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

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 (XP)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05159752
• Other study ID #: CUV156
• Status: Recruiting
• Phase: Phase 2
• Start date: October 19, 2021
• Est. completion date: October 2024

Study information

• Verified date: June 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 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 6
• Est. completion date: October 2024
• Est. primary completion date: March 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;
• 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.

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
Germany	CLINUVEL investigative site	Regensburg

Sponsors (1)

Lead Sponsor	Collaborator
Clinuvel Europe Limited

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in UV-induced DNA damage and repair capacity.	Analysis of UV photoproducts and DNA repair mechanisms.	From Baseline to Day 76.
Secondary	Change in skin disease severity (A).	The higher the score, the more severe the disease.	From Baseline to Day 238.
Secondary	Change in skin disease severity (B).	The higher the score, the more severe the disease.	From Baseline to Day 238.
Secondary	Change in skin disease severity (C).	The higher the score, the more severe the disease.	From Baseline to Day 238.
Secondary	Change in dermal melanin density.	Non-invasive quantitative skin reflectance measurement.	From Baseline to Day 238.
Secondary	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.
Secondary	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.