Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients withXeroderma Pigmentosum (XP)

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 6

Inclusion Criteria

-Male or female patient with a molecular-genetically confirmed diagnosis of XP-C.
-Aged 18-75 years.
-Providing written Informed Consent prior to the performance of any study-specific procedure.
-Willing and able to comply with the conditions specified in the protocol and study procedures, in the opinion of the Investigator.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

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.
-Existing melanoma
-Female who is pregnant (confirmed by positive urine ß-HCG pregnancy test) or lactating.
-Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, 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 adequate 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.
-Not suitable for trial participation in the opinion of the Investigator.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: •Evaluate the impact of afamelanotide on minimal erythema dose (MED) in patients with XP;Secondary Objective: •Evaluate the impact of afamelanotide on UV-induced DNA damage and repair capacity in patients with XP.<br>•Evaluate the safety and tolerability of afamelanotide in patients with XP. <br>•Evaluate the impact of afamelanotide on the skin disease severity of patients with XP.<br>•Evaluate the impact of afamelanotide on the skin melanin density of patients with XP.<br>•Evaluate the impact of afamelanotide on the quality of life of patients with XP.;Primary end point(s): •The change in MED (analysis will compare the individual fold increase in MED from Day 0 to the post-treatment assessment at Day 76). <br><br>;Timepoint(s) of evaluation of this end point: from Day 0 to the post-treatment assessment at Day 76

Secondary Outcome Measures

Name	Time	Method

Related Trials