A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)

Clinuvel Europe Limited6 enrolled

Overview

The CUV156 study will evaluate the safety of afamelanotide in XP-C patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. It will assess whether SCENESSE® increases the amount of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Xeroderma Pigmentosum

Interventions

AfamelanotideDRUG

Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female patient with a molecular-genetically confirmed diagnosis of XP-C; * Aged 18-75 years. Exclusion Criteria: * Known allergy to afamelanotide or the polymer contained in the implant; * Presence of severe hepatic disease or hepatic impairment; * Renal impairment; * Any other medical condition which may interfere with the study protocol; * Female who is pregnant (confirmed by positive urine beta-Human chorionic gonadotropin pregnancy test) or lactating; * Females of child-bearing potential (pre-menopausal, not surgically sterile) not using highly effective contraceptive measures with a failure rate of less than 1% per year when used consistently and correctly (i.e. oral contraceptives, intrauterine device) or a life-style excluding pregnancy, for up to three months after the last implant administration; * Sexually active man with a partner of child-bearing potential (pre-menopausal, not surgically sterile) who is not using highly effective contraceptive measures, as described above; * Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening visit; * Participation in a clinical trial for an investigational agent within 30 days prior to the Screening visit.

Locations (1)

CLINUVEL investigative site

Regensburg, Germany

RECRUITING

Outcomes

Primary Outcomes

Change in minimal erythema dose (MED).

MED is the lowest dose of UV light that causes reddening of the skin.

Time frame: From Baseline to Day 76.

Secondary Outcomes

Change in UV-induced DNA damage and repair capacity.

Analysis of UV photoproducts and DNA repair mechanisms.

Time frame: From Baseline to Day 76.

Change in skin disease severity (A).

The higher the score, the more severe the disease.