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

Phase 2

Recruiting

• Conditions: Xeroderma Pigmentosum
• Interventions: Drug: Afamelanotide

• Registration Number: NCT05159752
• Lead Sponsor: Clinuvel Europe Limited

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-10-19
• Study First Submitted: 2021-11-09
• Study First Submitted QC: 2021-12-02
• Study First Posted: 2021-12-16
• Status Verified: 2023-06
• Last Update Submitted: 2023-06-15
• Last Update Posted: 2023-06-18
• Primary Completion: 2024-03
• Study Completion: 2024-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Afamelanotide	Afamelanotide	-

Primary Outcome Measures

Name	Time	Method
Change in minimal erythema dose (MED).	From Baseline to Day 76.	MED is the lowest dose of UV light that causes reddening of the skin.

Secondary Outcome Measures

Name	Time	Method
Change in skin disease severity (C).	From Baseline to Day 238.	The higher the score, the more severe the disease.
Change in UV-induced DNA damage and repair capacity.	From Baseline to Day 76.	Analysis of UV photoproducts and DNA repair mechanisms.
Change in skin disease severity (A).	From Baseline to Day 238.	The higher the score, the more severe the disease.
Change in quality of life assessed by a disease specific tool (A)	From Baseline to Day 238.	Higher scores represent worse health-related quality of life.
Change in quality of life assessed by a validated global quality of life tool (B)	From Baseline to Day 238.	Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
Change in skin disease severity (B).	From Baseline to Day 238.	The higher the score, the more severe the disease.
Change in dermal melanin density.	From Baseline to Day 238.	Non-invasive quantitative skin reflectance measurement.

Trial Locations

Locations (1)

CLINUVEL investigative site; 🇩🇪 Regensburg, Germany