can improve the quality of life of EPP<br /><br>patients<br /><br>- To determine the effect of afamelanotide on free protoporphyrin IX levels</p><br>