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

A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)

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 Overview

• Status: Recruiting
• Conditions: Xeroderma Pigmentosum
• Intervention / Treatment: Drug: Afamelanotide

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Head of Clinical Operations; Phone Number: +441372860765; Email: mail@clinuvel.com

Study Locations

• Germany
  • Regensburg, Germany
    • Recruiting
    • CLINUVEL investigative site
    • Contact: Head of Clinical Operations; Email: mail@clinuvel.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Afamelanotide	Drug: Afamelanotide; Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Clinuvel Europe Limited

Study record dates

Study Major Dates

• Study Start (Actual): October 19, 2021
• Primary Completion (Estimated): March 1, 2024
• Study Completion (Estimated): October 1, 2024

Study Registration Dates

• First Submitted: November 9, 2021
• First Submitted That Met QC Criteria: December 2, 2021
• First Posted (Actual): December 16, 2021

Study Record Updates

• Last Update Posted (Estimated): June 16, 2023
• Last Update Submitted That Met QC Criteria: June 15, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Skin Diseases; Neoplasms; Congenital Abnormalities; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Skin Diseases, Genetic; DNA Repair-Deficiency Disorders; Precancerous Conditions; Skin Abnormalities; Pigmentation Disorders; Keratosis; Photosensitivity Disorders; Ichthyosis; Xeroderma Pigmentosum; Dermatologic Agents; Afamelanotide
• Other Study ID Numbers: CUV156

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes