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

NCT ID: NCT05854784

Last Updated: 2025-04-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-03-28
• Study Completion Date: 2023-12-28

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Afamelanotide

Group Type: EXPERIMENTAL

Afamelanotide 16 MG

Intervention Type: DRUG

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

Interventions

Afamelanotide 16 MG

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

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Clinuvel Pharmaceuticals Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

CLINUVEL investigational site

Rotterdam, , Netherlands

Countries

Netherlands

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

CUV040

Identifier Type: -

Identifier Source: org_study_id