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

Xeroderma Pigmentosum Clinical Trial

Official title:

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 C and V (XPC and XPV)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05370235
• Other study ID #: CUV152
• Status: Recruiting
• Phase: Phase 2
• Start date: March 28, 2022
• Est. completion date: December 2024

Study information

• Verified date: September 2023
• Source: Clinuvel Pharmaceuticals Limited
• Contact: Head of Clinical Operations
• Phone: +441372860765
• Email: mail[@]clinuvel.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 6
• Est. completion date: December 2024
• Est. primary completion date: June 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Ichthyosis
• Xeroderma Pigmentosum

Intervention

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

Locations

Country	Name	City	State
Belgium	CLINUVEL Investigational site	Clinuvel Investigational Site
Spain	CLINUVEL Investigational site	Clinuvel Investigational Site

Sponsors (1)

Lead Sponsor	Collaborator
Clinuvel Europe Limited

Countries where clinical trial is conducted

Belgium,  Spain, 

Outcome

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