Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)

• Status: Recruiting
• Trial end date: 2024-03-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective is to evaluate the impact of afamelanotide on the severity of skin disease in patients with Variegate Porphyria (VP). The secondary objectives are to evaluate the safety and tolerability of afamelanotide in patients with VP and evaluate the impact of afamelanotide on the quality of life of patients with VP.

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

Details

• Lead Sponsor: Clinuvel (UK) Ltd.
• Treatments: Afamelanotide

Criteria

Inclusion Criteria:

• Male or female patients with confirmed diagnosis of VP.

• Patients with VP-related skin symptoms.

• Aged 18-70 years.

Exclusion Criteria:

• Allergy to afamelanotide or polymer or to local anaesthetic to be used if applicable.

• Had two or more acute attacks of hepatic porphyria, as defined by the occurrence or acute porphyric symptoms within 12 months prior to the Screening period.

• Individual history of malignant or premalignant skin lesions.

• Individual or family history of melanoma.

• Presence of severe hepatic disease.

• Renal impairment.

• Female who is pregnant or lactating.

• Females of child-bearing potential not using adequate contraceptive or a life-style excluding pregnancy.

• Sexually active man with a partner of child-bearing potential not using adequate contraceptive measures.

• Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening period.

• Participation in a clinical trial within 30 days prior to the Screening period.