A Study to Evaluate the Pharmacokinetics of Afamelanotide in Patients with Erythropoietic Protoporphyria (EPP).

Phase 1

Recruiting

• Conditions: Erythropoietic Protoporphyria (EPP); MedDRA version: 21.1Level: LLTClassification code: 10015289Term: Erythropoietic protoporphyria Class: 10010331; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2023-507311-35-00
• Lead Sponsor: Clinuvel Europe Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-10-16
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 34

Inclusion Criteria

Fourteen adult EPP adult patients aged between 18 and 70 years (inclusive) and fourteen EPP adolescents aged between 12 and 17 (inclusive)., Agree not to use any medications, Able to understand and sign the written Informed Consent Form ((and parents or legal representative in case of adolescent patients), Able and willing to follow the Protocol requirements

Exclusion Criteria

Any personal or direct family history of melanoma., Any factor that may interfere with the skin reflectance measurements., Current cigarette smokers., History of drug abuse, licit or illicit., Regularly drinks more than four standard drinks of alcohol per day., Participation in any clinical trial during the 60 days before the study screening period., Has received afamelanotide in the last 60 days., Has donated 400 mL or more of blood or had significant blood loss during the eight weeks preceding screening., Any significant history of allergy and/or sensitivity to any of the contents of study drug product., Any significant history of allergy and/or sensitivity to lignocaine or other local anaesthetics if used., Any significant illness during the four weeks before the study screening period., Any evidence of hepatic or renal impairment., Any contraindication to blood sampling., Female who is pregnant or lactating., Females of child-bearing potential not using adequate contraceptive measures or a life-style excluding pregnancy, for up to three months after the implant administration., Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the pharmacokinetics of afamelanotide following administration of afamelanotide to adolescent and adult EPP patients.<br>To determine the relative comparability of the PK profiles of afamelanotide in adolescent and adult EPP patients.;Secondary Objective: To determine the safety and tolerability of afamelanotide in adolescent and adult EPP patients.;Primary end point(s): Cmax, AUC(0-t)