NCT05854784

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

Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)-Related Skin Disease.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Clinuvel Pharmaceuticals Limited · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Variegate Porphyria

Interventions

Type	Name	Description
DRUG	Afamelanotide 16 MG	Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.

Timeline

• Start date: 2023-03-28
• Primary completion: 2023-12-28
• Completion: 2023-12-28
• First posted: 2023-05-11
• Last updated: 2025-04-27
• Results posted: 2025-04-27

Locations

1 site across 1 country: Netherlands

Regulatory

• FDA-regulated drug study