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

Clinuvel Pharmaceuticals Limited6 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Variegate Porphyria

Interventions

Afamelanotide 16 MGDRUG

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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.

Locations (1)

CLINUVEL investigational site

Rotterdam, Netherlands

Outcomes

Primary Outcomes

The Change in Disease Severity in Patients With VP as Measured by CGIC.

The higher the score, the less severe the disease. Scale ranges from +3 to -3.

Time frame: From baseline to Day 168

Secondary Outcomes

The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.

A lower score indicates a reduced severity of the disease.Scale ranges from 0 to 10.

Time frame: Median change from baseline to Day 168.

The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.

A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 4.

Time frame: Median change from baseline to Day 168

The Change in Disease Severity in Patients With VP as Measured by PGIC.

The higher the score, the less severe the disease. Score ranges from +3 to -3.

Time frame: Median change from baseline to Day 168

The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.

A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 10.

Time frame: Median change from baseline to Day 168

The Change in Number of New Skin Lesions Formed.

A lower number indicates a reduced severity of the disease.

Time frame: Median change from baseline to Day 168.

The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.

Higher score indicates greater impairment and less productivity. Score is percentage impairment.

Data from ClinicalTrials.gov

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