A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V

Phase 2

Recruiting

• Conditions: Xeroderma Pigmentosum
• Interventions: Drug: Afamelanotide

• Registration Number: NCT05370235
• Lead Sponsor: Clinuvel Europe Limited

Brief Summary

The CUV152 study will evaluate the safety of afamelanotide in XP-C and XP-V 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: 2022-03-28
• Study First Submitted: 2022-04-21
• Study First Submitted QC: 2022-05-06
• Study First Posted: 2022-05-11
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-20
• Last Update Posted: 2023-09-21
• Primary Completion: 2024-06
• Study Completion: 2024-12

Recruitment & Eligibility

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

Inclusion Criteria

• Male or female patient with a molecular-genetically confirmed diagnosis of XP-C or XP-V;
• 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;
• Existing melanoma;
• Female who is pregnant or lactating;
• Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures;
• Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures;
• Use of any other prior and concomitant therapy which may interfere with the objective of the study;
• Participation in a clinical trial for an investigational agent.

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) in patients with XP-C.	From baseline to day 76.	MED is the lowest dose of UV light that causes reddening of the skin.
Change in MED in patients with XP-V.	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
Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-C.	From baseline to day 76.	Analysis of UV photoproducts from skin samples.
Changes in UV-induced DNA damage repair capacity, as assessed by quantification of DNA repair mechanisms in patients with XP-C.	From baseline to day 76.	Analysis of DNA repair mechanisms from skin samples.
Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-V.	From baseline to day 76.	Analysis of UV photoproducts from skin samples.
Changes in UV-induced DNA damage repair capacity, as assessed by quantification of DNA repair mechanisms in patients with XP-V.	From baseline to day 76.	Analysis of DNA repair mechanisms from skin samples.
Change in skin disease severity in patients with XP-C (A).	From baseline to day 76.	The higher the score, the more severe the disease.
Change in skin disease severity in patients with XP-V (A)	From baseline to day 76.	The higher the score, the more severe the disease.
Change in skin disease severity in patients with XP-C (B).	From baseline to day 76.	The higher the score, the more severe the disease.
Change in skin disease severity in patients with XP-V (B).	From baseline to day 76.	The higher the score, the more severe the disease.
Change in skin disease severity in patients with XP-C (C).	From baseline to day 76.	The higher the score, the more severe the disease.
Change in skin disease severity in patients with XP-V (C).	From baseline to day 76.	The higher the score, the more severe the disease.
Change in quality of life assessed by a disease specific tool (A) in patients with XP-C.	From baseline to day 76.	Higher scores represent worse health-related quality of life.
Change in quality of life assessed by a disease specific tool (A) in patients with XP-V.	From baseline to day 76.	Higher scores represent worse health-related quality of life.
Change in quality of life assessed by a validated global quality of life tool (B) in patients with XP-C.	From baseline to day 76.	Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
Change in quality of life assessed by a validated global quality of life tool (B) in patients with XP-V.	From baseline to day 76.	Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.

Trial Locations

Locations (1)

CLINUVEL Investigational site; 🇪🇸 Clinuvel Investigational Site, Spain